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This multicenter, open-label, Phase 3 study with randomized and non-randomized arms is designed to investigate the efficacy, safety, and pharmacokinetics of emicizumab in participants with hemophilia A regardless of factor VIII (FVIII) inhibitor status. Participants greater than or equal to (≥)12 years old who received episodic therapy with FVIII or bypassing agents prior to study entry and experienced at least 5 bleeds over the prior 24 weeks will be randomized in a 2:2:1 ratio to the following regimens: Arm A: Emicizumab prophylaxis at 3 milligrams per kilogram (mg/kg) once every week (QW) subcutaneously (SC) for 4 weeks, followed by 1.5 mg/kg QW SC; Arm B: Emicizumab prophylaxis at 3 mg/kg QW SC for 4 weeks, followed by 6 mg/kg once every 4 weeks (Q4W) SC; and Arm C: No prophylaxis (control arm). In addition, pediatric participants less than (<)12 years old with hemophilia A and FVIII inhibitors who received episodic therapy with bypassing agents prior to study entry will be enrolled to Arm D: Emicizumab prophylaxis at 3 mg/kg QW SC for 4 weeks, followed by 1.5 mg/kg QW SC.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Arm C (Control): No Prophylaxis, Then Emicizumab | Active Comparator | Participants ≥12 years old with hemophilia A (with or without FVIII inhibitors) who are randomized to Arm C will not receive any prophylactic treatment for at least 24 weeks. After 24 weeks, participants will have the opportunity to switch to receive emicizumab prophylaxis at 3 mg/kg QW via SC injection for 4 weeks, followed by 6 mg/kg Q4W until marketing authorization as part of this study or a separate extension study, as long as they derive clinical benefit. Participants will continue to receive standard-of-care treatments on an episodic basis for the treatment of breakthrough bleeds during the study. |
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| Arm A: Emicizumab Prophylaxis at 1.5 mg/kg QW | Experimental | Participants ≥12 years old with hemophilia A (with or without FVIII inhibitors) who are randomized to Arm A will receive prophylactic emicizumab at a dose of 3 mg/kg via SC injection QW for first 4 weeks, followed by 1.5 mg/kg via SC injection Q4W for at least 24 weeks. After 24 weeks treatment, participants will be allowed to continue emicizumab until marketing authorization as part of this study or a separate extension study, as long as they derive clinical benefit. Participants will continue to receive standard-of-care treatments on an episodic basis for the treatment of breakthrough bleeds during the study. |
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| Arm B: Emicizumab Prophylaxis at 6 mg/kg Q4W | Experimental | Participants ≥12 years old with hemophilia A (with or without FVIII inhibitors) who are randomized to Arm B will receive prophylactic emicizumab at a dose of 3 mg/kg via SC injection QW for first 4 weeks, followed by 6 mg/kg via SC injection Q4W for at least 24 weeks. After 24 weeks treatment, participants will be allowed to continue emicizumab until marketing authorization as part of this study or a separate extension study, as long as they derive clinical benefit. Participants will continue to receive standard-of-care treatments on an episodic basis for the treatment of breakthrough bleeds during the study. |
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Emicizumab | Drug | Emicizumab will be administered via subcutaneous (SC) injection, as described for each treatment arm. |
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| Measure | Description | Time Frame |
|---|---|---|
| Model-Based Annualized Bleeding Rate for Treated Bleeds | The number of treated bleeds over the efficacy period was estimated as an annualized bleeding rate (ABR) using a negative binomial regression model, which accounts for different follow-up times. A treated bleed was defined as a bleed that was directly followed by a hemophilia medication reported to be a "treatment for bleed". The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed. Bleeds due to surgery/procedure were excluded. | From Baseline to at least 24 weeks |
| Mean Calculated Annualized Bleeding Rate for Treated Bleeds | The number of treated bleeds over the efficacy period is presented here as a calculated annualized bleeding rate (ABR) that was annualized for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25. A treated bleed was defined as a bleed that was directly followed by a hemophilia medication reported to be a "treatment for bleed". The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed. Bleeds due to surgery/procedure were excluded. | From Baseline to at least 24 weeks |
| Median Calculated Annualized Bleeding Rate for Treated Bleeds | The number of treated bleeds over the efficacy period is presented here as a calculated annualized bleeding rate (ABR) that was annualized for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25. A treated bleed was defined as a bleed that was directly followed by a hemophilia medication reported to be a "treatment for bleed". The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed. Bleeds due to surgery/procedure were excluded. | From Baseline to at least 24 weeks |
| Measure | Description | Time Frame |
|---|---|---|
| Model-Based Annualized Bleeding Rate for All Bleeds | The number of all bleeds over the efficacy period was estimated as an annualized bleeding rate (ABR) using a negative binomial regression model, which accounts for different follow-up times. In this outcome measure, all bleeds are included, irrespective of treatment with coagulation factors, with the following exception: bleeds due to surgery/procedure are excluded. As "all bleeds" comprises both treated and non-treated bleeds, the 72-hour rule was implemented separately for treated and non-treated bleeds. For treated bleeds, the 72-hour rule meant that two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed. For non-treated bleeds, the 72-hour rule was implemented by calculating a treatment-free period of 72 hours from the bleed itself. |
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Inclusion Criteria:
Inclusion Criteria for Arms A, B, and C:
Inclusion Criteria for Arm D:
Exclusion Criteria:
Exclusion Criteria for Arms A, B, and C:
Exclusion Criteria for Arm D:
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| Name | Affiliation | Role |
|---|---|---|
| Clinical Trials | Hoffmann-La Roche | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Beijing Children's Hospital, Capital Medical University | Beijing | 100045 | China | |||
| Peking Union Medical College Hospital |
For eligible studies, qualified researchers may request access to individual patient level clinical data. See Roche's commitment to transparency of clinical study information here: https://go.roche.com/data\_sharing
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A total of 76 patients were screened for eligibility, 6 of whom were deemed ineligible, and 70 participants were randomized to this study (Arms A, B, and C). Arm D was added later in a protocol amendment (Version 4) after the study had already started. For Arm D, a total of 16 patients were screened and 15 patients were enrolled.
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| ID | Title | Description |
|---|---|---|
| FG000 | Arm C: No Prophylaxis (Control), Then Emicizumab | Participants ≥12 years old with hemophilia A (with or without FVIII inhibitors) who were randomized to Arm C did not receive any prophylactic treatment for at least 24 weeks (Control). After 24 weeks, participants had the opportunity to switch to receive emicizumab prophylaxis at 3 mg/kg QW via SC injection for 4 weeks, followed by 6 mg/kg Q4W until marketing authorization, as part of this study or a separate extension study, as long as they derived clinical benefit. Participants continued to receive standard-of-care treatments on an episodic basis for the treatment of breakthrough bleeds during the study. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Apr 22, 2023 | Jul 17, 2023 |
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| Arm D: Emicizumab Prophylaxis at 1.5 mg/kg QW | Experimental | Participants <12 years old with hemophilia A and FVIII inhibitors who are enrolled to Arm D will receive prophylactic emicizumab at a dose of 3 mg/kg via SC injection QW for first 4 weeks, followed by 1.5 mg/kg via SC injection QW for at least 24 weeks. After 24 weeks treatment, participants will be allowed to continue emicizumab until marketing authorization as part of this study or a separate extension study, as long as they derive clinical benefit. Participants will continue to receive standard-of-care treatments on an episodic basis for the treatment of breakthrough bleeds during the study. |
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| From Baseline to at least 24 weeks |
| Mean Calculated Annualized Bleeding Rate for All Bleeds | The number of all bleeds over the efficacy period is presented here as a calculated annualized bleeding rate (ABR) that was annualized for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25. In this outcome measure, all bleeds are included, irrespective of treatment with coagulation factors, with the following exception: bleeds due to surgery/procedure are excluded. As "all bleeds" comprises both treated and non-treated bleeds, the 72-hour rule was implemented separately for treated and non-treated bleeds. For treated bleeds, the 72-hour rule meant that two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed. For non-treated bleeds, the 72-hour rule was implemented by calculating a treatment-free period of 72 hours from the bleed itself. | From Baseline to at least 24 weeks |
| Median Calculated Annualized Bleeding Rate for All Bleeds | The number of all bleeds over the efficacy period is presented here as a calculated annualized bleeding rate (ABR) that was annualized for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25. In this outcome measure, all bleeds are included, irrespective of treatment with coagulation factors, with the following exception: bleeds due to surgery/procedure are excluded. As "all bleeds" comprises both treated and non-treated bleeds, the 72-hour rule was implemented separately for treated and non-treated bleeds. For treated bleeds, the 72-hour rule meant that two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed. For non-treated bleeds, the 72-hour rule was implemented by calculating a treatment-free period of 72 hours from the bleed itself. | From Baseline to at least 24 weeks |
| Model-Based Annualized Bleeding Rate for Treated Spontaneous Bleeds | The number of treated spontaneous bleeds over the efficacy period was estimated as an annualized bleeding rate (ABR) using a negative binomial regression model, which accounts for different follow-up times. A treated spontaneous bleed was defined as a treated bleed (bleed directly followed by a hemophilia medication reported to be a "treatment for bleed") with no other known contributing factor such as trauma or procedure/surgery. The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed. Bleeds due to surgery/procedure were excluded. | From Baseline to at least 24 weeks |
| Mean Calculated Annualized Bleeding Rate for Treated Spontaneous Bleeds | The number of treated spontaneous bleeds over the efficacy period is presented here as a calculated annualized bleeding rate (ABR) that was annualized for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25. A treated spontaneous bleed was defined as a treated bleed (bleed directly followed by a hemophilia medication reported to be a "treatment for bleed") with no other known contributing factor such as trauma or procedure/surgery. The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed. Bleeds due to surgery/procedure were excluded. | From Baseline to at least 24 weeks |
| Median Calculated Annualized Bleeding Rate for Treated Spontaneous Bleeds | The number of treated spontaneous bleeds over the efficacy period is presented here as a calculated annualized bleeding rate (ABR) that was annualized for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25. A treated spontaneous bleed was defined as a treated bleed (bleed directly followed by a hemophilia medication reported to be a "treatment for bleed") with no other known contributing factor such as trauma or procedure/surgery. The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed. Bleeds due to surgery/procedure were excluded. | From Baseline to at least 24 weeks |
| Model-Based Annualized Bleeding Rate for Treated Joint Bleeds | The number of treated joint bleeds over the efficacy period was estimated as an annualized bleeding rate (ABR) using a negative binomial regression model, which accounts for different follow-up times. A treated joint bleed was defined as a bleed with type reported as "joint" based on at least one of the following symptoms: increasing swelling or warmth of the skin over the joint and/or increasing pain, decreased range of motion, or difficulty using the joint compared with baseline, and the bleed was directly followed by a hemophilia medication reported to be a "treatment for bleed". The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed. Bleeds due to surgery/procedure were excluded. | From Baseline to at least 24 weeks |
| Mean Calculated Annualized Bleeding Rate for Treated Joint Bleeds | The number of treated joint bleeds over the efficacy period is presented here as a calculated annualized bleeding rate (ABR) that was annualized for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25. A treated joint bleed was defined as a bleed with type reported as "joint" based on at least one of the following symptoms: increasing swelling or warmth of the skin over the joint and/or increasing pain, decreased range of motion, or difficulty using the joint compared with baseline, and the bleed was directly followed by a hemophilia medication reported to be a "treatment for bleed". The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed. Bleeds due to surgery/procedure were excluded. | From Baseline to at least 24 weeks |
| Median Calculated Annualized Bleeding Rate for Treated Joint Bleeds | The number of treated joint bleeds over the efficacy period is presented here as a calculated annualized bleeding rate (ABR) that was annualized for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25. A treated joint bleed was defined as a bleed with type reported as "joint" based on at least one of the following symptoms: increasing swelling or warmth of the skin over the joint and/or increasing pain, decreased range of motion, or difficulty using the joint compared with baseline, and the bleed was directly followed by a hemophilia medication reported to be a "treatment for bleed". The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed. Bleeds due to surgery/procedure were excluded. | From Baseline to at least 24 weeks |
| Model-Based Annualized Bleeding Rate for Treated Target Joint Bleeds | The number of treated target joint bleeds over the efficacy period was estimated as an annualized bleeding rate (ABR) using a negative binomial regression model, which accounts for different follow-up times. A treated target joint bleed was defined as a joint bleed in a target joint, which is a joint location where at least 3 bleeds have occurred over the last 24 weeks prior to study entry or an unresolved target joint (target joint that does not fulfil ≤2 bleeds into this joint within a consecutive 12-month period), and the bleed was directly followed by a hemophilia medication reported to be a "treatment for bleed". The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed. Bleeds due to surgery/procedure were excluded. | From Baseline to at least 24 weeks |
| Mean Calculated Annualized Bleeding Rate for Treated Target Joint Bleeds | The number of treated target joint bleeds over the efficacy period is presented here as a calculated annualized bleeding rate (ABR) that was annualized for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25. A treated target joint bleed was defined as a joint bleed in a target joint, which is a joint location where at least 3 bleeds have occurred over the last 24 weeks prior to study entry or an unresolved target joint (target joint that does not fulfil ≤2 bleeds into this joint within a consecutive 12-month period), and the bleed was directly followed by a hemophilia medication reported to be a "treatment for bleed". The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed. Bleeds due to surgery/procedure were excluded. | From Baseline to at least 24 weeks |
| Median Calculated Annualized Bleeding Rate for Treated Target Joint Bleeds | The number of treated target joint bleeds over the efficacy period is presented here as a calculated annualized bleeding rate (ABR) that was annualized for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25. A treated target joint bleed was defined as a joint bleed in a target joint, which is a joint location where at least 3 bleeds have occurred over the last 24 weeks prior to study entry or an unresolved target joint (target joint that does not fulfil ≤2 bleeds into this joint within a consecutive 12-month period), and the bleed was directly followed by a hemophilia medication reported to be a "treatment for bleed". The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed. Bleeds due to surgery/procedure were excluded. | From Baseline to at least 24 weeks |
| Intra-Participant Comparison of the Calculated Annualized Bleeding Rate for Treated Bleeds With Emicizumab Prophylaxis On-Study Versus With Previous Episodic Therapy Pre-Study | This is an intra-participant comparison of the annualized bleeding rate (ABR) for treated bleeds pre-study versus on-study in the non-interventional study (NIS) population previously treated with episodic therapy in NIS BH29768. The number of treated bleeds over the efficacy period is presented here as a calculated ABR that was annualized for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25. A treated bleed was defined as a bleed that was directly followed by a hemophilia medication reported to be a "treatment for bleed". The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed. Bleeds due to surgery/procedure were excluded. | Efficacy periods: At least 24 weeks prior to study entry (mean [min-max] for A+B NIS-Previous Episodic Therapy: 183 [169-221] days); and From Baseline to at least 24 weeks on study (mean [min-max] for A+B NIS-Emicizumab: 363 [324-422] days) |
| Intra-Participant Comparison of the Calculated Annualized Bleeding Rate for All Bleeds With Emicizumab Prophylaxis On-Study Versus With Previous Episodic Therapy Pre-Study | This is an intra-participant comparison of the annualized bleeding rate (ABR) for all bleeds pre-study versus on-study in the non-interventional study (NIS) population previously treated with episodic therapy in NIS BH29768. The ABR was calculated for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25. All bleeds are included, irrespective of treatment with coagulation factors, with the following exception: bleeds due to surgery/procedure are excluded. "All bleeds" comprises both treated and non-treated bleeds, and the 72-hour rule was implemented separately for each type. For treated bleeds, the 72-hour rule meant that two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed. For non-treated bleeds, the 72-hour rule was calculated as a treatment-free period of 72 hours from the bleed itself. | Efficacy periods: At least 24 weeks prior to study entry (mean [min-max] for A+B NIS-Previous Episodic Therapy: 183 [169-221] days); and From Baseline to at least 24 weeks on study (mean [min-max] for A+B NIS-Emicizumab: 363 [324-422] days) |
| Arms A, B, and C: Adjusted Mean Hemophilia A Quality of Life (Haem-A-QoL) Questionnaire Physical Health Domain Score at Week 25 in Participants ≥18 Years of Age | The Haem-A-QoL questionnaire has been developed and used in hemophilia A participants, assessing very specific aspects of dealing with hemophilia. The questionnaire consists of items pertaining to 10 domains: physical health, sports and leisure, school and work, dealing with hemophilia, family planning, feeling, relationships, treatment, view of yourself, and outlook for the future. The total score for each domain ranges from 0 to 100 with lower scores reflective of better quality of life. Physical Health domain score is reported (range 0 to 100, with lower scores reflective of better physical health). The means were derived via an analysis of covariance (ANCOVA) model and have been adjusted for the following co-variates: baseline score, treatment group, and treatment by baseline interaction term. | Baseline and Week 25 |
| Arms A, B, and C: Change From Baseline to Week 25 in Haem-A-QoL Questionnaire Physical Health Domain Score for Participants ≥18 Years of Age | The Haem-A-QoL questionnaire has been developed and used in hemophilia A participants, assessing very specific aspects of dealing with hemophilia. The questionnaire consists of items pertaining to 10 domains: physical health, sports and leisure, school and work, dealing with hemophilia, family planning, feeling, relationships, treatment, view of yourself, and outlook for the future. The total score for each domain ranges from 0 to 100 with lower scores reflective of better quality of life. Physical Health domain score is reported (range 0 to 100, with lower scores reflective of better physical health). | Baseline and Week 25 |
| Arms A, B, and C: Adjusted Mean Haem-A-QoL Questionnaire Total Score at Week 25 in Participants ≥18 Years of Age | The Haem-A-QoL questionnaire has been developed and used in hemophilia A participants, assessing very specific aspects of dealing with hemophilia. The questionnaire consists of items pertaining to 10 domains: physical health, sports and leisure, school and work, dealing with hemophilia, family planning, feeling, relationships, treatment, view of yourself, and outlook for the future. The total score for each domain ranges from 0 to 100 with lower scores reflective of better quality of life. Haem-A-QoL Total Score is the average of all domain scores and it ranges from 0 to 100, with lower scores reflective of better quality of life. The means were derived via an analysis of covariance (ANCOVA) model and have been adjusted for the following co-variates: baseline score, treatment group, and treatment by baseline interaction term. | Baseline and Week 25 |
| Arms A, B, and C: Change From Baseline to Week 25 in Haem-A-QoL Questionnaire Total Score for Participants ≥18 Years of Age | The Haem-A-QoL questionnaire has been developed and used in hemophilia A participants, assessing very specific aspects of dealing with hemophilia. The questionnaire consists of items pertaining to 10 domains: physical health, sports and leisure, school and work, dealing with hemophilia, family planning, feeling, relationships, treatment, view of yourself, and outlook for the future. The total score for each domain ranges from 0 to 100 with lower scores reflective of better quality of life. Haem-A-QoL Total Score is the average of all domain scores and it ranges from 0 to 100, with lower scores reflective of better quality of life. | Baseline and Week 25 |
| Arms A, B, and C: Hemophilia-Specific Quality of Life Short Form (Haemo-QoL-SF) Questionnaire Total Score at Baseline and Week 25 in Participants 12 to 17 Years of Age | The Haemo-QoL-SF contains 35 items covering nine dimensions considered relevant for the adolescent's (aged 12-17 years) health-related quality of life (HRQoL). Items are rated with five respective response options: never, seldom, sometimes, often, and always. The score ranges from 0 to 100, and a higher score is indicative of poorer HRQoL. According to the pre-specified statistical analysis plan, no statistical analyses were performed on the protocol-defined endpoints for the Haemo-QoL-SF due to the small number of adolescents randomized to Arms A, B and C. | Baseline and Week 25 |
