| Primary | Model-Based Annualized Bleed Rate for Treated Bleeds | The number of treated bleeds over the efficacy period was estimated as an annualized bleed 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. | The Treated Population comprises all participants who received at least one dose of emicizumab. | Posted | | Number | 95% Confidence Interval | Treated bleeds per year | | From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks) | | | | ID | Title | Description |
|---|
| OG000 | Emicizumab | Participants with mild and moderate hemophilia A without factor VIII (FVIII) inhibitors were enrolled to receive the emicizumab loading dose regimen (emicizumab 3 mg/kg administered subcutaneously [SC] QW for 4 weeks) followed by participant preference of one of the following 3 emicizumab SC maintenance dose regimens: 1.5 mg/kg QW, 3 mg/kg Q2W, or 6 mg/kg Q4W. |
| | | Title | Denominators | Categories |
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| | |
| |
| Primary | Mean Calculated Annualized Bleed Rate for Treated Bleeds | The number of treated bleeds over the efficacy period is presented here as a calculated annualized bleed 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. | The Treated Population comprises all participants who received at least one dose of emicizumab. | Posted | | Mean | 95% Confidence Interval | Treated bleeds per year | | From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks) | | | | ID | Title | Description |
|---|
| OG000 | Emicizumab | Participants with mild and moderate hemophilia A without factor VIII (FVIII) inhibitors were enrolled to receive the emicizumab loading dose regimen (emicizumab 3 mg/kg administered subcutaneously [SC] QW for 4 weeks) followed by participant preference of one of the following 3 emicizumab SC maintenance dose regimens: 1.5 mg/kg QW, 3 mg/kg Q2W, or 6 mg/kg Q4W. |
| |
| Primary | Median Calculated Annualized Bleed Rate for Treated Bleeds | The number of treated bleeds over the efficacy period is presented here as a calculated annualized bleed 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. | The Treated Population comprises all participants who received at least one dose of emicizumab. | Posted | | Median | Inter-Quartile Range | Treated bleeds per year | | From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks) | | | | ID | Title | Description |
|---|
| OG000 | Emicizumab | Participants with mild and moderate hemophilia A without factor VIII (FVIII) inhibitors were enrolled to receive the emicizumab loading dose regimen (emicizumab 3 mg/kg administered subcutaneously [SC] QW for 4 weeks) followed by participant preference of one of the following 3 emicizumab SC maintenance dose regimens: 1.5 mg/kg QW, 3 mg/kg Q2W, or 6 mg/kg Q4W. |
| |
| Secondary | Model-Based Annualized Bleed Rate for All Bleeds | The number of all bleeds over the efficacy period was estimated as an annualized bleed 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. | The Treated Population comprises all participants who received at least one dose of emicizumab. | Posted | | Number | 95% Confidence Interval | All bleeds per year | | From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks) | | | | ID | Title | Description |
|---|
| OG000 | Emicizumab | Participants with mild and moderate hemophilia A without factor VIII (FVIII) inhibitors were enrolled to receive the emicizumab loading dose regimen (emicizumab 3 mg/kg administered subcutaneously [SC] QW for 4 weeks) followed by participant preference of one of the following 3 emicizumab SC maintenance dose regimens: 1.5 mg/kg QW, 3 mg/kg Q2W, or 6 mg/kg Q4W. |
| |
| Secondary | Mean Calculated Annualized Bleed Rate for All Bleeds | The number of all bleeds over the efficacy period is presented here as a calculated annualized bleed 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. | The Treated Population comprises all participants who received at least one dose of emicizumab. | Posted | | Mean | 95% Confidence Interval | All bleeds per year | | From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks) | | | | ID | Title | Description |
|---|
| OG000 | Emicizumab | Participants with mild and moderate hemophilia A without factor VIII (FVIII) inhibitors were enrolled to receive the emicizumab loading dose regimen (emicizumab 3 mg/kg administered subcutaneously [SC] QW for 4 weeks) followed by participant preference of one of the following 3 emicizumab SC maintenance dose regimens: 1.5 mg/kg QW, 3 mg/kg Q2W, or 6 mg/kg Q4W. |
|
| Secondary | Median Calculated Annualized Bleed Rate for All Bleeds | The number of all bleeds over the efficacy period is presented here as a calculated annualized bleed 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. | The Treated Population comprises all participants who received at least one dose of emicizumab. | Posted | | Median | Inter-Quartile Range | All bleeds per year | | From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks) | | | | ID | Title | Description |
|---|
| OG000 | Emicizumab | Participants with mild and moderate hemophilia A without factor VIII (FVIII) inhibitors were enrolled to receive the emicizumab loading dose regimen (emicizumab 3 mg/kg administered subcutaneously [SC] QW for 4 weeks) followed by participant preference of one of the following 3 emicizumab SC maintenance dose regimens: 1.5 mg/kg QW, 3 mg/kg Q2W, or 6 mg/kg Q4W. |
|
| Secondary | Model-Based Annualized Bleed Rate for Treated Joint Bleeds | The number of treated joint bleeds over the efficacy period was estimated as an annualized bleed 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. | The Treated Population comprises all participants who received at least one dose of emicizumab. | Posted | | Number | 95% Confidence Interval | Treated joint bleeds per year | | From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks) | | | | ID | Title | Description |
|---|
| OG000 | Emicizumab | Participants with mild and moderate hemophilia A without factor VIII (FVIII) inhibitors were enrolled to receive the emicizumab loading dose regimen (emicizumab 3 mg/kg administered subcutaneously [SC] QW for 4 weeks) followed by participant preference of one of the following 3 emicizumab SC maintenance dose regimens: 1.5 mg/kg QW, 3 mg/kg Q2W, or 6 mg/kg Q4W. |
| |