| Arms A, B, and C: Adjusted Mean European Quality of Life 5-Dimensions-5 Levels Questionnaire (EQ-5D-5L) Questionnaire Visual Analog Scale (VAS) Score at Week 25 | EQ-5D-5L is a standardized, participant-rated questionnaire to assess health-related quality of life. The EQ-5D-5L includes 2 components: the EQ-5D-5L health state profile (descriptive system) and the EQ-5D-5L VAS. The VAS is designed to rate the participant's current health state on a scale from 0 to 100, where 0 represents the worst imaginable health state and 100 represents the best imaginable health state. The means were derived via an analysis of covariance (ANCOVA) model and have been adjusted for the following co-variates: baseline score, treatment group, and treatment by baseline interaction term. | Baseline and Week 25 |
| Arms A, B, and C: Change From Baseline in EQ-5D-5L Questionnaire VAS Score at Week 25 | The European Quality of Life 5-Dimensions-5 Levels Questionnaire (EQ-5D-5L) is a standardized, participant-rated questionnaire to assess health-related quality of life. The EQ-5D-5L includes 2 components: the EQ-5D-5L health state profile (descriptive system) and the EQ-5D-5L VAS. The VAS is designed to rate the participant's current health state on a scale from 0 to 100, where 0 represents the worst imaginable health state and 100 represents the best imaginable health state. | Baseline and Week 25 |
| Arms A, B, and C: Adjusted Mean EQ-5D-5L Index Utility Score at Week 25 | The European Quality of Life 5-Dimensions-5 Levels Questionnaire (EQ-5D-5L) is a standardized, participant-rated questionnaire to assess health-related quality of life. The EQ-5D-5L includes 2 components: the EQ-5D-5L health state profile (descriptive system) and the EQ-5D-5L VAS. The EQ-5D-5L health state profile is designed to record the participant's current health state in 5 domains: mobility, selfcare, usual activities, pain/discomfort, and anxiety/depression. Responses from the five domains are used to calculate a single index utility score on a scale of 0 to 1, with higher scores reflective of better quality of life. The means were derived via an analysis of covariance (ANCOVA) model and have been adjusted for the following co-variates: baseline score, treatment group, and treatment by baseline interaction term. | Baseline and Week 25 |
| Arms A, B, and C: Change From Baseline in EQ-5D-5L Index Utility Score at Week 25 | The European Quality of Life 5-Dimensions-5 Levels Questionnaire (EQ-5D-5L) is a standardized, participant-rated questionnaire to assess health-related quality of life. The EQ-5D-5L includes 2 components: the EQ-5D-5L health state profile (descriptive system) and the EQ-5D-5L VAS. The EQ-5D-5L health state profile is designed to record the participant's current health state in 5 domains: mobility, selfcare, usual activities, pain/discomfort, and anxiety/depression. Responses from the five domains are used to calculate a single index utility score on a scale of 0 to 1, with higher scores reflective of better quality of life. | Baseline and Week 25 |
| Arm D: Change From Baseline in Haemo-QoL-SF Questionnaire Physical Health and Total Scores at Week 25 in Participants 8 to 12 Years of Age | The Hemophilia-Specific Quality of Life Short Form (Haemo-QoL-SF) questionnaire contains 35 items covering nine dimensions considered relevant for the children's health-related quality of life (HRQoL). Items are rated with five respective response options: never, seldom, sometimes, often, and always. The scores range from 0 to 100, and higher scores are indicative of poorer HRQoL. | Baseline and Week 25 |
| Arm D: Caregiver-Reported Adapted Health-Related Quality of Life for Hemophilia Patients With Inhibitors (Adapted Inhib-QoL) Including Aspects of Caregiver Burden Questionnaire Transformed Total Score Over Time | Proxy assessment of HRQoL and aspects of caregiver burden for all children, regardless of age, were collected using the Adapted Inhib-QoL with Aspects of Caregiver Burden questionnaire. The questionnaire comprises two parts. The first part asks the caregiver for his/her opinion on the child's HRQoL (proxy HRQoL). The second part asks the caregiver to rate how the child's situation is for them (i.e., the impact of the child's disease and treatment on the caregiver). Items are rated with 5 respective response options: never, seldom, sometimes, often, and all the time. Scores range from 0 to 100, with lower scores reflective of better HRQoL. A total score is calculated as the sum of all of the items in the scale. | Baseline (Week 1) and Weeks 13 and 25 |
| Number of Participants With at Least One Adverse Event, Severity According to the World Health Organization (WHO) Toxicity Grading Scale, Primary Analysis of Randomized Comparison Arms | The number of participants experiencing at least one adverse event (AE), including all non-serious and serious AEs, is reported here. According to the ICH guideline for Good Clinical Practice, an AE is any untoward medical occurrence in a clinical investigation subject administered a pharmaceutical product, regardless of causal attribution. The terms "severe" and "serious" are not synonymous. A serious AE is any AE that is a significant medical event meeting any of the standard criteria. Severity refers to the intensity of an AE (e.g., rated as mild, moderate, or severe, or according to the World Health Organization [WHO] toxicity grading scale); the event itself may be of relatively minor medical significance. Severity and seriousness were independently assessed by the investigator for each reported AE. | From Baseline until primary cutoff date (at least 24 weeks): Arm C (Control) No Prophylaxis, median (range): 24.0 (23.9-28.0) weeks; Arms A & B Emicizumab, median (range): Arm A: 43.7 (28.1-60.3) weeks; Arm B: 46.1 (24.0-58.7) weeks |
| Number of Participants With at Least One Adverse Event, Severity According to the World Health Organization (WHO) Toxicity Grading Scale, Final Analysis | The number of participants experiencing at least one adverse event (AE), including all non-serious and serious AEs, is reported here. According to the ICH guideline for Good Clinical Practice, an AE is any untoward medical occurrence in a clinical investigation subject administered a pharmaceutical product, regardless of causal attribution. The terms "severe" and "serious" are not synonymous. A serious AE is any AE that is a significant medical event meeting any of the standard criteria. Severity refers to the intensity of an AE (e.g., rated as mild, moderate, or severe, or according to the World Health Organization [WHO] toxicity grading scale); the event itself may be of relatively minor medical significance. Severity and seriousness were independently assessed by the investigator for each reported AE. | From first dose of emicizumab until end of study: Arms A to C: up to 88 months; Arm D: up to 52.4 months |
| Number of Participants With at Least One Adverse Event Leading to Study Drug Discontinuation, Final Analysis | According to the ICH guideline for Good Clinical Practice, an adverse event (AE) is any untoward medical occurrence in a clinical investigation subject administered a pharmaceutical product, regardless of causal attribution. The terms "severe" and "serious" are not synonymous. A serious AE is any AE that is a significant medical event meeting any of the standard criteria. Severity refers to the intensity of an AE (e.g., rated as 1 = mild, 2 = moderate, 3 = severe, or 4 = potentially life-threatening according to the World Health Organization [WHO] toxicity grading scale); the event itself may be of relatively minor medical significance. Severity and seriousness were independently assessed by the investigator for each reported AE. | From first dose of emicizumab until end of study: Arms A to C: up to 88 months; Arm D: up to 52.4 months |
| Number of Participants With at Least One Systemic Hypersensitivity, Anaphylaxis, or Anaphylactoid Reaction, Severity According to the WHO Toxicity Grading Scale, Final Analysis | According to the ICH guideline for Good Clinical Practice, an adverse event (AE) is any untoward medical occurrence in a clinical investigation subject administered a pharmaceutical product, regardless of causal attribution. The terms "severe" and "serious" are not synonymous. A serious AE is any AE that is a significant medical event meeting any of the standard criteria. Severity refers to the intensity of an AE (e.g., rated as 1 = mild, 2 = moderate, 3 = severe, or 4 = potentially life-threatening according to the World Health Organization [WHO] toxicity grading scale); the event itself may be of relatively minor medical significance. Severity and seriousness were independently assessed by the investigator for each reported AE. | From first dose of emicizumab until end of study: Arms A to C: up to 88 months; Arm D: up to 52.4 months |
| Number of Participants With at Least One Thromboembolic Event, Severity According to the WHO Toxicity Grading Scale, Final Analysis | According to the ICH guideline for Good Clinical Practice, an adverse event (AE) is any untoward medical occurrence in a clinical investigation subject administered a pharmaceutical product, regardless of causal attribution. The terms "severe" and "serious" are not synonymous. A serious AE is any AE that is a significant medical event meeting any of the standard criteria. Severity refers to the intensity of an AE (e.g., rated as 1 = mild, 2 = moderate, 3 = severe, or 4 = potentially life-threatening according to the World Health Organization [WHO] toxicity grading scale); the event itself may be of relatively minor medical significance. Severity and seriousness were independently assessed by the investigator for each reported AE. | From first dose of emicizumab until end of study: Arms A to C: up to 88 months; Arm D: up to 52.4 months |
| Number of Participants With at Least One Thrombotic Microangiopathy, Severity According to the WHO Toxicity Grading Scale, Final Analysis | According to the ICH guideline for Good Clinical Practice, an adverse event (AE) is any untoward medical occurrence in a clinical investigation subject administered a pharmaceutical product, regardless of causal attribution. The terms "severe" and "serious" are not synonymous. A serious AE is any AE that is a significant medical event meeting any of the standard criteria. Severity refers to the intensity of an AE (e.g., rated as 1 = mild, 2 = moderate, 3 = severe, or 4 = potentially life-threatening according to the World Health Organization [WHO] toxicity grading scale); the event itself may be of relatively minor medical significance. Severity and seriousness were independently assessed by the investigator for each reported AE. | From first dose of emicizumab until end of study: Arms A to C: up to 88 months; Arm D: up to 52.4 months |
| Number of Participants With at Least One Injection-Site Reaction, Severity According to the WHO Toxicity Grading Scale, Final Analysis | According to the ICH guideline for Good Clinical Practice, an adverse event (AE) is any untoward medical occurrence in a clinical investigation subject administered a pharmaceutical product, regardless of causal attribution. The terms "severe" and "serious" are not synonymous. A serious AE is any AE that is a significant medical event meeting any of the standard criteria. Severity refers to the intensity of an AE (e.g., rated as 1 = mild, 2 = moderate, 3 = severe, or 4 = potentially life-threatening according to the World Health Organization [WHO] toxicity grading scale); the event itself may be of relatively minor medical significance. Severity and seriousness were independently assessed by the investigator for each reported AE. | From first dose of emicizumab until end of study: Arms A to C: up to 88 months; Arm D: up to 52.4 months |
| Number of Participants With Serum Chemistry Laboratory Abnormalities by Shift From Baseline to Highest WHO Grade Post-Baseline | The number of participants with abnormal shifts in laboratory chemistry parameters while on emicizumab throughout the study are provided below as shifts from baseline to the highest WHO grade post-baseline (1 = mild, 2 = moderate, 3 = severe, or 4 = potentially life-threatening). Not every laboratory abnormality qualifies as an adverse event. A laboratory test result must be reported as an adverse event if it meets any of the following criteria: Is accompanied by clinical symptoms; Results in a change in study treatment; Results in a medical intervention or a change in concomitant therapy; Is clinically significant in the investigator's judgment. It was the investigator's responsibility to review all laboratory findings. SGOT/AST = serum glutamic-oxaloacetic transaminase/aspartate aminotransferase; SGPT/ALT = serum glutamic-pyruvic transaminase/alanine aminotransferase | From first dose of emicizumab until end of study: Arms A to C: up to 88 months; Arm D: up to 52.4 months |
| Number of Participants With Hematology Laboratory Abnormalities by Shift From Baseline to Highest WHO Grade Post-Baseline | The number of participants with abnormal shifts in laboratory hematology parameters while on emicizumab throughout the study are provided below as shifts from baseline to the highest WHO grade post-baseline (1 = mild, 2 = moderate, 3 = severe, or 4 = potentially life-threatening). Not every laboratory abnormality qualifies as an adverse event. A laboratory test result must be reported as an adverse event if it meets any of the following criteria: Is accompanied by clinical symptoms; Results in a change in study treatment; Results in a medical intervention or a change in concomitant therapy; Is clinically significant in the investigator's judgment. It was the investigator's responsibility to review all laboratory findings. | From first dose of emicizumab until end of study: Arms A to C: up to 88 months; Arm D: up to 52.4 months |
| Change From Baseline in Body Temperature Over Time | The data for Arm C are from the emicizumab prophylaxis period and are labelled to correspond to the visit schedule of the other study arms. For Arm C, this is relative to the point at which participants switched to start receiving emicizumab (after having completed 24 weeks of no prophylaxis). | Baseline and Weeks 5, 25, 49, and 73 |
| Change From Baseline in Pulse Rate Over Time | The data for Arm C are from the emicizumab prophylaxis period and are labelled to correspond to the visit schedule of the other study arms. For Arm C, this is relative to the point at which participants switched to start receiving emicizumab (after having completed 24 weeks of no prophylaxis). | Baseline, Weeks 5, 25, 49, and 73 |
| Change From Baseline in Respiratory Rate Over Time | The data for Arm C are from the emicizumab prophylaxis period and are labelled to correspond to the visit schedule of the other study arms. For Arm C, this is relative to the point at which participants switched to start receiving emicizumab (after having completed 24 weeks of no prophylaxis). | Baseline, Weeks 5, 25, 49, and 73 |
| Change From Baseline in Systolic Blood Pressure Over Time | The data for Arm C are from the emicizumab prophylaxis period and are labelled to correspond to the visit schedule of the other study arms. For Arm C, this is relative to the point at which participants switched to start receiving emicizumab (after having completed 24 weeks of no prophylaxis). | Baseline, Weeks 5, 25, 49, and 73 |
| Change From Baseline in Diastolic Blood Pressure Over Time | The data for Arm C are from the emicizumab prophylaxis period and are labelled to correspond to the visit schedule of the other study arms. For Arm C, this is relative to the point at which participants switched to start receiving emicizumab (after having completed 24 weeks of no prophylaxis). | Baseline, Weeks 5, 25, 49, and 73 |
| Number of Participants by Post-Baseline Anti-Emicizumab Antibody (ADA) Status | Participants were considered anti-drug antibody (ADA)-positive if they were ADA-negative at baseline but developed an ADA response following study drug administration, or if they were ADA-positive at baseline and the titer of one or more post-baseline samples was at least 4-fold greater than the titer of the baseline sample. Participants were considered ADA-negative if they were ADA-negative at baseline and all post-baseline samples were negative following drug administration, or if they were ADA-positive at baseline but did not have any post-baseline (following drug administration) samples with a titer that was at least 4-fold greater than the titer of the baseline sample. | Samples taken at Baseline and at prespecified times post-baseline from first dose of emicizumab until data cutoff date, median (range) time of exposure to emicizumab: All Arms: 196.14 (20.1-222.1) weeks; Arm D only: 64.14 (61.1-67.3) weeks |
| Plasma Trough Concentration (Ctrough) of Emicizumab | The data for Arm C are from the emicizumab prophylaxis period and are labelled to correspond to the visit schedule of the other study arms. For Arm C, this is relative to the point at which participants switched to start receiving emicizumab (after having completed 24 weeks of no prophylaxis). | Arms A & D, QW (up to Week 49 for Arm D only): Weeks 2, 3, 4, 5, 7, 9, 13, 17, 21, 25, 33, 41, 49, 61, 73, 85, 97, 109, 121, and 133; Arms B & C, Q4W: Weeks 2, 3, 4, 5, 9, 13, 17, 21, 25, 37, 49, 61, 73, 85, 97, 109, 121, and 133 |
| Beijing |
| 100730 |
| China |
| Xiangya Hospital of Centre-South University | Changsha | 410008 | China |
| Southern Medical University Nanfang Hospital | Guangdong Province Guangzhou City | 510515 | China |
| Ruijin Hospital Shanghai Jiaotong University School of Medicine | Shanghai | 200025 | China |
| Tianjin Institute of Hematology & Blood Diseases Hospital | Tianjin | 300020 | China |
| Xiehe Hospital, Tongji Medical College Huazhong University of Science & Technology | Wuhan | 430022 | China |
| Tongji Hosp, Tongji Med. Col, Huazhong Univ. of Sci. & Tech | Wuhan | 430030 | China |
| Queen Mary Hospital | Hong Kong | Hong Kong |
| Prince of Wales Hospital | Shatin | Hong Kong |
| Penang General Hospital | George Town | Pulau Pinang | 10990 | Malaysia |
| Queen Elizabeth Hospital | Sabah | Sabah | 88586 | Malaysia |
| Ramathibodi Hospital | Bangkok | 10400 | Thailand |
| FG001 | Arm A: 1.5 mg/kg Emicizumab Prophylaxis QW | Participants ≥12 years old with hemophilia A (with or without FVIII inhibitors) who were randomized to Arm A received prophylactic emicizumab at a dose of 3 mg/kg via SC injection QW for the first 4 weeks, followed by 1.5 mg/kg via SC injection QW for at least 24 weeks. After 24 weeks of treatment, participants were allowed to continue receiving emicizumab until marketing authorization, as part of this study or a separate extension study, as long as they derived clinical benefit. Participants continued to receive standard-of-care treatments on an episodic basis for the treatment of breakthrough bleeds during the study. |
| FG002 | Arm B: 6 mg/kg Emicizumab Prophylaxis Q4W | Participants ≥12 years old with hemophilia A (with or without FVIII inhibitors) who were randomized to Arm B received prophylactic emicizumab at a dose of 3 mg/kg via SC injection QW for the first 4 weeks, followed by 6 mg/kg via SC injection Q4W for at least 24 weeks. After 24 weeks of treatment, participants were allowed to continue receiving emicizumab until marketing authorization, as part of this study or a separate extension study, as long as they derived clinical benefit. Participants continued to receive standard-of-care treatments on an episodic basis for the treatment of breakthrough bleeds during the study. |
| FG003 | Arm D: 1.5 mg/kg Emicizumab Prophylaxis QW | Participants <12 years old with hemophilia A and FVIII inhibitors who were enrolled to Arm D received prophylactic emicizumab at a dose of 3 mg/kg via SC injection QW for the first 4 weeks, followed by 1.5 mg/kg via SC injection QW for at least 24 weeks. After 24 weeks of treatment, participants were allowed to continue receiving emicizumab until marketing authorization, as part of this study or a separate extension study, as long as they derived clinical benefit. Participants continued to receive standard-of-care treatments on an episodic basis for the treatment of breakthrough bleeds during the study. |
| Completed 24 Weeks in the Study |
|
| Completed Treatment With Emicizumab |
|
| COMPLETED |
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| NOT COMPLETED |
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|
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | Arm C: No Prophylaxis (Control), Then Emicizumab | Participants ≥12 years old with hemophilia A (with or without FVIII inhibitors) who were randomized to Arm C did not receive any prophylactic treatment for at least 24 weeks (Control). After 24 weeks, participants had the opportunity to switch to receive emicizumab prophylaxis at 3 mg/kg QW via SC injection for 4 weeks, followed by 6 mg/kg Q4W until marketing authorization, as part of this study or a separate extension study, as long as they derived clinical benefit. Participants continued to receive standard-of-care treatments on an episodic basis for the treatment of breakthrough bleeds during the study. |
| BG001 | Arm A: 1.5 mg/kg Emicizumab Prophylaxis QW | Participants ≥12 years old with hemophilia A (with or without FVIII inhibitors) who were randomized to Arm A received prophylactic emicizumab at a dose of 3 mg/kg via SC injection QW for the first 4 weeks, followed by 1.5 mg/kg via SC injection QW for at least 24 weeks. After 24 weeks of treatment, participants were allowed to continue receiving emicizumab until marketing authorization, as part of this study or a separate extension study, as long as they derived clinical benefit. Participants continued to receive standard-of-care treatments on an episodic basis for the treatment of breakthrough bleeds during the study. |
| BG002 | Arm B: 6 mg/kg Emicizumab Prophylaxis Q4W | Participants ≥12 years old with hemophilia A (with or without FVIII inhibitors) who were randomized to Arm B received prophylactic emicizumab at a dose of 3 mg/kg via SC injection QW for the first 4 weeks, followed by 6 mg/kg via SC injection Q4W for at least 24 weeks. After 24 weeks of treatment, participants were allowed to continue receiving emicizumab until marketing authorization, as part of this study or a separate extension study, as long as they derived clinical benefit. Participants continued to receive standard-of-care treatments on an episodic basis for the treatment of breakthrough bleeds during the study. |
| BG003 | Arm D: 1.5 mg/kg Emicizumab Prophylaxis QW | Participants <12 years old with hemophilia A and FVIII inhibitors who were enrolled to Arm D received prophylactic emicizumab at a dose of 3 mg/kg via SC injection QW for the first 4 weeks, followed by 1.5 mg/kg via SC injection QW for at least 24 weeks. After 24 weeks of treatment, participants were allowed to continue receiving emicizumab until marketing authorization, as part of this study or a separate extension study, as long as they derived clinical benefit. Participants continued to receive standard-of-care treatments on an episodic basis for the treatment of breakthrough bleeds during the study. |
| BG004 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | Years |
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| Age, Customized | Count of Participants | Participants |
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| Sex: Female, Male | Count of Participants | Participants |
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| Race/Ethnicity, Customized | Count of Participants | Participants |
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| Race/Ethnicity, Customized | Count of Participants | Participants |
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| Factor VIII (FVIII) Inhibitor Status at Study Entry | Count of Participants | Participants |
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| Categorical Number of Bleeds (<9 or ≥9) in the Past 24 Weeks Prior to Study Entry | Count of Participants | Participants |
|
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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| Secondary | Model-Based Annualized Bleeding Rate for All Bleeds | The number of all bleeds over the efficacy period was estimated as an annualized bleeding rate (ABR) using a negative binomial regression model, which accounts for different follow-up times. In this outcome measure, all bleeds are included, irrespective of treatment with coagulation factors, with the following exception: bleeds due to surgery/procedure are excluded. As "all bleeds" comprises both treated and non-treated bleeds, the 72-hour rule was implemented separately for treated and non-treated bleeds. For treated bleeds, the 72-hour rule meant that two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed. For non-treated bleeds, the 72-hour rule was implemented by calculating a treatment-free period of 72 hours from the bleed itself. | All participants, which includes those randomized to Arms A, B, and C, and those enrolled in Arm D of the study. | Posted | Number | 95% Confidence Interval | All bleeds per year | From Baseline to at least 24 weeks |
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| Secondary | Mean Calculated Annualized Bleeding Rate for All Bleeds | The number of all bleeds over the efficacy period is presented here as a calculated annualized bleeding rate (ABR) that was annualized for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25. In this outcome measure, all bleeds are included, irrespective of treatment with coagulation factors, with the following exception: bleeds due to surgery/procedure are excluded. As "all bleeds" comprises both treated and non-treated bleeds, the 72-hour rule was implemented separately for treated and non-treated bleeds. For treated bleeds, the 72-hour rule meant that two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed. For non-treated bleeds, the 72-hour rule was implemented by calculating a treatment-free period of 72 hours from the bleed itself. | All participants, which includes those randomized to Arms A, B, and C, and those enrolled in Arm D of the study. | Posted | Mean | 95% Confidence Interval | All bleeds per year | From Baseline to at least 24 weeks |
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| Secondary | Median Calculated Annualized Bleeding Rate for All Bleeds | The number of all bleeds over the efficacy period is presented here as a calculated annualized bleeding rate (ABR) that was annualized for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25. In this outcome measure, all bleeds are included, irrespective of treatment with coagulation factors, with the following exception: bleeds due to surgery/procedure are excluded. As "all bleeds" comprises both treated and non-treated bleeds, the 72-hour rule was implemented separately for treated and non-treated bleeds. For treated bleeds, the 72-hour rule meant that two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed. For non-treated bleeds, the 72-hour rule was implemented by calculating a treatment-free period of 72 hours from the bleed itself. | All participants, which includes those randomized to Arms A, B, and C, and those enrolled in Arm D of the study. | Posted | Median | Inter-Quartile Range | All bleeds per year | From Baseline to at least 24 weeks |
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| Secondary | Model-Based Annualized Bleeding Rate for Treated Spontaneous Bleeds | The number of treated spontaneous bleeds over the efficacy period was estimated as an annualized bleeding rate (ABR) using a negative binomial regression model, which accounts for different follow-up times. A treated spontaneous bleed was defined as a treated bleed (bleed directly followed by a hemophilia medication reported to be a "treatment for bleed") with no other known contributing factor such as trauma or procedure/surgery. The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed. Bleeds due to surgery/procedure were excluded. | All participants, which includes those randomized to Arms A, B, and C, and those enrolled in Arm D of the study. | Posted | Number | 95% Confidence Interval | Treated spontaneous bleeds per year | From Baseline to at least 24 weeks |
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| Secondary | Mean Calculated Annualized Bleeding Rate for Treated Spontaneous Bleeds | The number of treated spontaneous bleeds over the efficacy period is presented here as a calculated annualized bleeding rate (ABR) that was annualized for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25. A treated spontaneous bleed was defined as a treated bleed (bleed directly followed by a hemophilia medication reported to be a "treatment for bleed") with no other known contributing factor such as trauma or procedure/surgery. The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed. Bleeds due to surgery/procedure were excluded. | All participants, which includes those randomized to Arms A, B, and C, and those enrolled in Arm D of the study. | Posted | Mean | 95% Confidence Interval | Treated spontaneous bleeds per year | From Baseline to at least 24 weeks |
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| Secondary | Median Calculated Annualized Bleeding Rate for Treated Spontaneous Bleeds | The number of treated spontaneous bleeds over the efficacy period is presented here as a calculated annualized bleeding rate (ABR) that was annualized for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25. A treated spontaneous bleed was defined as a treated bleed (bleed directly followed by a hemophilia medication reported to be a "treatment for bleed") with no other known contributing factor such as trauma or procedure/surgery. The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed. Bleeds due to surgery/procedure were excluded. | All participants, which includes those randomized to Arms A, B, and C, and those enrolled in Arm D of the study. | Posted | Median | Inter-Quartile Range | Treated spontaneous bleeds per year | From Baseline to at least 24 weeks |
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| Secondary | Model-Based Annualized Bleeding Rate for Treated Joint Bleeds | The number of treated joint bleeds over the efficacy period was estimated as an annualized bleeding rate (ABR) using a negative binomial regression model, which accounts for different follow-up times. A treated joint bleed was defined as a bleed with type reported as "joint" based on at least one of the following symptoms: increasing swelling or warmth of the skin over the joint and/or increasing pain, decreased range of motion, or difficulty using the joint compared with baseline, and the bleed was directly followed by a hemophilia medication reported to be a "treatment for bleed". The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed. Bleeds due to surgery/procedure were excluded. | All participants, which includes those randomized to Arms A, B, and C, and those enrolled in Arm D of the study. | Posted | Number | 95% Confidence Interval | Treated joint bleeds per year | From Baseline to at least 24 weeks |
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| Secondary | Mean Calculated Annualized Bleeding Rate for Treated Joint Bleeds | The number of treated joint bleeds over the efficacy period is presented here as a calculated annualized bleeding rate (ABR) that was annualized for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25. A treated joint bleed was defined as a bleed with type reported as "joint" based on at least one of the following symptoms: increasing swelling or warmth of the skin over the joint and/or increasing pain, decreased range of motion, or difficulty using the joint compared with baseline, and the bleed was directly followed by a hemophilia medication reported to be a "treatment for bleed". The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed. Bleeds due to surgery/procedure were excluded. | All participants, which includes those randomized to Arms A, B, and C, and those enrolled in Arm D of the study. | Posted | Mean | 95% Confidence Interval | Treated joint bleeds per year | From Baseline to at least 24 weeks |
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| Secondary | Median Calculated Annualized Bleeding Rate for Treated Joint Bleeds | The number of treated joint bleeds over the efficacy period is presented here as a calculated annualized bleeding rate (ABR) that was annualized for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25. A treated joint bleed was defined as a bleed with type reported as "joint" based on at least one of the following symptoms: increasing swelling or warmth of the skin over the joint and/or increasing pain, decreased range of motion, or difficulty using the joint compared with baseline, and the bleed was directly followed by a hemophilia medication reported to be a "treatment for bleed". The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed. Bleeds due to surgery/procedure were excluded. | All participants, which includes those randomized to Arms A, B, and C, and those enrolled in Arm D of the study. | Posted | Median | Inter-Quartile Range | Treated joint bleeds per year | From Baseline to at least 24 weeks |
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| Secondary | Model-Based Annualized Bleeding Rate for Treated Target Joint Bleeds | The number of treated target joint bleeds over the efficacy period was estimated as an annualized bleeding rate (ABR) using a negative binomial regression model, which accounts for different follow-up times. A treated target joint bleed was defined as a joint bleed in a target joint, which is a joint location where at least 3 bleeds have occurred over the last 24 weeks prior to study entry or an unresolved target joint (target joint that does not fulfil ≤2 bleeds into this joint within a consecutive 12-month period), and the bleed was directly followed by a hemophilia medication reported to be a "treatment for bleed". The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed. Bleeds due to surgery/procedure were excluded. | All participants, which includes those randomized to Arms A, B, and C, and those enrolled in Arm D of the study. | Posted | Number | 95% Confidence Interval | Treated target joint bleeds per year | From Baseline to at least 24 weeks |
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| Secondary | Mean Calculated Annualized Bleeding Rate for Treated Target Joint Bleeds | The number of treated target joint bleeds over the efficacy period is presented here as a calculated annualized bleeding rate (ABR) that was annualized for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25. A treated target joint bleed was defined as a joint bleed in a target joint, which is a joint location where at least 3 bleeds have occurred over the last 24 weeks prior to study entry or an unresolved target joint (target joint that does not fulfil ≤2 bleeds into this joint within a consecutive 12-month period), and the bleed was directly followed by a hemophilia medication reported to be a "treatment for bleed". The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed. Bleeds due to surgery/procedure were excluded. | All participants, which includes those randomized to Arms A, B, and C, and those enrolled in Arm D of the study. | Posted | Mean | 95% Confidence Interval | Treated target joint bleeds per year | From Baseline to at least 24 weeks |
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| Secondary | Median Calculated Annualized Bleeding Rate for Treated Target Joint Bleeds | The number of treated target joint bleeds over the efficacy period is presented here as a calculated annualized bleeding rate (ABR) that was annualized for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25. A treated target joint bleed was defined as a joint bleed in a target joint, which is a joint location where at least 3 bleeds have occurred over the last 24 weeks prior to study entry or an unresolved target joint (target joint that does not fulfil ≤2 bleeds into this joint within a consecutive 12-month period), and the bleed was directly followed by a hemophilia medication reported to be a "treatment for bleed". The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed. Bleeds due to surgery/procedure were excluded. | All participants, which includes those randomized to Arms A, B, and C, and those enrolled in Arm D of the study. | Posted | Median | Inter-Quartile Range | Treated target joint bleeds per year | From Baseline to at least 24 weeks |
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| Secondary | Intra-Participant Comparison of the Calculated Annualized Bleeding Rate for Treated Bleeds With Emicizumab Prophylaxis On-Study Versus With Previous Episodic Therapy Pre-Study | This is an intra-participant comparison of the annualized bleeding rate (ABR) for treated bleeds pre-study versus on-study in the non-interventional study (NIS) population previously treated with episodic therapy in NIS BH29768. The number of treated bleeds over the efficacy period is presented here as a calculated ABR that was annualized for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25. A treated bleed was defined as a bleed that was directly followed by a hemophilia medication reported to be a "treatment for bleed". The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed. Bleeds due to surgery/procedure were excluded. | A total of 4 participants, pooled from Arm A and Arm B (2 per Arm), who participated in the NIS BH29768 before entering this study were included for this intraparticipant analysis. | Posted | Mean | Full Range | Treated bleeds per year | Efficacy periods: At least 24 weeks prior to study entry (mean [min-max] for A+B NIS-Previous Episodic Therapy: 183 [169-221] days); and From Baseline to at least 24 weeks on study (mean [min-max] for A+B NIS-Emicizumab: 363 [324-422] days) |
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| Secondary | Intra-Participant Comparison of the Calculated Annualized Bleeding Rate for All Bleeds With Emicizumab Prophylaxis On-Study Versus With Previous Episodic Therapy Pre-Study | This is an intra-participant comparison of the annualized bleeding rate (ABR) for all bleeds pre-study versus on-study in the non-interventional study (NIS) population previously treated with episodic therapy in NIS BH29768. The ABR was calculated for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25. All bleeds are included, irrespective of treatment with coagulation factors, with the following exception: bleeds due to surgery/procedure are excluded. "All bleeds" comprises both treated and non-treated bleeds, and the 72-hour rule was implemented separately for each type. For treated bleeds, the 72-hour rule meant that two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed. For non-treated bleeds, the 72-hour rule was calculated as a treatment-free period of 72 hours from the bleed itself. | A total of 4 participants, pooled from Arm A and Arm B (2 per Arm), who participated in the NIS BH29768 before entering this study were included for this intraparticipant analysis. | Posted | Mean | Full Range | All bleeds per year | Efficacy periods: At least 24 weeks prior to study entry (mean [min-max] for A+B NIS-Previous Episodic Therapy: 183 [169-221] days); and From Baseline to at least 24 weeks on study (mean [min-max] for A+B NIS-Emicizumab: 363 [324-422] days) |
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| Secondary | Arms A, B, and C: Adjusted Mean Hemophilia A Quality of Life (Haem-A-QoL) Questionnaire Physical Health Domain Score at Week 25 in Participants ≥18 Years of Age | The Haem-A-QoL questionnaire has been developed and used in hemophilia A participants, assessing very specific aspects of dealing with hemophilia. The questionnaire consists of items pertaining to 10 domains: physical health, sports and leisure, school and work, dealing with hemophilia, family planning, feeling, relationships, treatment, view of yourself, and outlook for the future. The total score for each domain ranges from 0 to 100 with lower scores reflective of better quality of life. Physical Health domain score is reported (range 0 to 100, with lower scores reflective of better physical health). The means were derived via an analysis of covariance (ANCOVA) model and have been adjusted for the following co-variates: baseline score, treatment group, and treatment by baseline interaction term. | Adult participants randomized to Arms A, B, and C. The number analyzed represents participants who provided responses at Baseline and Week 25. | Posted | Mean | Standard Deviation | score on a scale | Baseline and Week 25 |
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| Secondary | Arms A, B, and C: Change From Baseline to Week 25 in Haem-A-QoL Questionnaire Physical Health Domain Score for Participants ≥18 Years of Age | The Haem-A-QoL questionnaire has been developed and used in hemophilia A participants, assessing very specific aspects of dealing with hemophilia. The questionnaire consists of items pertaining to 10 domains: physical health, sports and leisure, school and work, dealing with hemophilia, family planning, feeling, relationships, treatment, view of yourself, and outlook for the future. The total score for each domain ranges from 0 to 100 with lower scores reflective of better quality of life. Physical Health domain score is reported (range 0 to 100, with lower scores reflective of better physical health). | Adult participants randomized to Arms A, B, and C. The number analyzed represents participants who provided responses at Baseline and Week 25. | Posted | Mean | Standard Deviation | score on a scale | Baseline and Week 25 |
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| Secondary | Arms A, B, and C: Adjusted Mean Haem-A-QoL Questionnaire Total Score at Week 25 in Participants ≥18 Years of Age | The Haem-A-QoL questionnaire has been developed and used in hemophilia A participants, assessing very specific aspects of dealing with hemophilia. The questionnaire consists of items pertaining to 10 domains: physical health, sports and leisure, school and work, dealing with hemophilia, family planning, feeling, relationships, treatment, view of yourself, and outlook for the future. The total score for each domain ranges from 0 to 100 with lower scores reflective of better quality of life. Haem-A-QoL Total Score is the average of all domain scores and it ranges from 0 to 100, with lower scores reflective of better quality of life. The means were derived via an analysis of covariance (ANCOVA) model and have been adjusted for the following co-variates: baseline score, treatment group, and treatment by baseline interaction term. | Adult participants randomized to Arms A, B, and C. The number analyzed represents participants who provided responses at Baseline and Week 25. | Posted | Mean | Standard Deviation | score on a scale | Baseline and Week 25 |
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| Secondary | Arms A, B, and C: Change From Baseline to Week 25 in Haem-A-QoL Questionnaire Total Score for Participants ≥18 Years of Age | The Haem-A-QoL questionnaire has been developed and used in hemophilia A participants, assessing very specific aspects of dealing with hemophilia. The questionnaire consists of items pertaining to 10 domains: physical health, sports and leisure, school and work, dealing with hemophilia, family planning, feeling, relationships, treatment, view of yourself, and outlook for the future. The total score for each domain ranges from 0 to 100 with lower scores reflective of better quality of life. Haem-A-QoL Total Score is the average of all domain scores and it ranges from 0 to 100, with lower scores reflective of better quality of life. | Adult participants randomized to Arms A, B, and C. The number analyzed represents participants who provided responses at Baseline and Week 25. | Posted | Mean | Standard Deviation | score on a scale | Baseline and Week 25 |
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| Secondary | Arms A, B, and C: Hemophilia-Specific Quality of Life Short Form (Haemo-QoL-SF) Questionnaire Total Score at Baseline and Week 25 in Participants 12 to 17 Years of Age | The Haemo-QoL-SF contains 35 items covering nine dimensions considered relevant for the adolescent's (aged 12-17 years) health-related quality of life (HRQoL). Items are rated with five respective response options: never, seldom, sometimes, often, and always. The score ranges from 0 to 100, and a higher score is indicative of poorer HRQoL. According to the pre-specified statistical analysis plan, no statistical analyses were performed on the protocol-defined endpoints for the Haemo-QoL-SF due to the small number of adolescents randomized to Arms A, B and C. | Only adolescents (aged 12-17 years) randomized to Arms A, B, and C were included in this analysis. The number analyzed represents participants who provided responses at Baseline and Week 25. | Posted | Mean | Full Range | score on a scale | Baseline and Week 25 |
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| Secondary | Arms A, B, and C: Adjusted Mean European Quality of Life 5-Dimensions-5 Levels Questionnaire (EQ-5D-5L) Questionnaire Visual Analog Scale (VAS) Score at Week 25 | EQ-5D-5L is a standardized, participant-rated questionnaire to assess health-related quality of life. The EQ-5D-5L includes 2 components: the EQ-5D-5L health state profile (descriptive system) and the EQ-5D-5L VAS. The VAS is designed to rate the participant's current health state on a scale from 0 to 100, where 0 represents the worst imaginable health state and 100 represents the best imaginable health state. The means were derived via an analysis of covariance (ANCOVA) model and have been adjusted for the following co-variates: baseline score, treatment group, and treatment by baseline interaction term. | Participants randomized to Arms A, B, and C. The number analyzed represents participants who provided responses at Baseline and Week 25. | Posted | Mean | Standard Deviation | score on a scale | Baseline and Week 25 |
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| Secondary | Arms A, B, and C: Change From Baseline in EQ-5D-5L Questionnaire VAS Score at Week 25 | The European Quality of Life 5-Dimensions-5 Levels Questionnaire (EQ-5D-5L) is a standardized, participant-rated questionnaire to assess health-related quality of life. The EQ-5D-5L includes 2 components: the EQ-5D-5L health state profile (descriptive system) and the EQ-5D-5L VAS. The VAS is designed to rate the participant's current health state on a scale from 0 to 100, where 0 represents the worst imaginable health state and 100 represents the best imaginable health state. | The number analyzed indicates the number of participants who responded to the questionnaire scale at a given timepoint. For the change from baseline analysis, only participants who responded at both Baseline and Week 25 were included. | Posted | Mean | Standard Deviation | score on a scale | Baseline and Week 25 |
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| Secondary | Arms A, B, and C: Adjusted Mean EQ-5D-5L Index Utility Score at Week 25 | The European Quality of Life 5-Dimensions-5 Levels Questionnaire (EQ-5D-5L) is a standardized, participant-rated questionnaire to assess health-related quality of life. The EQ-5D-5L includes 2 components: the EQ-5D-5L health state profile (descriptive system) and the EQ-5D-5L VAS. The EQ-5D-5L health state profile is designed to record the participant's current health state in 5 domains: mobility, selfcare, usual activities, pain/discomfort, and anxiety/depression. Responses from the five domains are used to calculate a single index utility score on a scale of 0 to 1, with higher scores reflective of better quality of life. The means were derived via an analysis of covariance (ANCOVA) model and have been adjusted for the following co-variates: baseline score, treatment group, and treatment by baseline interaction term. | Participants randomized to Arms A, B, and C. The number analyzed represents participants who provided responses at Baseline and Week 25. | Posted | Mean | Standard Deviation | score on a scale | Baseline and Week 25 |
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| Secondary | Arms A, B, and C: Change From Baseline in EQ-5D-5L Index Utility Score at Week 25 | The European Quality of Life 5-Dimensions-5 Levels Questionnaire (EQ-5D-5L) is a standardized, participant-rated questionnaire to assess health-related quality of life. The EQ-5D-5L includes 2 components: the EQ-5D-5L health state profile (descriptive system) and the EQ-5D-5L VAS. The EQ-5D-5L health state profile is designed to record the participant's current health state in 5 domains: mobility, selfcare, usual activities, pain/discomfort, and anxiety/depression. Responses from the five domains are used to calculate a single index utility score on a scale of 0 to 1, with higher scores reflective of better quality of life. | Participants randomized to Arms A, B, and C. The number analyzed indicates the number of participants who responded to the questionnaire scale at a given timepoint. For the change from baseline analysis, only participants who responded at both Baseline and Week 25 were included. | Posted | Mean | Standard Deviation | score on a scale | Baseline and Week 25 |
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| Secondary | Arm D: Change From Baseline in Haemo-QoL-SF Questionnaire Physical Health and Total Scores at Week 25 in Participants 8 to 12 Years of Age | The Hemophilia-Specific Quality of Life Short Form (Haemo-QoL-SF) questionnaire contains 35 items covering nine dimensions considered relevant for the children's health-related quality of life (HRQoL). Items are rated with five respective response options: never, seldom, sometimes, often, and always. The scores range from 0 to 100, and higher scores are indicative of poorer HRQoL. | This outcome measure was only applicable for participants enrolled in Arm D. The number analyzed for each visit represents the number of participants who responded to the questionnaire. | Posted | Mean | Standard Deviation | score on a scale | Baseline and Week 25 |
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| Secondary | Arm D: Caregiver-Reported Adapted Health-Related Quality of Life for Hemophilia Patients With Inhibitors (Adapted Inhib-QoL) Including Aspects of Caregiver Burden Questionnaire Transformed Total Score Over Time | Proxy assessment of HRQoL and aspects of caregiver burden for all children, regardless of age, were collected using the Adapted Inhib-QoL with Aspects of Caregiver Burden questionnaire. The questionnaire comprises two parts. The first part asks the caregiver for his/her opinion on the child's HRQoL (proxy HRQoL). The second part asks the caregiver to rate how the child's situation is for them (i.e., the impact of the child's disease and treatment on the caregiver). Items are rated with 5 respective response options: never, seldom, sometimes, often, and all the time. Scores range from 0 to 100, with lower scores reflective of better HRQoL. A total score is calculated as the sum of all of the items in the scale. | The analysis population included caregivers for pediatric patients enrolled in Arm D only. The number analyzed represents the number of caregivers who responded to the questionnaire at a given timepoint. | Posted | Mean | Standard Deviation | score on a scale | Baseline (Week 1) and Weeks 13 and 25 |
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| Secondary | Number of Participants With at Least One Adverse Event, Severity According to the World Health Organization (WHO) Toxicity Grading Scale, Primary Analysis of Randomized Comparison Arms | The number of participants experiencing at least one adverse event (AE), including all non-serious and serious AEs, is reported here. According to the ICH guideline for Good Clinical Practice, an AE is any untoward medical occurrence in a clinical investigation subject administered a pharmaceutical product, regardless of causal attribution. The terms "severe" and "serious" are not synonymous. A serious AE is any AE that is a significant medical event meeting any of the standard criteria. Severity refers to the intensity of an AE (e.g., rated as mild, moderate, or severe, or according to the World Health Organization [WHO] toxicity grading scale); the event itself may be of relatively minor medical significance. Severity and seriousness were independently assessed by the investigator for each reported AE. | Safety Population 1 included the randomized arms: all participants in Arms A and B who received at least one dose of emicizumab and all participants in Arm C (Control) who started the no prophylaxis study period. | Posted | Count of Participants | Participants | From Baseline until primary cutoff date (at least 24 weeks): Arm C (Control) No Prophylaxis, median (range): 24.0 (23.9-28.0) weeks; Arms A & B Emicizumab, median (range): Arm A: 43.7 (28.1-60.3) weeks; Arm B: 46.1 (24.0-58.7) weeks |
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| Secondary | Number of Participants With at Least One Adverse Event, Severity According to the World Health Organization (WHO) Toxicity Grading Scale, Final Analysis | The number of participants experiencing at least one adverse event (AE), including all non-serious and serious AEs, is reported here. According to the ICH guideline for Good Clinical Practice, an AE is any untoward medical occurrence in a clinical investigation subject administered a pharmaceutical product, regardless of causal attribution. The terms "severe" and "serious" are not synonymous. A serious AE is any AE that is a significant medical event meeting any of the standard criteria. Severity refers to the intensity of an AE (e.g., rated as mild, moderate, or severe, or according to the World Health Organization [WHO] toxicity grading scale); the event itself may be of relatively minor medical significance. Severity and seriousness were independently assessed by the investigator for each reported AE. | Safety Population 2 included all participants from all arms who received at least one dose of emicizumab. | Posted | Count of Participants | Participants | From first dose of emicizumab until end of study: Arms A to C: up to 88 months; Arm D: up to 52.4 months |
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| Secondary | Number of Participants With at Least One Adverse Event Leading to Study Drug Discontinuation, Final Analysis | According to the ICH guideline for Good Clinical Practice, an adverse event (AE) is any untoward medical occurrence in a clinical investigation subject administered a pharmaceutical product, regardless of causal attribution. The terms "severe" and "serious" are not synonymous. A serious AE is any AE that is a significant medical event meeting any of the standard criteria. Severity refers to the intensity of an AE (e.g., rated as 1 = mild, 2 = moderate, 3 = severe, or 4 = potentially life-threatening according to the World Health Organization [WHO] toxicity grading scale); the event itself may be of relatively minor medical significance. Severity and seriousness were independently assessed by the investigator for each reported AE. | Safety Population 2 included all participants from all arms who received at least one dose of emicizumab. | Posted | Count of Participants | Participants | From first dose of emicizumab until end of study: Arms A to C: up to 88 months; Arm D: up to 52.4 months |
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| Secondary | Number of Participants With at Least One Systemic Hypersensitivity, Anaphylaxis, or Anaphylactoid Reaction, Severity According to the WHO Toxicity Grading Scale, Final Analysis | According to the ICH guideline for Good Clinical Practice, an adverse event (AE) is any untoward medical occurrence in a clinical investigation subject administered a pharmaceutical product, regardless of causal attribution. The terms "severe" and "serious" are not synonymous. A serious AE is any AE that is a significant medical event meeting any of the standard criteria. Severity refers to the intensity of an AE (e.g., rated as 1 = mild, 2 = moderate, 3 = severe, or 4 = potentially life-threatening according to the World Health Organization [WHO] toxicity grading scale); the event itself may be of relatively minor medical significance. Severity and seriousness were independently assessed by the investigator for each reported AE. | Safety Population 2 included all participants from all arms who received at least one dose of emicizumab. | Posted | Count of Participants | Participants | From first dose of emicizumab until end of study: Arms A to C: up to 88 months; Arm D: up to 52.4 months |
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| Secondary | Number of Participants With at Least One Thromboembolic Event, Severity According to the WHO Toxicity Grading Scale, Final Analysis | According to the ICH guideline for Good Clinical Practice, an adverse event (AE) is any untoward medical occurrence in a clinical investigation subject administered a pharmaceutical product, regardless of causal attribution. The terms "severe" and "serious" are not synonymous. A serious AE is any AE that is a significant medical event meeting any of the standard criteria. Severity refers to the intensity of an AE (e.g., rated as 1 = mild, 2 = moderate, 3 = severe, or 4 = potentially life-threatening according to the World Health Organization [WHO] toxicity grading scale); the event itself may be of relatively minor medical significance. Severity and seriousness were independently assessed by the investigator for each reported AE. | Safety Population 2 included all participants from all arms who received at least one dose of emicizumab. | Posted | Count of Participants | Participants | From first dose of emicizumab until end of study: Arms A to C: up to 88 months; Arm D: up to 52.4 months |
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| Secondary | Number of Participants With at Least One Thrombotic Microangiopathy, Severity According to the WHO Toxicity Grading Scale, Final Analysis | According to the ICH guideline for Good Clinical Practice, an adverse event (AE) is any untoward medical occurrence in a clinical investigation subject administered a pharmaceutical product, regardless of causal attribution. The terms "severe" and "serious" are not synonymous. A serious AE is any AE that is a significant medical event meeting any of the standard criteria. Severity refers to the intensity of an AE (e.g., rated as 1 = mild, 2 = moderate, 3 = severe, or 4 = potentially life-threatening according to the World Health Organization [WHO] toxicity grading scale); the event itself may be of relatively minor medical significance. Severity and seriousness were independently assessed by the investigator for each reported AE. | Safety Population 2 included all participants from all arms who received at least one dose of emicizumab. | Posted | Count of Participants | Participants | From first dose of emicizumab until end of study: Arms A to C: up to 88 months; Arm D: up to 52.4 months |
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| Secondary | Number of Participants With at Least One Injection-Site Reaction, Severity According to the WHO Toxicity Grading Scale, Final Analysis | According to the ICH guideline for Good Clinical Practice, an adverse event (AE) is any untoward medical occurrence in a clinical investigation subject administered a pharmaceutical product, regardless of causal attribution. The terms "severe" and "serious" are not synonymous. A serious AE is any AE that is a significant medical event meeting any of the standard criteria. Severity refers to the intensity of an AE (e.g., rated as 1 = mild, 2 = moderate, 3 = severe, or 4 = potentially life-threatening according to the World Health Organization [WHO] toxicity grading scale); the event itself may be of relatively minor medical significance. Severity and seriousness were independently assessed by the investigator for each reported AE. | Safety Population 2 included all participants from all arms who received at least one dose of emicizumab. | Posted | Count of Participants | Participants | From first dose of emicizumab until end of study: Arms A to C: up to 88 months; Arm D: up to 52.4 months |
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| Secondary | Number of Participants With Serum Chemistry Laboratory Abnormalities by Shift From Baseline to Highest WHO Grade Post-Baseline | The number of participants with abnormal shifts in laboratory chemistry parameters while on emicizumab throughout the study are provided below as shifts from baseline to the highest WHO grade post-baseline (1 = mild, 2 = moderate, 3 = severe, or 4 = potentially life-threatening). Not every laboratory abnormality qualifies as an adverse event. A laboratory test result must be reported as an adverse event if it meets any of the following criteria: Is accompanied by clinical symptoms; Results in a change in study treatment; Results in a medical intervention or a change in concomitant therapy; Is clinically significant in the investigator's judgment. It was the investigator's responsibility to review all laboratory findings. SGOT/AST = serum glutamic-oxaloacetic transaminase/aspartate aminotransferase; SGPT/ALT = serum glutamic-pyruvic transaminase/alanine aminotransferase | Safety Population 2 included all participants from all arms who received at least one dose of emicizumab. | Posted | Count of Participants | Participants | From first dose of emicizumab until end of study: Arms A to C: up to 88 months; Arm D: up to 52.4 months |
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| Secondary | Number of Participants With Hematology Laboratory Abnormalities by Shift From Baseline to Highest WHO Grade Post-Baseline | The number of participants with abnormal shifts in laboratory hematology parameters while on emicizumab throughout the study are provided below as shifts from baseline to the highest WHO grade post-baseline (1 = mild, 2 = moderate, 3 = severe, or 4 = potentially life-threatening). Not every laboratory abnormality qualifies as an adverse event. A laboratory test result must be reported as an adverse event if it meets any of the following criteria: Is accompanied by clinical symptoms; Results in a change in study treatment; Results in a medical intervention or a change in concomitant therapy; Is clinically significant in the investigator's judgment. It was the investigator's responsibility to review all laboratory findings. | Safety Population 2 included all participants from all arms who received at least one dose of emicizumab. | Posted | Count of Participants | Participants | From first dose of emicizumab until end of study: Arms A to C: up to 88 months; Arm D: up to 52.4 months |
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| Secondary | Change From Baseline in Body Temperature Over Time | The data for Arm C are from the emicizumab prophylaxis period and are labelled to correspond to the visit schedule of the other study arms. For Arm C, this is relative to the point at which participants switched to start receiving emicizumab (after having completed 24 weeks of no prophylaxis). | Safety Population 2 included all participants from all arms who received at least one dose of emicizumab. The number analyzed indicates those with evaluable data at a given timepoint. There is no recorded data for Arm C participants at Week 73 because they spent the first 24 weeks of the study on no prophylaxis (i.e., before receiving emicizumab). | Posted | Mean | Standard Deviation | Celsius (C) | Baseline and Weeks 5, 25, 49, and 73 |
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| Secondary | Change From Baseline in Pulse Rate Over Time | The data for Arm C are from the emicizumab prophylaxis period and are labelled to correspond to the visit schedule of the other study arms. For Arm C, this is relative to the point at which participants switched to start receiving emicizumab (after having completed 24 weeks of no prophylaxis). | Safety Population 2 included all participants from all arms who received at least one dose of emicizumab. The number analyzed indicates those with evaluable data at a given timepoint. There is no recorded data for Arm C participants at Week 73 because they spent the first 24 weeks of the study on no prophylaxis (i.e., before receiving emicizumab). | Posted | Mean | Standard Deviation | beats per minute | Baseline, Weeks 5, 25, 49, and 73 |
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| Secondary | Change From Baseline in Respiratory Rate Over Time | The data for Arm C are from the emicizumab prophylaxis period and are labelled to correspond to the visit schedule of the other study arms. For Arm C, this is relative to the point at which participants switched to start receiving emicizumab (after having completed 24 weeks of no prophylaxis). | Safety Population 2 included all participants from all arms who received at least one dose of emicizumab. The number analyzed indicates those with evaluable data at a given timepoint. There is no recorded data for Arm C participants at Week 73 because they spent the first 24 weeks of the study on no prophylaxis (i.e., before receiving emicizumab). | Posted | Mean | Standard Deviation | breaths per minute | Baseline, Weeks 5, 25, 49, and 73 |
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| Secondary | Change From Baseline in Systolic Blood Pressure Over Time | The data for Arm C are from the emicizumab prophylaxis period and are labelled to correspond to the visit schedule of the other study arms. For Arm C, this is relative to the point at which participants switched to start receiving emicizumab (after having completed 24 weeks of no prophylaxis). | Safety Population 2 included all participants from all arms who received at least one dose of emicizumab. The number analyzed indicates those with evaluable data at a given timepoint. There is no recorded data for Arm C participants at Week 73 because they spent the first 24 weeks of the study on no prophylaxis (i.e., before receiving emicizumab). | Posted | Mean | Standard Deviation | millimetres of mercury (mmHg) | Baseline, Weeks 5, 25, 49, and 73 |
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| Secondary | Change From Baseline in Diastolic Blood Pressure Over Time | The data for Arm C are from the emicizumab prophylaxis period and are labelled to correspond to the visit schedule of the other study arms. For Arm C, this is relative to the point at which participants switched to start receiving emicizumab (after having completed 24 weeks of no prophylaxis). | Safety Population 2 included all participants from all arms who received at least one dose of emicizumab. The number analyzed indicates those with evaluable data at a given timepoint. There is no recorded data for Arm C participants at Week 73 because they spent the first 24 weeks of the study on no prophylaxis (i.e., before receiving emicizumab). | Posted | Mean | Standard Deviation | millimetres of mercury (mmHg) | Baseline, Weeks 5, 25, 49, and 73 |
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| Secondary | Number of Participants by Post-Baseline Anti-Emicizumab Antibody (ADA) Status | Participants were considered anti-drug antibody (ADA)-positive if they were ADA-negative at baseline but developed an ADA response following study drug administration, or if they were ADA-positive at baseline and the titer of one or more post-baseline samples was at least 4-fold greater than the titer of the baseline sample. Participants were considered ADA-negative if they were ADA-negative at baseline and all post-baseline samples were negative following drug administration, or if they were ADA-positive at baseline but did not have any post-baseline (following drug administration) samples with a titer that was at least 4-fold greater than the titer of the baseline sample. | Safety Population 2 included all participants from all arms who received at least one dose of emicizumab. | Posted | Count of Participants | Participants | Samples taken at Baseline and at prespecified times post-baseline from first dose of emicizumab until data cutoff date, median (range) time of exposure to emicizumab: All Arms: 196.14 (20.1-222.1) weeks; Arm D only: 64.14 (61.1-67.3) weeks |
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Plasma Trough Concentration (Ctrough) of Emicizumab | The data for Arm C are from the emicizumab prophylaxis period and are labelled to correspond to the visit schedule of the other study arms. For Arm C, this is relative to the point at which participants switched to start receiving emicizumab (after having completed 24 weeks of no prophylaxis). | The Pharmacokinetics Population included all participants from all arms who received at least one dose of emicizumab. One participant from Arm C was excluded from analysis due to a dosing protocol deviation. The number analyzed indicates those with evaluable data at a given timepoint. | Posted | Geometric Mean | Geometric Coefficient of Variation | microgram per millilitre (μg/mL) | Arms A & D, QW (up to Week 49 for Arm D only): Weeks 2, 3, 4, 5, 7, 9, 13, 17, 21, 25, 33, 41, 49, 61, 73, 85, 97, 109, 121, and 133; Arms B & C, Q4W: Weeks 2, 3, 4, 5, 9, 13, 17, 21, 25, 37, 49, 61, 73, 85, 97, 109, 121, and 133 |
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Primary | Model-Based Annualized Bleeding Rate for Treated Bleeds | The number of treated bleeds over the efficacy period was estimated as an annualized bleeding rate (ABR) using a negative binomial regression model, which accounts for different follow-up times. A treated bleed was defined as a bleed that was directly followed by a hemophilia medication reported to be a "treatment for bleed". The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed. Bleeds due to surgery/procedure were excluded. | All participants, which includes those randomized to Arms A, B, and C, and those enrolled in Arm D of the study. | Posted | Number | 95% Confidence Interval | Treated bleeds per year | From Baseline to at least 24 weeks |
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Primary | Mean Calculated Annualized Bleeding Rate for Treated Bleeds | The number of treated bleeds over the efficacy period is presented here as a calculated annualized bleeding rate (ABR) that was annualized for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25. A treated bleed was defined as a bleed that was directly followed by a hemophilia medication reported to be a "treatment for bleed". The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed. Bleeds due to surgery/procedure were excluded. | All participants, which includes those randomized to Arms A, B, and C, and those enrolled in Arm D of the study. | Posted | Mean | 95% Confidence Interval | Treated bleeds per year | From Baseline to at least 24 weeks |
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Primary | Median Calculated Annualized Bleeding Rate for Treated Bleeds | The number of treated bleeds over the efficacy period is presented here as a calculated annualized bleeding rate (ABR) that was annualized for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25. A treated bleed was defined as a bleed that was directly followed by a hemophilia medication reported to be a "treatment for bleed". The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed. Bleeds due to surgery/procedure were excluded. | All participants, which includes those randomized to Arms A, B, and C, and those enrolled in Arm D of the study. | Posted | Median | Inter-Quartile Range | Treated bleeds per year | From Baseline to at least 24 weeks |
|
From the date of randomization or enrollment until the end of study (Arm C [Control] No Prophylaxis: up to 28 weeks; Arms A to C Emicizumab: up to 88 months; Arm D Emicizumab: up to 52.4 months)
After randomization (randomized arms A, B, and C) or initiation of study drug (non-randomized arm D), all adverse events were reported until the patient completed their last study visit. After this period, the investigator reported any serious adverse events that were believed to be related to prior study drug treatment.
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Arm C (Control): No Prophylaxis | Participants ≥12 years old with hemophilia A (with or without FVIII inhibitors) who were randomized to Arm C did not receive any prophylactic treatment for the first 24 weeks of the study (Control). Participants continued to receive standard-of-care treatments on an episodic basis for the treatment of bleeds during the study. | 0 | 14 | 0 | 14 | 2 | 14 |
| EG001 | Arm A: 1.5 mg/kg Emicizumab Prophylaxis QW | Participants ≥12 years old with hemophilia A (with or without FVIII inhibitors) who were randomized to Arm A received prophylactic emicizumab at a dose of 3 mg/kg via SC injection QW for the first 4 weeks, followed by 1.5 mg/kg via SC injection QW for at least 24 weeks. After 24 weeks of treatment, participants were allowed to continue receiving emicizumab until marketing authorization, as part of this study or a separate extension study, as long as they derived clinical benefit. Participants continued to receive standard-of-care treatments on an episodic basis for the treatment of breakthrough bleeds during the study. | 1 | 29 | 9 | 29 | 28 | 29 |
| EG002 | Arm B: 6 mg/kg Emicizumab Prophylaxis Q4W | Participants ≥12 years old with hemophilia A (with or without FVIII inhibitors) who were randomized to Arm B received prophylactic emicizumab at a dose of 3 mg/kg via SC injection QW for the first 4 weeks, followed by 6 mg/kg via SC injection Q4W for at least 24 weeks. After 24 weeks of treatment, participants were allowed to continue receiving emicizumab until marketing authorization, as part of this study or a separate extension study, as long as they derived clinical benefit. Participants continued to receive standard-of-care treatments on an episodic basis for the treatment of breakthrough bleeds during the study. | 0 | 27 | 6 | 27 | 24 | 27 |
| EG003 | Arm C (Emi): 6 mg/kg Emicizumab Prophylaxis Q4W | After 24 weeks on no prophylaxis, participants were given the opportunity to switch to receive emicizumab prophylaxis at 3 mg/kg QW via SC injection for 4 weeks, followed by 6 mg/kg Q4W until marketing authorization as part of this study or a separate extension study, as long as they derive clinical benefit. Participants continued to receive standard-of-care treatments on an episodic basis for the treatment of breakthrough bleeds during the study. | 0 | 14 | 4 | 14 | 14 | 14 |
| EG004 | Arm D: 1.5 mg/kg Emicizumab Prophylaxis QW | Participants <12 years old with hemophilia A and FVIII inhibitors who were enrolled to Arm D received prophylactic emicizumab at a dose of 3 mg/kg via SC injection QW for the first 4 weeks, followed by 1.5 mg/kg via SC injection QW for at least 24 weeks. After 24 weeks of treatment, participants were allowed to continue receiving emicizumab until marketing authorization, as part of this study or a separate extension study, as long as they derived clinical benefit. Participants continued to receive standard-of-care treatments on an episodic basis for the treatment of breakthrough bleeds during the study. | 0 | 15 | 2 | 15 | 15 | 15 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Gastrointestinal haemorrhage | Gastrointestinal disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Ileal ulcer | Gastrointestinal disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Intra-abdominal haemorrhage | Gastrointestinal disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Mallory-Weiss syndrome | Gastrointestinal disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Pancreatitis | Gastrointestinal disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Mass | General disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Haemarthrosis | Musculoskeletal and connective tissue disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Haemophilic arthropathy | Musculoskeletal and connective tissue disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Haemorrhage | Vascular disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Shock haemorrhagic | Vascular disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Anaemia | Blood and lymphatic system disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Cholelithiasis | Hepatobiliary disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Infective exacerbation of bronchiectasis | Infections and infestations | MedDRA version 28.0 | Systematic Assessment |
| |
| Limb fracture | Injury, poisoning and procedural complications | MedDRA version 28.0 | Systematic Assessment |
| |
| Adenocarcinoma of colon | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA version 28.0 | Systematic Assessment |
| |
| Ureteric dilatation | Renal and urinary disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Ureterolithiasis | Renal and urinary disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Bronchiectasis | Respiratory, thoracic and mediastinal disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Anal fistula | Gastrointestinal disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Haemorrhoidal haemorrhage | Gastrointestinal disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Large intestine polyp | Gastrointestinal disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Lower gastrointestinal haemorrhage | Gastrointestinal disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Mouth haemorrhage | Gastrointestinal disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Rectal ulcer | Gastrointestinal disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Iron deficiency anaemia | Blood and lymphatic system disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Cardio-respiratory arrest | Cardiac disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Cataract | Eye disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Necrosis | General disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA version 28.0 | Systematic Assessment |
| |
| Bone contusion | Injury, poisoning and procedural complications | MedDRA version 28.0 | Systematic Assessment |
| |
| Fracture | Injury, poisoning and procedural complications | MedDRA version 28.0 | Systematic Assessment |
| |
| Cerebrovascular accident | Nervous system disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Nephrolithiasis | Renal and urinary disorders | MedDRA version 28.0 | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Upper respiratory tract infection | Infections and infestations | MedDRA version 28.0 | Systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA version 28.0 | Systematic Assessment |
| |
| Pharyngitis | Infections and infestations | MedDRA version 28.0 | Systematic Assessment |
| |
| Bronchitis | Infections and infestations | MedDRA version 28.0 | Systematic Assessment |
| |
| Gingivitis | Infections and infestations | MedDRA version 28.0 | Systematic Assessment |
| |
| Tonsillitis | Infections and infestations | MedDRA version 28.0 | Systematic Assessment |
| |
| Herpes zoster | Infections and infestations | MedDRA version 28.0 | Systematic Assessment |
| |
| Otitis media | Infections and infestations | MedDRA version 28.0 | Systematic Assessment |
| |
| Rhinitis | Infections and infestations | MedDRA version 28.0 | Systematic Assessment |
| |
| Laryngitis | Infections and infestations | MedDRA version 28.0 | Systematic Assessment |
| |
| Viral infection | Infections and infestations | MedDRA version 28.0 | Systematic Assessment |
| |
| Aspartate aminotransferase increased | Investigations | MedDRA version 28.0 | Systematic Assessment |
| |
| Alanine aminotransferase increased | Investigations | MedDRA version 28.0 | Systematic Assessment |
| |
| Blood uric acid increased | Investigations | MedDRA version 28.0 | Systematic Assessment |
| |
| Blood glucose increased | Investigations | MedDRA version 28.0 | Systematic Assessment |
| |
| Blood bilirubin increased | Investigations | MedDRA version 28.0 | Systematic Assessment |
| |
| Alanine aminotransferase abnormal | Investigations | MedDRA version 28.0 | Systematic Assessment |
| |
| Bilirubin conjugated increased | Investigations | MedDRA version 28.0 | Systematic Assessment |
| |
| Blood potassium decreased | Investigations | MedDRA version 28.0 | Systematic Assessment |
| |
| Weight decreased | Investigations | MedDRA version 28.0 | Systematic Assessment |
| |
| Aspartate aminotransferase abnormal | Investigations | MedDRA version 28.0 | Systematic Assessment |
| |
| Blood ketone body increased | Investigations | MedDRA version 28.0 | Systematic Assessment |
| |
| Differential white blood cell count abnormal | Investigations | MedDRA version 28.0 | Systematic Assessment |
| |
| Neutrophil count abnormal | Investigations | MedDRA version 28.0 | Systematic Assessment |
| |
| White blood cell count abnormal | Investigations | MedDRA version 28.0 | Systematic Assessment |
| |
| White blood cells urine positive | Investigations | MedDRA version 28.0 | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Dental caries | Gastrointestinal disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Abdominal distension | Gastrointestinal disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Toothache | Gastrointestinal disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Dyspepsia | Gastrointestinal disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Mouth ulceration | Gastrointestinal disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Injection site reaction | General disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Influenza like illness | General disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Chest discomfort | General disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Asthenia | General disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Hyperuricaemia | Metabolism and nutrition disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Groin pain | Musculoskeletal and connective tissue disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Muscular weakness | Musculoskeletal and connective tissue disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Contusion | Injury, poisoning and procedural complications | MedDRA version 28.0 | Systematic Assessment |
| |
| Ligament sprain | Injury, poisoning and procedural complications | MedDRA version 28.0 | Systematic Assessment |
| |
| Limb injury | Injury, poisoning and procedural complications | MedDRA version 28.0 | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Dizziness | Nervous system disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Head discomfort | Nervous system disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Hypertension | Vascular disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Cold sweat | Skin and subcutaneous tissue disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Anaemia | Blood and lymphatic system disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Iron deficiency anaemia | Blood and lymphatic system disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Hepatic function abnormal | Hepatobiliary disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Gallbladder polyp | Hepatobiliary disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Hepatic steatosis | Hepatobiliary disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Palipitations | Cardiac disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Sinus bradycardia | Cardiac disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Tinnitus | Ear and labyrinth disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Breast hyperplasia | Reproductive system and breast disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Leukocytosis | Blood and lymphatic system disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Splenomegaly | Blood and lymphatic system disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Hyperthyroidism | Endocrine disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Conjunctival haemorrhage | Eye disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Aphthous ulcer | Gastrointestinal disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Chronic gastritis | Gastrointestinal disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Colitis | Gastrointestinal disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Gastritis | Gastrointestinal disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Gastrooesophageal reflux disease | Gastrointestinal disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Haemorrhoids | Gastrointestinal disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Tooth impacted | Gastrointestinal disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Appendicitis | Infections and infestations | MedDRA version 28.0 | Systematic Assessment |
| |
| COVID-19 | Infections and infestations | MedDRA version 28.0 | Systematic Assessment |
| |
| Suspected COVID-19 | Infections and infestations | MedDRA version 28.0 | Systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA version 28.0 | Systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA version 28.0 | Systematic Assessment |
| |
| Varicella | Infections and infestations | MedDRA version 28.0 | Systematic Assessment |
| |
| Gastroenteritis | Infections and infestations | MedDRA version 28.0 | Systematic Assessment |
| |
| Animal bite | Injury, poisoning and procedural complications | MedDRA version 28.0 | Systematic Assessment |
| |
| Arthropod bite | Injury, poisoning and procedural complications | MedDRA version 28.0 | Systematic Assessment |
| |
| Closed globe injury | Injury, poisoning and procedural complications | MedDRA version 28.0 | Systematic Assessment |
| |
| Meniscus injury | Injury, poisoning and procedural complications | MedDRA version 28.0 | Systematic Assessment |
| |
| Muscle strain | Injury, poisoning and procedural complications | MedDRA version 28.0 | Systematic Assessment |
| |
| Skin abrasion | Injury, poisoning and procedural complications | MedDRA version 28.0 | Systematic Assessment |
| |
| Soft tissue injury | Injury, poisoning and procedural complications | MedDRA version 28.0 | Systematic Assessment |
| |
| Weight increased | Investigations | MedDRA version 28.0 | Systematic Assessment |
| |
| Gamma-glutamyltransferase increased | Investigations | MedDRA version 28.0 | Systematic Assessment |
| |
| Neutrophil count increased | Investigations | MedDRA version 28.0 | Systematic Assessment |
| |
| Electrolyte imbalance | Metabolism and nutrition disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Hyperlipidaemia | Metabolism and nutrition disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Hypoglycaemia | Metabolism and nutrition disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Iron deficiency | Metabolism and nutrition disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Haemarthrosis | Musculoskeletal and connective tissue disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Haemophilic arthropathy | Musculoskeletal and connective tissue disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Syncope | Nervous system disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Insomnia | Psychiatric disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Haematuria | Renal and urinary disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Nephrolithiasis | Renal and urinary disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Ureterolithiasis | Renal and urinary disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Bronchiectasis | Respiratory, thoracic and mediastinal disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Pulmonary mass | Respiratory, thoracic and mediastinal disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Dermatitis | Skin and subcutaneous tissue disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Rash | Skin and subcutaneous tissue disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Haematoma | Vascular disorders | MedDRA version 28.0 | Systematic Assessment |
|
The Study being conducted under this Agreement is part of the Overall Study. Investigator is free to publish in reputable journals or to present at professional conferences the results of the Study, but only after the first publication or presentation that involves the Overall Study. The Sponsor may request that Confidential Information be deleted and/or the publication be postponed in order to protect the Sponsor's intellectual property rights.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Medical Communications | Hoffmann-La Roche | 800-821-8590 | genentech@druginfo.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Jul 22, 2019 | Jul 17, 2023 | SAP_001.pdf |
| ID | Term |
|---|---|
| D006467 | Hemophilia A |
| ID | Term |
|---|---|
| D025861 | Blood Coagulation Disorders, Inherited |
| D001778 | Blood Coagulation Disorders |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D020147 | Coagulation Protein Disorders |
| D006474 | Hemorrhagic Disorders |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
Not provided
Not provided
| ID | Term |
|---|---|
| C000608208 | emicizumab |
Not provided
Not provided
Not provided
| ≥18 to <65 Years Old |
|
| ≥65 Years Old |
|
| Male |
|
| FVIII Inhibitor Negative (Non-Inhibitor) |
|
| ≥9 Bleeds |
|
|
H0 (null hypothesis): ABR Ratio = 1; versus, H1 (alternative hypothesis): ABR Ratio ≠ 1 |
| Stratified Wald test |
| <0.0001 |
Statistical significance was controlled at a 2-sided alpha level of 0.05. Hierarchical testing was used to account for multiple comparisons. |
| ABR Ratio |
| 0.05 |
| 2-Sided |
| 95 |
| 0.028 |
| 0.092 |
The ABR ratio was calculated as Arm B vs. Arm C. |
| Superiority |
| Arm A: 1.5 mg/kg Emicizumab Prophylaxis QW |
Participants ≥12 years old with hemophilia A (with or without FVIII inhibitors) who were randomized to Arm A received prophylactic emicizumab at a dose of 3 mg/kg via SC injection QW for the first 4 weeks, followed by 1.5 mg/kg via SC injection QW for at least 24 weeks. After 24 weeks of treatment, participants were allowed to continue receiving emicizumab until marketing authorization, as part of this study or a separate extension study, as long as they derived clinical benefit. Participants continued to receive standard-of-care treatments on an episodic basis for the treatment of breakthrough bleeds during the study. |
| OG002 | Arm B: 6 mg/kg Emicizumab Prophylaxis Q4W | Participants ≥12 years old with hemophilia A (with or without FVIII inhibitors) who were randomized to Arm B received prophylactic emicizumab at a dose of 3 mg/kg via SC injection QW for the first 4 weeks, followed by 6 mg/kg via SC injection Q4W for at least 24 weeks. After 24 weeks of treatment, participants were allowed to continue receiving emicizumab until marketing authorization, as part of this study or a separate extension study, as long as they derived clinical benefit. Participants continued to receive standard-of-care treatments on an episodic basis for the treatment of breakthrough bleeds during the study. |
| OG003 | Arm D: 1.5 mg/kg Emicizumab Prophylaxis QW | Participants <12 years old with hemophilia A and FVIII inhibitors who were enrolled to Arm D received prophylactic emicizumab at a dose of 3 mg/kg via SC injection QW for the first 4 weeks, followed by 1.5 mg/kg via SC injection QW for at least 24 weeks. After 24 weeks of treatment, participants were allowed to continue receiving emicizumab until marketing authorization, as part of this study or a separate extension study, as long as they derived clinical benefit. Participants continued to receive standard-of-care treatments on an episodic basis for the treatment of breakthrough bleeds during the study. |
|
|
| Arm A: 1.5 mg/kg Emicizumab Prophylaxis QW |
Participants ≥12 years old with hemophilia A (with or without FVIII inhibitors) who were randomized to Arm A received prophylactic emicizumab at a dose of 3 mg/kg via SC injection QW for the first 4 weeks, followed by 1.5 mg/kg via SC injection QW for at least 24 weeks. After 24 weeks of treatment, participants were allowed to continue receiving emicizumab until marketing authorization, as part of this study or a separate extension study, as long as they derived clinical benefit. Participants continued to receive standard-of-care treatments on an episodic basis for the treatment of breakthrough bleeds during the study. |
| OG002 | Arm B: 6 mg/kg Emicizumab Prophylaxis Q4W | Participants ≥12 years old with hemophilia A (with or without FVIII inhibitors) who were randomized to Arm B received prophylactic emicizumab at a dose of 3 mg/kg via SC injection QW for the first 4 weeks, followed by 6 mg/kg via SC injection Q4W for at least 24 weeks. After 24 weeks of treatment, participants were allowed to continue receiving emicizumab until marketing authorization, as part of this study or a separate extension study, as long as they derived clinical benefit. Participants continued to receive standard-of-care treatments on an episodic basis for the treatment of breakthrough bleeds during the study. |
| OG003 | Arm D: 1.5 mg/kg Emicizumab Prophylaxis QW | Participants <12 years old with hemophilia A and FVIII inhibitors who were enrolled to Arm D received prophylactic emicizumab at a dose of 3 mg/kg via SC injection QW for the first 4 weeks, followed by 1.5 mg/kg via SC injection QW for at least 24 weeks. After 24 weeks of treatment, participants were allowed to continue receiving emicizumab until marketing authorization, as part of this study or a separate extension study, as long as they derived clinical benefit. Participants continued to receive standard-of-care treatments on an episodic basis for the treatment of breakthrough bleeds during the study. |
|
|
| OG002 | Arm B: 6 mg/kg Emicizumab Prophylaxis Q4W | Participants ≥12 years old with hemophilia A (with or without FVIII inhibitors) who were randomized to Arm B received prophylactic emicizumab at a dose of 3 mg/kg via SC injection QW for the first 4 weeks, followed by 6 mg/kg via SC injection Q4W for at least 24 weeks. After 24 weeks of treatment, participants were allowed to continue receiving emicizumab until marketing authorization, as part of this study or a separate extension study, as long as they derived clinical benefit. Participants continued to receive standard-of-care treatments on an episodic basis for the treatment of breakthrough bleeds during the study. |
| OG003 | Arm D: 1.5 mg/kg Emicizumab Prophylaxis QW | Participants <12 years old with hemophilia A and FVIII inhibitors who were enrolled to Arm D received prophylactic emicizumab at a dose of 3 mg/kg via SC injection QW for the first 4 weeks, followed by 1.5 mg/kg via SC injection QW for at least 24 weeks. After 24 weeks of treatment, participants were allowed to continue receiving emicizumab until marketing authorization, as part of this study or a separate extension study, as long as they derived clinical benefit. Participants continued to receive standard-of-care treatments on an episodic basis for the treatment of breakthrough bleeds during the study. |
|
|
|
Participants ≥12 years old with hemophilia A (with or without FVIII inhibitors) who were randomized to Arm A received prophylactic emicizumab at a dose of 3 mg/kg via SC injection QW for the first 4 weeks, followed by 1.5 mg/kg via SC injection QW for at least 24 weeks. After 24 weeks of treatment, participants were allowed to continue receiving emicizumab until marketing authorization, as part of this study or a separate extension study, as long as they derived clinical benefit. Participants continued to receive standard-of-care treatments on an episodic basis for the treatment of breakthrough bleeds during the study.
| OG002 | Arm B: 6 mg/kg Emicizumab Prophylaxis Q4W | Participants ≥12 years old with hemophilia A (with or without FVIII inhibitors) who were randomized to Arm B received prophylactic emicizumab at a dose of 3 mg/kg via SC injection QW for the first 4 weeks, followed by 6 mg/kg via SC injection Q4W for at least 24 weeks. After 24 weeks of treatment, participants were allowed to continue receiving emicizumab until marketing authorization, as part of this study or a separate extension study, as long as they derived clinical benefit. Participants continued to receive standard-of-care treatments on an episodic basis for the treatment of breakthrough bleeds during the study. |
| OG003 | Arm D: 1.5 mg/kg Emicizumab Prophylaxis QW | Participants <12 years old with hemophilia A and FVIII inhibitors who were enrolled to Arm D received prophylactic emicizumab at a dose of 3 mg/kg via SC injection QW for the first 4 weeks, followed by 1.5 mg/kg via SC injection QW for at least 24 weeks. After 24 weeks of treatment, participants were allowed to continue receiving emicizumab until marketing authorization, as part of this study or a separate extension study, as long as they derived clinical benefit. Participants continued to receive standard-of-care treatments on an episodic basis for the treatment of breakthrough bleeds during the study. |
|
|
Participants ≥12 years old with hemophilia A (with or without FVIII inhibitors) who were randomized to Arm A received prophylactic emicizumab at a dose of 3 mg/kg via SC injection QW for the first 4 weeks, followed by 1.5 mg/kg via SC injection QW for at least 24 weeks. After 24 weeks of treatment, participants were allowed to continue receiving emicizumab until marketing authorization, as part of this study or a separate extension study, as long as they derived clinical benefit. Participants continued to receive standard-of-care treatments on an episodic basis for the treatment of breakthrough bleeds during the study.
| OG002 | Arm B: 6 mg/kg Emicizumab Prophylaxis Q4W | Participants ≥12 years old with hemophilia A (with or without FVIII inhibitors) who were randomized to Arm B received prophylactic emicizumab at a dose of 3 mg/kg via SC injection QW for the first 4 weeks, followed by 6 mg/kg via SC injection Q4W for at least 24 weeks. After 24 weeks of treatment, participants were allowed to continue receiving emicizumab until marketing authorization, as part of this study or a separate extension study, as long as they derived clinical benefit. Participants continued to receive standard-of-care treatments on an episodic basis for the treatment of breakthrough bleeds during the study. |
| OG003 | Arm D: 1.5 mg/kg Emicizumab Prophylaxis QW | Participants <12 years old with hemophilia A and FVIII inhibitors who were enrolled to Arm D received prophylactic emicizumab at a dose of 3 mg/kg via SC injection QW for the first 4 weeks, followed by 1.5 mg/kg via SC injection QW for at least 24 weeks. After 24 weeks of treatment, participants were allowed to continue receiving emicizumab until marketing authorization, as part of this study or a separate extension study, as long as they derived clinical benefit. Participants continued to receive standard-of-care treatments on an episodic basis for the treatment of breakthrough bleeds during the study. |
|
|
Participants ≥12 years old with hemophilia A (with or without FVIII inhibitors) who were randomized to Arm A received prophylactic emicizumab at a dose of 3 mg/kg via SC injection QW for the first 4 weeks, followed by 1.5 mg/kg via SC injection QW for at least 24 weeks. After 24 weeks of treatment, participants were allowed to continue receiving emicizumab until marketing authorization, as part of this study or a separate extension study, as long as they derived clinical benefit. Participants continued to receive standard-of-care treatments on an episodic basis for the treatment of breakthrough bleeds during the study. |
| OG002 | Arm B: 6 mg/kg Emicizumab Prophylaxis Q4W | Participants ≥12 years old with hemophilia A (with or without FVIII inhibitors) who were randomized to Arm B received prophylactic emicizumab at a dose of 3 mg/kg via SC injection QW for the first 4 weeks, followed by 6 mg/kg via SC injection Q4W for at least 24 weeks. After 24 weeks of treatment, participants were allowed to continue receiving emicizumab until marketing authorization, as part of this study or a separate extension study, as long as they derived clinical benefit. Participants continued to receive standard-of-care treatments on an episodic basis for the treatment of breakthrough bleeds during the study. |
| OG003 | Arm D: 1.5 mg/kg Emicizumab Prophylaxis QW | Participants <12 years old with hemophilia A and FVIII inhibitors who were enrolled to Arm D received prophylactic emicizumab at a dose of 3 mg/kg via SC injection QW for the first 4 weeks, followed by 1.5 mg/kg via SC injection QW for at least 24 weeks. After 24 weeks of treatment, participants were allowed to continue receiving emicizumab until marketing authorization, as part of this study or a separate extension study, as long as they derived clinical benefit. Participants continued to receive standard-of-care treatments on an episodic basis for the treatment of breakthrough bleeds during the study. |
|
|
|
| Arm A: 1.5 mg/kg Emicizumab Prophylaxis QW |
Participants ≥12 years old with hemophilia A (with or without FVIII inhibitors) who were randomized to Arm A received prophylactic emicizumab at a dose of 3 mg/kg via SC injection QW for the first 4 weeks, followed by 1.5 mg/kg via SC injection QW for at least 24 weeks. After 24 weeks of treatment, participants were allowed to continue receiving emicizumab until marketing authorization, as part of this study or a separate extension study, as long as they derived clinical benefit. Participants continued to receive standard-of-care treatments on an episodic basis for the treatment of breakthrough bleeds during the study. |
| OG002 | Arm B: 6 mg/kg Emicizumab Prophylaxis Q4W | Participants ≥12 years old with hemophilia A (with or without FVIII inhibitors) who were randomized to Arm B received prophylactic emicizumab at a dose of 3 mg/kg via SC injection QW for the first 4 weeks, followed by 6 mg/kg via SC injection Q4W for at least 24 weeks. After 24 weeks of treatment, participants were allowed to continue receiving emicizumab until marketing authorization, as part of this study or a separate extension study, as long as they derived clinical benefit. Participants continued to receive standard-of-care treatments on an episodic basis for the treatment of breakthrough bleeds during the study. |
| OG003 | Arm D: 1.5 mg/kg Emicizumab Prophylaxis QW | Participants <12 years old with hemophilia A and FVIII inhibitors who were enrolled to Arm D received prophylactic emicizumab at a dose of 3 mg/kg via SC injection QW for the first 4 weeks, followed by 1.5 mg/kg via SC injection QW for at least 24 weeks. After 24 weeks of treatment, participants were allowed to continue receiving emicizumab until marketing authorization, as part of this study or a separate extension study, as long as they derived clinical benefit. Participants continued to receive standard-of-care treatments on an episodic basis for the treatment of breakthrough bleeds during the study. |
|
|
| Arm A: 1.5 mg/kg Emicizumab Prophylaxis QW |
Participants ≥12 years old with hemophilia A (with or without FVIII inhibitors) who were randomized to Arm A received prophylactic emicizumab at a dose of 3 mg/kg via SC injection QW for the first 4 weeks, followed by 1.5 mg/kg via SC injection QW for at least 24 weeks. After 24 weeks of treatment, participants were allowed to continue receiving emicizumab until marketing authorization, as part of this study or a separate extension study, as long as they derived clinical benefit. Participants continued to receive standard-of-care treatments on an episodic basis for the treatment of breakthrough bleeds during the study. |
| OG002 | Arm B: 6 mg/kg Emicizumab Prophylaxis Q4W | Participants ≥12 years old with hemophilia A (with or without FVIII inhibitors) who were randomized to Arm B received prophylactic emicizumab at a dose of 3 mg/kg via SC injection QW for the first 4 weeks, followed by 6 mg/kg via SC injection Q4W for at least 24 weeks. After 24 weeks of treatment, participants were allowed to continue receiving emicizumab until marketing authorization, as part of this study or a separate extension study, as long as they derived clinical benefit. Participants continued to receive standard-of-care treatments on an episodic basis for the treatment of breakthrough bleeds during the study. |
| OG003 | Arm D: 1.5 mg/kg Emicizumab Prophylaxis QW | Participants <12 years old with hemophilia A and FVIII inhibitors who were enrolled to Arm D received prophylactic emicizumab at a dose of 3 mg/kg via SC injection QW for the first 4 weeks, followed by 1.5 mg/kg via SC injection QW for at least 24 weeks. After 24 weeks of treatment, participants were allowed to continue receiving emicizumab until marketing authorization, as part of this study or a separate extension study, as long as they derived clinical benefit. Participants continued to receive standard-of-care treatments on an episodic basis for the treatment of breakthrough bleeds during the study. |
|
|
| Arm A: 1.5 mg/kg Emicizumab Prophylaxis QW |
Participants ≥12 years old with hemophilia A (with or without FVIII inhibitors) who were randomized to Arm A received prophylactic emicizumab at a dose of 3 mg/kg via SC injection QW for the first 4 weeks, followed by 1.5 mg/kg via SC injection QW for at least 24 weeks. After 24 weeks of treatment, participants were allowed to continue receiving emicizumab until marketing authorization, as part of this study or a separate extension study, as long as they derived clinical benefit. Participants continued to receive standard-of-care treatments on an episodic basis for the treatment of breakthrough bleeds during the study. |
| OG002 | Arm B: 6 mg/kg Emicizumab Prophylaxis Q4W | Participants ≥12 years old with hemophilia A (with or without FVIII inhibitors) who were randomized to Arm B received prophylactic emicizumab at a dose of 3 mg/kg via SC injection QW for the first 4 weeks, followed by 6 mg/kg via SC injection Q4W for at least 24 weeks. After 24 weeks of treatment, participants were allowed to continue receiving emicizumab until marketing authorization, as part of this study or a separate extension study, as long as they derived clinical benefit. Participants continued to receive standard-of-care treatments on an episodic basis for the treatment of breakthrough bleeds during the study. |
| OG003 | Arm D: 1.5 mg/kg Emicizumab Prophylaxis QW | Participants <12 years old with hemophilia A and FVIII inhibitors who were enrolled to Arm D received prophylactic emicizumab at a dose of 3 mg/kg via SC injection QW for the first 4 weeks, followed by 1.5 mg/kg via SC injection QW for at least 24 weeks. After 24 weeks of treatment, participants were allowed to continue receiving emicizumab until marketing authorization, as part of this study or a separate extension study, as long as they derived clinical benefit. Participants continued to receive standard-of-care treatments on an episodic basis for the treatment of breakthrough bleeds during the study. |
|
|
|
| OG001 |
| Arm A: 1.5 mg/kg Emicizumab Prophylaxis QW |
Participants ≥12 years old with hemophilia A (with or without FVIII inhibitors) who were randomized to Arm A received prophylactic emicizumab at a dose of 3 mg/kg via SC injection QW for the first 4 weeks, followed by 1.5 mg/kg via SC injection QW for at least 24 weeks. After 24 weeks of treatment, participants were allowed to continue receiving emicizumab until marketing authorization, as part of this study or a separate extension study, as long as they derived clinical benefit. Participants continued to receive standard-of-care treatments on an episodic basis for the treatment of breakthrough bleeds during the study. |
| OG002 | Arm B: 6 mg/kg Emicizumab Prophylaxis Q4W | Participants ≥12 years old with hemophilia A (with or without FVIII inhibitors) who were randomized to Arm B received prophylactic emicizumab at a dose of 3 mg/kg via SC injection QW for the first 4 weeks, followed by 6 mg/kg via SC injection Q4W for at least 24 weeks. After 24 weeks of treatment, participants were allowed to continue receiving emicizumab until marketing authorization, as part of this study or a separate extension study, as long as they derived clinical benefit. Participants continued to receive standard-of-care treatments on an episodic basis for the treatment of breakthrough bleeds during the study. |
| OG003 | Arm D: 1.5 mg/kg Emicizumab Prophylaxis QW | Participants <12 years old with hemophilia A and FVIII inhibitors who were enrolled to Arm D received prophylactic emicizumab at a dose of 3 mg/kg via SC injection QW for the first 4 weeks, followed by 1.5 mg/kg via SC injection QW for at least 24 weeks. After 24 weeks of treatment, participants were allowed to continue receiving emicizumab until marketing authorization, as part of this study or a separate extension study, as long as they derived clinical benefit. Participants continued to receive standard-of-care treatments on an episodic basis for the treatment of breakthrough bleeds during the study. |
|
|
| OG001 |
| Arm A: 1.5 mg/kg Emicizumab Prophylaxis QW |
Participants ≥12 years old with hemophilia A (with or without FVIII inhibitors) who were randomized to Arm A received prophylactic emicizumab at a dose of 3 mg/kg via SC injection QW for the first 4 weeks, followed by 1.5 mg/kg via SC injection QW for at least 24 weeks. After 24 weeks of treatment, participants were allowed to continue receiving emicizumab until marketing authorization, as part of this study or a separate extension study, as long as they derived clinical benefit. Participants continued to receive standard-of-care treatments on an episodic basis for the treatment of breakthrough bleeds during the study. |
| OG002 | Arm B: 6 mg/kg Emicizumab Prophylaxis Q4W | Participants ≥12 years old with hemophilia A (with or without FVIII inhibitors) who were randomized to Arm B received prophylactic emicizumab at a dose of 3 mg/kg via SC injection QW for the first 4 weeks, followed by 6 mg/kg via SC injection Q4W for at least 24 weeks. After 24 weeks of treatment, participants were allowed to continue receiving emicizumab until marketing authorization, as part of this study or a separate extension study, as long as they derived clinical benefit. Participants continued to receive standard-of-care treatments on an episodic basis for the treatment of breakthrough bleeds during the study. |
| OG003 | Arm D: 1.5 mg/kg Emicizumab Prophylaxis QW | Participants <12 years old with hemophilia A and FVIII inhibitors who were enrolled to Arm D received prophylactic emicizumab at a dose of 3 mg/kg via SC injection QW for the first 4 weeks, followed by 1.5 mg/kg via SC injection QW for at least 24 weeks. After 24 weeks of treatment, participants were allowed to continue receiving emicizumab until marketing authorization, as part of this study or a separate extension study, as long as they derived clinical benefit. Participants continued to receive standard-of-care treatments on an episodic basis for the treatment of breakthrough bleeds during the study. |
|
|
| OG001 | A+B NIS: Emicizumab Prophylaxis On-Study | Only participants with hemophilia A (with or without FVIII inhibitors), pooled from Arm A and Arm B, who previously participated in the NIS BH29768 are included here for analysis. On this study, the participants received prophylactic emicizumab at a dose of 3 mg/kg via SC injection QW for the first 4 weeks, followed by either 1.5 mg/kg (in Arm A) or 6 mg/kg (in Arm B) via SC injection Q4W for at least 24 weeks. After 24 weeks of treatment, participants were allowed to continue receiving emicizumab until marketing authorization, as part of this study or a separate extension study, as long as they derived clinical benefit. Participants continued to receive standard-of-care treatments on an episodic basis for the treatment of breakthrough bleeds during the study. |
|
|
Only participants with hemophilia A (with or without FVIII inhibitors), pooled from Arm A and Arm B, who previously participated in the NIS BH29768 are included here for analysis. This previous episodic therapy analysis group includes historical bleed data during participation in NIS BH29768 prior to enrollment in this study from 2 patients enrolled in Arm A who had FVIII inhibitors and had received episodic therapy with bypassing agents and 2 patients enrolled in Arm B who did not have FVIII inhibitors and had received episodic therapy with FVIII.
| OG001 | A+B NIS: Emicizumab Prophylaxis On-Study | Only participants with hemophilia A (with or without FVIII inhibitors), pooled from Arm A and Arm B, who previously participated in the NIS BH29768 are included here for analysis. On this study, the participants received prophylactic emicizumab at a dose of 3 mg/kg via SC injection QW for the first 4 weeks, followed by either 1.5 mg/kg (in Arm A) or 6 mg/kg (in Arm B) via SC injection Q4W for at least 24 weeks. After 24 weeks of treatment, participants were allowed to continue receiving emicizumab until marketing authorization, as part of this study or a separate extension study, as long as they derived clinical benefit. Participants continued to receive standard-of-care treatments on an episodic basis for the treatment of breakthrough bleeds during the study. |
|
|
| Arm A: 1.5 mg/kg Emicizumab Prophylaxis QW |
Participants ≥12 years old with hemophilia A (with or without FVIII inhibitors) who were randomized to Arm A received prophylactic emicizumab at a dose of 3 mg/kg via SC injection QW for the first 4 weeks, followed by 1.5 mg/kg via SC injection QW for at least 24 weeks. After 24 weeks of treatment, participants were allowed to continue receiving emicizumab until marketing authorization, as part of this study or a separate extension study, as long as they derived clinical benefit. Participants continued to receive standard-of-care treatments on an episodic basis for the treatment of breakthrough bleeds during the study. |
| OG002 | Arm B: 6 mg/kg Emicizumab Prophylaxis Q4W | Participants ≥12 years old with hemophilia A (with or without FVIII inhibitors) who were randomized to Arm B received prophylactic emicizumab at a dose of 3 mg/kg via SC injection QW for the first 4 weeks, followed by 6 mg/kg via SC injection Q4W for at least 24 weeks. After 24 weeks of treatment, participants were allowed to continue receiving emicizumab until marketing authorization, as part of this study or a separate extension study, as long as they derived clinical benefit. Participants continued to receive standard-of-care treatments on an episodic basis for the treatment of breakthrough bleeds during the study. |
|
|
|
| OG002 | Arm B: 6 mg/kg Emicizumab Prophylaxis Q4W | Participants ≥12 years old with hemophilia A (with or without FVIII inhibitors) who were randomized to Arm B received prophylactic emicizumab at a dose of 3 mg/kg via SC injection QW for the first 4 weeks, followed by 6 mg/kg via SC injection Q4W for at least 24 weeks. After 24 weeks of treatment, participants were allowed to continue receiving emicizumab until marketing authorization, as part of this study or a separate extension study, as long as they derived clinical benefit. Participants continued to receive standard-of-care treatments on an episodic basis for the treatment of breakthrough bleeds during the study. |
|
|
Participants ≥12 years old with hemophilia A (with or without FVIII inhibitors) who were randomized to Arm A received prophylactic emicizumab at a dose of 3 mg/kg via SC injection QW for the first 4 weeks, followed by 1.5 mg/kg via SC injection QW for at least 24 weeks. After 24 weeks of treatment, participants were allowed to continue receiving emicizumab until marketing authorization, as part of this study or a separate extension study, as long as they derived clinical benefit. Participants continued to receive standard-of-care treatments on an episodic basis for the treatment of breakthrough bleeds during the study. |
| OG002 | Arm B: 6 mg/kg Emicizumab Prophylaxis Q4W | Participants ≥12 years old with hemophilia A (with or without FVIII inhibitors) who were randomized to Arm B received prophylactic emicizumab at a dose of 3 mg/kg via SC injection QW for the first 4 weeks, followed by 6 mg/kg via SC injection Q4W for at least 24 weeks. After 24 weeks of treatment, participants were allowed to continue receiving emicizumab until marketing authorization, as part of this study or a separate extension study, as long as they derived clinical benefit. Participants continued to receive standard-of-care treatments on an episodic basis for the treatment of breakthrough bleeds during the study. |
|
|
|
| OG002 | Arm B: 6 mg/kg Emicizumab Prophylaxis Q4W | Participants ≥12 years old with hemophilia A (with or without FVIII inhibitors) who were randomized to Arm B received prophylactic emicizumab at a dose of 3 mg/kg via SC injection QW for the first 4 weeks, followed by 6 mg/kg via SC injection Q4W for at least 24 weeks. After 24 weeks of treatment, participants were allowed to continue receiving emicizumab until marketing authorization, as part of this study or a separate extension study, as long as they derived clinical benefit. Participants continued to receive standard-of-care treatments on an episodic basis for the treatment of breakthrough bleeds during the study. |
|
|
| OG002 | Arm B: 6 mg/kg Emicizumab Prophylaxis Q4W | Participants ≥12 years old with hemophilia A (with or without FVIII inhibitors) who were randomized to Arm B received prophylactic emicizumab at a dose of 3 mg/kg via SC injection QW for the first 4 weeks, followed by 6 mg/kg via SC injection Q4W for at least 24 weeks. After 24 weeks of treatment, participants were allowed to continue receiving emicizumab until marketing authorization, as part of this study or a separate extension study, as long as they derived clinical benefit. Participants continued to receive standard-of-care treatments on an episodic basis for the treatment of breakthrough bleeds during the study. |
|
|
| OG002 | Arm B: 6 mg/kg Emicizumab Prophylaxis Q4W | Participants ≥12 years old with hemophilia A (with or without FVIII inhibitors) who were randomized to Arm B received prophylactic emicizumab at a dose of 3 mg/kg via SC injection QW for the first 4 weeks, followed by 6 mg/kg via SC injection Q4W for at least 24 weeks. After 24 weeks of treatment, participants were allowed to continue receiving emicizumab until marketing authorization, as part of this study or a separate extension study, as long as they derived clinical benefit. Participants continued to receive standard-of-care treatments on an episodic basis for the treatment of breakthrough bleeds during the study. |
|
|
|
| OG002 | Arm B: 6 mg/kg Emicizumab Prophylaxis Q4W | Participants ≥12 years old with hemophilia A (with or without FVIII inhibitors) who were randomized to Arm B received prophylactic emicizumab at a dose of 3 mg/kg via SC injection QW for the first 4 weeks, followed by 6 mg/kg via SC injection Q4W for at least 24 weeks. After 24 weeks of treatment, participants were allowed to continue receiving emicizumab until marketing authorization, as part of this study or a separate extension study, as long as they derived clinical benefit. Participants continued to receive standard-of-care treatments on an episodic basis for the treatment of breakthrough bleeds during the study. |
|
|
Participants ≥12 years old with hemophilia A (with or without FVIII inhibitors) who were randomized to Arm A received prophylactic emicizumab at a dose of 3 mg/kg via SC injection QW for the first 4 weeks, followed by 1.5 mg/kg via SC injection QW for at least 24 weeks. After 24 weeks of treatment, participants were allowed to continue receiving emicizumab until marketing authorization, as part of this study or a separate extension study, as long as they derived clinical benefit. Participants continued to receive standard-of-care treatments on an episodic basis for the treatment of breakthrough bleeds during the study. |
| OG002 | Arm B: 6 mg/kg Emicizumab Prophylaxis Q4W | Participants ≥12 years old with hemophilia A (with or without FVIII inhibitors) who were randomized to Arm B received prophylactic emicizumab at a dose of 3 mg/kg via SC injection QW for the first 4 weeks, followed by 6 mg/kg via SC injection Q4W for at least 24 weeks. After 24 weeks of treatment, participants were allowed to continue receiving emicizumab until marketing authorization, as part of this study or a separate extension study, as long as they derived clinical benefit. Participants continued to receive standard-of-care treatments on an episodic basis for the treatment of breakthrough bleeds during the study. |
|
|
|
Participants ≥12 years old with hemophilia A (with or without FVIII inhibitors) who were randomized to Arm A received prophylactic emicizumab at a dose of 3 mg/kg via SC injection QW for the first 4 weeks, followed by 1.5 mg/kg via SC injection QW for at least 24 weeks. After 24 weeks of treatment, participants were allowed to continue receiving emicizumab until marketing authorization, as part of this study or a separate extension study, as long as they derived clinical benefit. Participants continued to receive standard-of-care treatments on an episodic basis for the treatment of breakthrough bleeds during the study.
| OG002 | Arm B: 6 mg/kg Emicizumab Prophylaxis Q4W | Participants ≥12 years old with hemophilia A (with or without FVIII inhibitors) who were randomized to Arm B received prophylactic emicizumab at a dose of 3 mg/kg via SC injection QW for the first 4 weeks, followed by 6 mg/kg via SC injection Q4W for at least 24 weeks. After 24 weeks of treatment, participants were allowed to continue receiving emicizumab until marketing authorization, as part of this study or a separate extension study, as long as they derived clinical benefit. Participants continued to receive standard-of-care treatments on an episodic basis for the treatment of breakthrough bleeds during the study. |
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| Units |
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| Counts |
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| Participants |
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| OG001 | Arm A: 1.5 mg/kg Emicizumab Prophylaxis QW | Participants ≥12 years old with hemophilia A (with or without FVIII inhibitors) who were randomized to Arm A received prophylactic emicizumab at a dose of 3 mg/kg via SC injection QW for the first 4 weeks, followed by 1.5 mg/kg via SC injection QW for at least 24 weeks. After 24 weeks of treatment, participants were allowed to continue receiving emicizumab until marketing authorization, as part of this study or a separate extension study, as long as they derived clinical benefit. Participants continued to receive standard-of-care treatments on an episodic basis for the treatment of breakthrough bleeds during the study. |
| OG002 | Arm B: 6 mg/kg Emicizumab Prophylaxis Q4W | Participants ≥12 years old with hemophilia A (with or without FVIII inhibitors) who were randomized to Arm B received prophylactic emicizumab at a dose of 3 mg/kg via SC injection QW for the first 4 weeks, followed by 6 mg/kg via SC injection Q4W for at least 24 weeks. After 24 weeks of treatment, participants were allowed to continue receiving emicizumab until marketing authorization, as part of this study or a separate extension study, as long as they derived clinical benefit. Participants continued to receive standard-of-care treatments on an episodic basis for the treatment of breakthrough bleeds during the study. |
|
|
| OG001 | Arm B: 6 mg/kg Emicizumab Prophylaxis Q4W | Participants ≥12 years old with hemophilia A (with or without FVIII inhibitors) who were randomized to Arm B received prophylactic emicizumab at a dose of 3 mg/kg via SC injection QW for the first 4 weeks, followed by 6 mg/kg via SC injection Q4W for at least 24 weeks. After 24 weeks of treatment, participants were allowed to continue receiving emicizumab until marketing authorization, as part of this study or a separate extension study, as long as they derived clinical benefit. Participants continued to receive standard-of-care treatments on an episodic basis for the treatment of breakthrough bleeds during the study. |
| OG002 | Arm C (Emi): 6 mg/kg Emicizumab Prophylaxis Q4W | After 24 weeks on no prophylaxis, participants were given the opportunity to switch to receive emicizumab prophylaxis at 3 mg/kg QW via SC injection for 4 weeks, followed by 6 mg/kg Q4W until marketing authorization as part of this study or a separate extension study, as long as they derive clinical benefit. Participants continued to receive standard-of-care treatments on an episodic basis for the treatment of breakthrough bleeds during the study. |
| OG003 | Arm D: 1.5 mg/kg Emicizumab Prophylaxis QW | Participants <12 years old with hemophilia A and FVIII inhibitors who were enrolled to Arm D received prophylactic emicizumab at a dose of 3 mg/kg via SC injection QW for the first 4 weeks, followed by 1.5 mg/kg via SC injection QW for at least 24 weeks. After 24 weeks of treatment, participants were allowed to continue receiving emicizumab until marketing authorization, as part of this study or a separate extension study, as long as they derived clinical benefit. Participants continued to receive standard-of-care treatments on an episodic basis for the treatment of breakthrough bleeds during the study. |
|
|
| OG001 | Arm B: 6 mg/kg Emicizumab Prophylaxis Q4W | Participants ≥12 years old with hemophilia A (with or without FVIII inhibitors) who were randomized to Arm B received prophylactic emicizumab at a dose of 3 mg/kg via SC injection QW for the first 4 weeks, followed by 6 mg/kg via SC injection Q4W for at least 24 weeks. After 24 weeks of treatment, participants were allowed to continue receiving emicizumab until marketing authorization, as part of this study or a separate extension study, as long as they derived clinical benefit. Participants continued to receive standard-of-care treatments on an episodic basis for the treatment of breakthrough bleeds during the study. |
| OG002 | Arm C (Emi): 6 mg/kg Emicizumab Prophylaxis Q4W | After 24 weeks on no prophylaxis, participants were given the opportunity to switch to receive emicizumab prophylaxis at 3 mg/kg QW via SC injection for 4 weeks, followed by 6 mg/kg Q4W until marketing authorization as part of this study or a separate extension study, as long as they derive clinical benefit. Participants continued to receive standard-of-care treatments on an episodic basis for the treatment of breakthrough bleeds during the study. |
| OG003 | Arm D: 1.5 mg/kg Emicizumab Prophylaxis QW | Participants <12 years old with hemophilia A and FVIII inhibitors who were enrolled to Arm D received prophylactic emicizumab at a dose of 3 mg/kg via SC injection QW for the first 4 weeks, followed by 1.5 mg/kg via SC injection QW for at least 24 weeks. After 24 weeks of treatment, participants were allowed to continue receiving emicizumab until marketing authorization, as part of this study or a separate extension study, as long as they derived clinical benefit. Participants continued to receive standard-of-care treatments on an episodic basis for the treatment of breakthrough bleeds during the study. |
|
|
| OG001 | Arm B: 6 mg/kg Emicizumab Prophylaxis Q4W | Participants ≥12 years old with hemophilia A (with or without FVIII inhibitors) who were randomized to Arm B received prophylactic emicizumab at a dose of 3 mg/kg via SC injection QW for the first 4 weeks, followed by 6 mg/kg via SC injection Q4W for at least 24 weeks. After 24 weeks of treatment, participants were allowed to continue receiving emicizumab until marketing authorization, as part of this study or a separate extension study, as long as they derived clinical benefit. Participants continued to receive standard-of-care treatments on an episodic basis for the treatment of breakthrough bleeds during the study. |
| OG002 | Arm C (Emi): 6 mg/kg Emicizumab Prophylaxis Q4W | After 24 weeks on no prophylaxis, participants were given the opportunity to switch to receive emicizumab prophylaxis at 3 mg/kg QW via SC injection for 4 weeks, followed by 6 mg/kg Q4W until marketing authorization as part of this study or a separate extension study, as long as they derive clinical benefit. Participants continued to receive standard-of-care treatments on an episodic basis for the treatment of breakthrough bleeds during the study. |
| OG003 | Arm D: 1.5 mg/kg Emicizumab Prophylaxis QW | Participants <12 years old with hemophilia A and FVIII inhibitors who were enrolled to Arm D received prophylactic emicizumab at a dose of 3 mg/kg via SC injection QW for the first 4 weeks, followed by 1.5 mg/kg via SC injection QW for at least 24 weeks. After 24 weeks of treatment, participants were allowed to continue receiving emicizumab until marketing authorization, as part of this study or a separate extension study, as long as they derived clinical benefit. Participants continued to receive standard-of-care treatments on an episodic basis for the treatment of breakthrough bleeds during the study. |
|
|
| OG001 | Arm B: 6 mg/kg Emicizumab Prophylaxis Q4W | Participants ≥12 years old with hemophilia A (with or without FVIII inhibitors) who were randomized to Arm B received prophylactic emicizumab at a dose of 3 mg/kg via SC injection QW for the first 4 weeks, followed by 6 mg/kg via SC injection Q4W for at least 24 weeks. After 24 weeks of treatment, participants were allowed to continue receiving emicizumab until marketing authorization, as part of this study or a separate extension study, as long as they derived clinical benefit. Participants continued to receive standard-of-care treatments on an episodic basis for the treatment of breakthrough bleeds during the study. |
| OG002 | Arm C (Emi): 6 mg/kg Emicizumab Prophylaxis Q4W | After 24 weeks on no prophylaxis, participants were given the opportunity to switch to receive emicizumab prophylaxis at 3 mg/kg QW via SC injection for 4 weeks, followed by 6 mg/kg Q4W until marketing authorization as part of this study or a separate extension study, as long as they derive clinical benefit. Participants continued to receive standard-of-care treatments on an episodic basis for the treatment of breakthrough bleeds during the study. |
| OG003 | Arm D: 1.5 mg/kg Emicizumab Prophylaxis QW | Participants <12 years old with hemophilia A and FVIII inhibitors who were enrolled to Arm D received prophylactic emicizumab at a dose of 3 mg/kg via SC injection QW for the first 4 weeks, followed by 1.5 mg/kg via SC injection QW for at least 24 weeks. After 24 weeks of treatment, participants were allowed to continue receiving emicizumab until marketing authorization, as part of this study or a separate extension study, as long as they derived clinical benefit. Participants continued to receive standard-of-care treatments on an episodic basis for the treatment of breakthrough bleeds during the study. |
|
|
| OG001 | Arm B: 6 mg/kg Emicizumab Prophylaxis Q4W | Participants ≥12 years old with hemophilia A (with or without FVIII inhibitors) who were randomized to Arm B received prophylactic emicizumab at a dose of 3 mg/kg via SC injection QW for the first 4 weeks, followed by 6 mg/kg via SC injection Q4W for at least 24 weeks. After 24 weeks of treatment, participants were allowed to continue receiving emicizumab until marketing authorization, as part of this study or a separate extension study, as long as they derived clinical benefit. Participants continued to receive standard-of-care treatments on an episodic basis for the treatment of breakthrough bleeds during the study. |
| OG002 | Arm C (Emi): 6 mg/kg Emicizumab Prophylaxis Q4W | After 24 weeks on no prophylaxis, participants were given the opportunity to switch to receive emicizumab prophylaxis at 3 mg/kg QW via SC injection for 4 weeks, followed by 6 mg/kg Q4W until marketing authorization as part of this study or a separate extension study, as long as they derive clinical benefit. Participants continued to receive standard-of-care treatments on an episodic basis for the treatment of breakthrough bleeds during the study. |
| OG003 | Arm D: 1.5 mg/kg Emicizumab Prophylaxis QW | Participants <12 years old with hemophilia A and FVIII inhibitors who were enrolled to Arm D received prophylactic emicizumab at a dose of 3 mg/kg via SC injection QW for the first 4 weeks, followed by 1.5 mg/kg via SC injection QW for at least 24 weeks. After 24 weeks of treatment, participants were allowed to continue receiving emicizumab until marketing authorization, as part of this study or a separate extension study, as long as they derived clinical benefit. Participants continued to receive standard-of-care treatments on an episodic basis for the treatment of breakthrough bleeds during the study. |
|
|
| OG001 | Arm B: 6 mg/kg Emicizumab Prophylaxis Q4W | Participants ≥12 years old with hemophilia A (with or without FVIII inhibitors) who were randomized to Arm B received prophylactic emicizumab at a dose of 3 mg/kg via SC injection QW for the first 4 weeks, followed by 6 mg/kg via SC injection Q4W for at least 24 weeks. After 24 weeks of treatment, participants were allowed to continue receiving emicizumab until marketing authorization, as part of this study or a separate extension study, as long as they derived clinical benefit. Participants continued to receive standard-of-care treatments on an episodic basis for the treatment of breakthrough bleeds during the study. |
| OG002 | Arm C (Emi): 6 mg/kg Emicizumab Prophylaxis Q4W | After 24 weeks on no prophylaxis, participants were given the opportunity to switch to receive emicizumab prophylaxis at 3 mg/kg QW via SC injection for 4 weeks, followed by 6 mg/kg Q4W until marketing authorization as part of this study or a separate extension study, as long as they derive clinical benefit. Participants continued to receive standard-of-care treatments on an episodic basis for the treatment of breakthrough bleeds during the study. |
| OG003 | Arm D: 1.5 mg/kg Emicizumab Prophylaxis QW | Participants <12 years old with hemophilia A and FVIII inhibitors who were enrolled to Arm D received prophylactic emicizumab at a dose of 3 mg/kg via SC injection QW for the first 4 weeks, followed by 1.5 mg/kg via SC injection QW for at least 24 weeks. After 24 weeks of treatment, participants were allowed to continue receiving emicizumab until marketing authorization, as part of this study or a separate extension study, as long as they derived clinical benefit. Participants continued to receive standard-of-care treatments on an episodic basis for the treatment of breakthrough bleeds during the study. |
|
|
| OG001 | Arm B: 6 mg/kg Emicizumab Prophylaxis Q4W | Participants ≥12 years old with hemophilia A (with or without FVIII inhibitors) who were randomized to Arm B received prophylactic emicizumab at a dose of 3 mg/kg via SC injection QW for the first 4 weeks, followed by 6 mg/kg via SC injection Q4W for at least 24 weeks. After 24 weeks of treatment, participants were allowed to continue receiving emicizumab until marketing authorization, as part of this study or a separate extension study, as long as they derived clinical benefit. Participants continued to receive standard-of-care treatments on an episodic basis for the treatment of breakthrough bleeds during the study. |
| OG002 | Arm C (Emi): 6 mg/kg Emicizumab Prophylaxis Q4W | After 24 weeks on no prophylaxis, participants were given the opportunity to switch to receive emicizumab prophylaxis at 3 mg/kg QW via SC injection for 4 weeks, followed by 6 mg/kg Q4W until marketing authorization as part of this study or a separate extension study, as long as they derive clinical benefit. Participants continued to receive standard-of-care treatments on an episodic basis for the treatment of breakthrough bleeds during the study. |
| OG003 | Arm D: 1.5 mg/kg Emicizumab Prophylaxis QW | Participants <12 years old with hemophilia A and FVIII inhibitors who were enrolled to Arm D received prophylactic emicizumab at a dose of 3 mg/kg via SC injection QW for the first 4 weeks, followed by 1.5 mg/kg via SC injection QW for at least 24 weeks. After 24 weeks of treatment, participants were allowed to continue receiving emicizumab until marketing authorization, as part of this study or a separate extension study, as long as they derived clinical benefit. Participants continued to receive standard-of-care treatments on an episodic basis for the treatment of breakthrough bleeds during the study. |
|
|
| OG001 | Arm B: 6 mg/kg Emicizumab Prophylaxis Q4W | Participants ≥12 years old with hemophilia A (with or without FVIII inhibitors) who were randomized to Arm B received prophylactic emicizumab at a dose of 3 mg/kg via SC injection QW for the first 4 weeks, followed by 6 mg/kg via SC injection Q4W for at least 24 weeks. After 24 weeks of treatment, participants were allowed to continue receiving emicizumab until marketing authorization, as part of this study or a separate extension study, as long as they derived clinical benefit. Participants continued to receive standard-of-care treatments on an episodic basis for the treatment of breakthrough bleeds during the study. |
| OG002 | Arm C (Emi): 6 mg/kg Emicizumab Prophylaxis Q4W | After 24 weeks on no prophylaxis, participants were given the opportunity to switch to receive emicizumab prophylaxis at 3 mg/kg QW via SC injection for 4 weeks, followed by 6 mg/kg Q4W until marketing authorization as part of this study or a separate extension study, as long as they derive clinical benefit. Participants continued to receive standard-of-care treatments on an episodic basis for the treatment of breakthrough bleeds during the study. |
| OG003 | Arm D: 1.5 mg/kg Emicizumab Prophylaxis QW | Participants <12 years old with hemophilia A and FVIII inhibitors who were enrolled to Arm D received prophylactic emicizumab at a dose of 3 mg/kg via SC injection QW for the first 4 weeks, followed by 1.5 mg/kg via SC injection QW for at least 24 weeks. After 24 weeks of treatment, participants were allowed to continue receiving emicizumab until marketing authorization, as part of this study or a separate extension study, as long as they derived clinical benefit. Participants continued to receive standard-of-care treatments on an episodic basis for the treatment of breakthrough bleeds during the study. |
|
|
Participants ≥12 years old with hemophilia A (with or without FVIII inhibitors) who were randomized to Arm B received prophylactic emicizumab at a dose of 3 mg/kg via SC injection QW for the first 4 weeks, followed by 6 mg/kg via SC injection Q4W for at least 24 weeks. After 24 weeks of treatment, participants were allowed to continue receiving emicizumab until marketing authorization, as part of this study or a separate extension study, as long as they derived clinical benefit. Participants continued to receive standard-of-care treatments on an episodic basis for the treatment of breakthrough bleeds during the study. |
| OG002 | Arm C (Emi): 6 mg/kg Emicizumab Prophylaxis Q4W | After 24 weeks on no prophylaxis, participants were given the opportunity to switch to receive emicizumab prophylaxis at 3 mg/kg QW via SC injection for 4 weeks, followed by 6 mg/kg Q4W until marketing authorization as part of this study or a separate extension study, as long as they derive clinical benefit. Participants continued to receive standard-of-care treatments on an episodic basis for the treatment of breakthrough bleeds during the study. |
| OG003 | Arm D: 1.5 mg/kg Emicizumab Prophylaxis QW | Participants <12 years old with hemophilia A and FVIII inhibitors who were enrolled to Arm D received prophylactic emicizumab at a dose of 3 mg/kg via SC injection QW for the first 4 weeks, followed by 1.5 mg/kg via SC injection QW for at least 24 weeks. After 24 weeks of treatment, participants were allowed to continue receiving emicizumab until marketing authorization, as part of this study or a separate extension study, as long as they derived clinical benefit. Participants continued to receive standard-of-care treatments on an episodic basis for the treatment of breakthrough bleeds during the study. |
|
|
Participants ≥12 years old with hemophilia A (with or without FVIII inhibitors) who were randomized to Arm B received prophylactic emicizumab at a dose of 3 mg/kg via SC injection QW for the first 4 weeks, followed by 6 mg/kg via SC injection Q4W for at least 24 weeks. After 24 weeks of treatment, participants were allowed to continue receiving emicizumab until marketing authorization, as part of this study or a separate extension study, as long as they derived clinical benefit. Participants continued to receive standard-of-care treatments on an episodic basis for the treatment of breakthrough bleeds during the study. |
| OG002 | Arm C (Emi): 6 mg/kg Emicizumab Prophylaxis Q4W | After 24 weeks on no prophylaxis, participants were given the opportunity to switch to receive emicizumab prophylaxis at 3 mg/kg QW via SC injection for 4 weeks, followed by 6 mg/kg Q4W until marketing authorization as part of this study or a separate extension study, as long as they derive clinical benefit. Participants continued to receive standard-of-care treatments on an episodic basis for the treatment of breakthrough bleeds during the study. |
| OG003 | Arm D: 1.5 mg/kg Emicizumab Prophylaxis QW | Participants <12 years old with hemophilia A and FVIII inhibitors who were enrolled to Arm D received prophylactic emicizumab at a dose of 3 mg/kg via SC injection QW for the first 4 weeks, followed by 1.5 mg/kg via SC injection QW for at least 24 weeks. After 24 weeks of treatment, participants were allowed to continue receiving emicizumab until marketing authorization, as part of this study or a separate extension study, as long as they derived clinical benefit. Participants continued to receive standard-of-care treatments on an episodic basis for the treatment of breakthrough bleeds during the study. |
|
|
Participants ≥12 years old with hemophilia A (with or without FVIII inhibitors) who were randomized to Arm B received prophylactic emicizumab at a dose of 3 mg/kg via SC injection QW for the first 4 weeks, followed by 6 mg/kg via SC injection Q4W for at least 24 weeks. After 24 weeks of treatment, participants were allowed to continue receiving emicizumab until marketing authorization, as part of this study or a separate extension study, as long as they derived clinical benefit. Participants continued to receive standard-of-care treatments on an episodic basis for the treatment of breakthrough bleeds during the study. |
| OG002 | Arm C (Emi): 6 mg/kg Emicizumab Prophylaxis Q4W | After 24 weeks on no prophylaxis, participants were given the opportunity to switch to receive emicizumab prophylaxis at 3 mg/kg QW via SC injection for 4 weeks, followed by 6 mg/kg Q4W until marketing authorization as part of this study or a separate extension study, as long as they derive clinical benefit. Participants continued to receive standard-of-care treatments on an episodic basis for the treatment of breakthrough bleeds during the study. |
| OG003 | Arm D: 1.5 mg/kg Emicizumab Prophylaxis QW | Participants <12 years old with hemophilia A and FVIII inhibitors who were enrolled to Arm D received prophylactic emicizumab at a dose of 3 mg/kg via SC injection QW for the first 4 weeks, followed by 1.5 mg/kg via SC injection QW for at least 24 weeks. After 24 weeks of treatment, participants were allowed to continue receiving emicizumab until marketing authorization, as part of this study or a separate extension study, as long as they derived clinical benefit. Participants continued to receive standard-of-care treatments on an episodic basis for the treatment of breakthrough bleeds during the study. |
|
|
Participants ≥12 years old with hemophilia A (with or without FVIII inhibitors) who were randomized to Arm B received prophylactic emicizumab at a dose of 3 mg/kg via SC injection QW for the first 4 weeks, followed by 6 mg/kg via SC injection Q4W for at least 24 weeks. After 24 weeks of treatment, participants were allowed to continue receiving emicizumab until marketing authorization, as part of this study or a separate extension study, as long as they derived clinical benefit. Participants continued to receive standard-of-care treatments on an episodic basis for the treatment of breakthrough bleeds during the study. |
| OG002 | Arm C (Emi): 6 mg/kg Emicizumab Prophylaxis Q4W | After 24 weeks on no prophylaxis, participants were given the opportunity to switch to receive emicizumab prophylaxis at 3 mg/kg QW via SC injection for 4 weeks, followed by 6 mg/kg Q4W until marketing authorization as part of this study or a separate extension study, as long as they derive clinical benefit. Participants continued to receive standard-of-care treatments on an episodic basis for the treatment of breakthrough bleeds during the study. |
| OG003 | Arm D: 1.5 mg/kg Emicizumab Prophylaxis QW | Participants <12 years old with hemophilia A and FVIII inhibitors who were enrolled to Arm D received prophylactic emicizumab at a dose of 3 mg/kg via SC injection QW for the first 4 weeks, followed by 1.5 mg/kg via SC injection QW for at least 24 weeks. After 24 weeks of treatment, participants were allowed to continue receiving emicizumab until marketing authorization, as part of this study or a separate extension study, as long as they derived clinical benefit. Participants continued to receive standard-of-care treatments on an episodic basis for the treatment of breakthrough bleeds during the study. |
|
|
Participants ≥12 years old with hemophilia A (with or without FVIII inhibitors) who were randomized to Arm B received prophylactic emicizumab at a dose of 3 mg/kg via SC injection QW for the first 4 weeks, followed by 6 mg/kg via SC injection Q4W for at least 24 weeks. After 24 weeks of treatment, participants were allowed to continue receiving emicizumab until marketing authorization, as part of this study or a separate extension study, as long as they derived clinical benefit. Participants continued to receive standard-of-care treatments on an episodic basis for the treatment of breakthrough bleeds during the study. |
| OG002 | Arm C (Emi): 6 mg/kg Emicizumab Prophylaxis Q4W | After 24 weeks on no prophylaxis, participants were given the opportunity to switch to receive emicizumab prophylaxis at 3 mg/kg QW via SC injection for 4 weeks, followed by 6 mg/kg Q4W until marketing authorization as part of this study or a separate extension study, as long as they derive clinical benefit. Participants continued to receive standard-of-care treatments on an episodic basis for the treatment of breakthrough bleeds during the study. |
| OG003 | Arm D: 1.5 mg/kg Emicizumab Prophylaxis QW | Participants <12 years old with hemophilia A and FVIII inhibitors who were enrolled to Arm D received prophylactic emicizumab at a dose of 3 mg/kg via SC injection QW for the first 4 weeks, followed by 1.5 mg/kg via SC injection QW for at least 24 weeks. After 24 weeks of treatment, participants were allowed to continue receiving emicizumab until marketing authorization, as part of this study or a separate extension study, as long as they derived clinical benefit. Participants continued to receive standard-of-care treatments on an episodic basis for the treatment of breakthrough bleeds during the study. |
|
|
| OG001 | Arm B: 6 mg/kg Emicizumab Prophylaxis Q4W | Participants ≥12 years old with hemophilia A (with or without FVIII inhibitors) who were randomized to Arm B received prophylactic emicizumab at a dose of 3 mg/kg via SC injection QW for the first 4 weeks, followed by 6 mg/kg via SC injection Q4W for at least 24 weeks. After 24 weeks of treatment, participants were allowed to continue receiving emicizumab until marketing authorization, as part of this study or a separate extension study, as long as they derived clinical benefit. Participants continued to receive standard-of-care treatments on an episodic basis for the treatment of breakthrough bleeds during the study. |
| OG002 | Arm C (Emi): 6 mg/kg Emicizumab Prophylaxis Q4W | After 24 weeks on no prophylaxis, participants were given the opportunity to switch to receive emicizumab prophylaxis at 3 mg/kg QW via SC injection for 4 weeks, followed by 6 mg/kg Q4W until marketing authorization as part of this study or a separate extension study, as long as they derive clinical benefit. Participants continued to receive standard-of-care treatments on an episodic basis for the treatment of breakthrough bleeds during the study. |
| OG003 | Arm D: 1.5 mg/kg Emicizumab Prophylaxis QW | Participants <12 years old with hemophilia A and FVIII inhibitors who were enrolled to Arm D received prophylactic emicizumab at a dose of 3 mg/kg via SC injection QW for the first 4 weeks, followed by 1.5 mg/kg via SC injection QW for at least 24 weeks. After 24 weeks of treatment, participants were allowed to continue receiving emicizumab until marketing authorization, as part of this study or a separate extension study, as long as they derived clinical benefit. Participants continued to receive standard-of-care treatments on an episodic basis for the treatment of breakthrough bleeds during the study. |
|
|
| OG001 | Arm B: 6 mg/kg Emicizumab Prophylaxis Q4W | Participants ≥12 years old with hemophilia A (with or without FVIII inhibitors) who were randomized to Arm B received prophylactic emicizumab at a dose of 3 mg/kg via SC injection QW for the first 4 weeks, followed by 6 mg/kg via SC injection Q4W for at least 24 weeks. After 24 weeks of treatment, participants were allowed to continue receiving emicizumab until marketing authorization, as part of this study or a separate extension study, as long as they derived clinical benefit. Participants continued to receive standard-of-care treatments on an episodic basis for the treatment of breakthrough bleeds during the study. |
| OG002 | Arm C (Emi): 6 mg/kg Emicizumab Prophylaxis Q4W | After 24 weeks in Arm C (Control) on no prophylaxis, participants were given the opportunity to switch to receive emicizumab prophylaxis at 3 mg/kg QW via SC injection for 4 weeks, followed by 6 mg/kg Q4W until marketing authorization as part of this study or a separate extension study, as long as they derive clinical benefit. Participants continued to receive standard-of-care treatments on an episodic basis for the treatment of breakthrough bleeds during the study. |
| OG003 | Arm D: 1.5 mg/kg Emicizumab Prophylaxis QW | Participants <12 years old with hemophilia A and FVIII inhibitors who were enrolled to Arm D received prophylactic emicizumab at a dose of 3 mg/kg via SC injection QW for the first 4 weeks, followed by 1.5 mg/kg via SC injection QW for at least 24 weeks. After 24 weeks of treatment, participants were allowed to continue receiving emicizumab until marketing authorization, as part of this study or a separate extension study, as long as they derived clinical benefit. Participants continued to receive standard-of-care treatments on an episodic basis for the treatment of breakthrough bleeds during the study. |
|
|
| OG002 | Arm B: 6 mg/kg Emicizumab Prophylaxis Q4W | Participants ≥12 years old with hemophilia A (with or without FVIII inhibitors) who were randomized to Arm B received prophylactic emicizumab at a dose of 3 mg/kg via SC injection QW for the first 4 weeks, followed by 6 mg/kg via SC injection Q4W for at least 24 weeks. After 24 weeks of treatment, participants were allowed to continue receiving emicizumab until marketing authorization, as part of this study or a separate extension study, as long as they derived clinical benefit. Participants continued to receive standard-of-care treatments on an episodic basis for the treatment of breakthrough bleeds during the study. |
| OG003 | Arm D: 1.5 mg/kg Emicizumab Prophylaxis QW | Participants <12 years old with hemophilia A and FVIII inhibitors who were enrolled to Arm D received prophylactic emicizumab at a dose of 3 mg/kg via SC injection QW for the first 4 weeks, followed by 1.5 mg/kg via SC injection QW for at least 24 weeks. After 24 weeks of treatment, participants were allowed to continue receiving emicizumab until marketing authorization, as part of this study or a separate extension study, as long as they derived clinical benefit. Participants continued to receive standard-of-care treatments on an episodic basis for the treatment of breakthrough bleeds during the study. |
|
|
|
| OG002 | Arm B: 6 mg/kg Emicizumab Prophylaxis Q4W | Participants ≥12 years old with hemophilia A (with or without FVIII inhibitors) who were randomized to Arm B received prophylactic emicizumab at a dose of 3 mg/kg via SC injection QW for the first 4 weeks, followed by 6 mg/kg via SC injection Q4W for at least 24 weeks. After 24 weeks of treatment, participants were allowed to continue receiving emicizumab until marketing authorization, as part of this study or a separate extension study, as long as they derived clinical benefit. Participants continued to receive standard-of-care treatments on an episodic basis for the treatment of breakthrough bleeds during the study. |
| OG003 | Arm D: 1.5 mg/kg Emicizumab Prophylaxis QW | Participants <12 years old with hemophilia A and FVIII inhibitors who were enrolled to Arm D received prophylactic emicizumab at a dose of 3 mg/kg via SC injection QW for the first 4 weeks, followed by 1.5 mg/kg via SC injection QW for at least 24 weeks. After 24 weeks of treatment, participants were allowed to continue receiving emicizumab until marketing authorization, as part of this study or a separate extension study, as long as they derived clinical benefit. Participants continued to receive standard-of-care treatments on an episodic basis for the treatment of breakthrough bleeds during the study. |
|
|
| OG002 | Arm B: 6 mg/kg Emicizumab Prophylaxis Q4W | Participants ≥12 years old with hemophilia A (with or without FVIII inhibitors) who were randomized to Arm B received prophylactic emicizumab at a dose of 3 mg/kg via SC injection QW for the first 4 weeks, followed by 6 mg/kg via SC injection Q4W for at least 24 weeks. After 24 weeks of treatment, participants were allowed to continue receiving emicizumab until marketing authorization, as part of this study or a separate extension study, as long as they derived clinical benefit. Participants continued to receive standard-of-care treatments on an episodic basis for the treatment of breakthrough bleeds during the study. |
| OG003 | Arm D: 1.5 mg/kg Emicizumab Prophylaxis QW | Participants <12 years old with hemophilia A and FVIII inhibitors who were enrolled to Arm D received prophylactic emicizumab at a dose of 3 mg/kg via SC injection QW for the first 4 weeks, followed by 1.5 mg/kg via SC injection QW for at least 24 weeks. After 24 weeks of treatment, participants were allowed to continue receiving emicizumab until marketing authorization, as part of this study or a separate extension study, as long as they derived clinical benefit. Participants continued to receive standard-of-care treatments on an episodic basis for the treatment of breakthrough bleeds during the study. |
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