| Secondary | Mean Calculated Annualized Bleed Rate for Treated Joint Bleeds | The number of treated joint bleeds over the efficacy period is presented here as a calculated annualized bleed 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. | The Treated Population comprises all participants who received at least one dose of emicizumab. | Posted | | Mean | 95% Confidence Interval | Treated joint bleeds per year | | From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks) | | | | ID | Title | Description |
|---|
| OG000 | Emicizumab | Participants with mild and moderate hemophilia A without factor VIII (FVIII) inhibitors were enrolled to receive the emicizumab loading dose regimen (emicizumab 3 mg/kg administered subcutaneously [SC] QW for 4 weeks) followed by participant preference of one of the following 3 emicizumab SC maintenance dose regimens: 1.5 mg/kg QW, 3 mg/kg Q2W, or 6 mg/kg Q4W. |
|
| Secondary | Median Calculated Annualized Bleed Rate for Treated Joint Bleeds | The number of treated joint bleeds over the efficacy period is presented here as a calculated annualized bleed 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. | The Treated Population comprises all participants who received at least one dose of emicizumab. | Posted | | Median | Inter-Quartile Range | Treated joint bleeds per year | | From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks) | | | | ID | Title | Description |
|---|
| OG000 | Emicizumab | Participants with mild and moderate hemophilia A without factor VIII (FVIII) inhibitors were enrolled to receive the emicizumab loading dose regimen (emicizumab 3 mg/kg administered subcutaneously [SC] QW for 4 weeks) followed by participant preference of one of the following 3 emicizumab SC maintenance dose regimens: 1.5 mg/kg QW, 3 mg/kg Q2W, or 6 mg/kg Q4W. |
|
| Secondary | Model-Based Annualized Bleed Rate for Treated Target Joint Bleeds | The number of treated target joint bleeds over the efficacy period was estimated as an annualized bleed 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. | The Treated Population comprises all participants who received at least one dose of emicizumab. | Posted | | Number | 95% Confidence Interval | Treated target joint bleeds per year | | From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks) | | | | ID | Title | Description |
|---|
| OG000 | Emicizumab | Participants with mild and moderate hemophilia A without factor VIII (FVIII) inhibitors were enrolled to receive the emicizumab loading dose regimen (emicizumab 3 mg/kg administered subcutaneously [SC] QW for 4 weeks) followed by participant preference of one of the following 3 emicizumab SC maintenance dose regimens: 1.5 mg/kg QW, 3 mg/kg Q2W, or 6 mg/kg Q4W. |
|
| Secondary | Mean Calculated Annualized Bleed Rate for Treated Target Joint Bleeds | The number of treated target joint bleeds over the efficacy period is presented here as a calculated annualized bleed 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. | The Treated Population comprises all participants who received at least one dose of emicizumab. | Posted | | Mean | 95% Confidence Interval | Treated target joint bleeds per year | | From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks) | | | | ID | Title | Description |
|---|
| OG000 | Emicizumab | Participants with mild and moderate hemophilia A without factor VIII (FVIII) inhibitors were enrolled to receive the emicizumab loading dose regimen (emicizumab 3 mg/kg administered subcutaneously [SC] QW for 4 weeks) followed by participant preference of one of the following 3 emicizumab SC maintenance dose regimens: 1.5 mg/kg QW, 3 mg/kg Q2W, or 6 mg/kg Q4W. |
|
| Secondary | Median Calculated Annualized Bleed Rate for Treated Target Joint Bleeds | The number of treated target joint bleeds over the efficacy period is presented here as a calculated annualized bleed 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. | The Treated Population comprises all participants who received at least one dose of emicizumab. | Posted | | Median | Inter-Quartile Range | Treated target joint bleeds per year | | From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks) | | | | ID | Title | Description |
|---|
| OG000 | Emicizumab | Participants with mild and moderate hemophilia A without factor VIII (FVIII) inhibitors were enrolled to receive the emicizumab loading dose regimen (emicizumab 3 mg/kg administered subcutaneously [SC] QW for 4 weeks) followed by participant preference of one of the following 3 emicizumab SC maintenance dose regimens: 1.5 mg/kg QW, 3 mg/kg Q2W, or 6 mg/kg Q4W. |
|
| Secondary | Model-Based Annualized Bleed Rate for Treated Spontaneous Bleeds | The number of treated spontaneous bleeds over the efficacy period was estimated as an annualized bleed 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. | The Treated Population comprises all participants who received at least one dose of emicizumab. | Posted | | Number | 95% Confidence Interval | Treated spontaneous bleeds | | From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks) | | | | ID | Title | Description |
|---|
| OG000 | Emicizumab | Participants with mild and moderate hemophilia A without factor VIII (FVIII) inhibitors were enrolled to receive the emicizumab loading dose regimen (emicizumab 3 mg/kg administered subcutaneously [SC] QW for 4 weeks) followed by participant preference of one of the following 3 emicizumab SC maintenance dose regimens: 1.5 mg/kg QW, 3 mg/kg Q2W, or 6 mg/kg Q4W. |
| |
| Secondary | Mean Calculated Annualized Bleed Rate for Treated Spontaneous Bleeds | The number of treated spontaneous bleeds over the efficacy period is presented here as a calculated annualized bleed 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. | The Treated Population comprises all participants who received at least one dose of emicizumab. | Posted | | Mean | 95% Confidence Interval | Treated spontaneous bleeds | | From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks) | | | | ID | Title | Description |
|---|
| OG000 | Emicizumab | Participants with mild and moderate hemophilia A without factor VIII (FVIII) inhibitors were enrolled to receive the emicizumab loading dose regimen (emicizumab 3 mg/kg administered subcutaneously [SC] QW for 4 weeks) followed by participant preference of one of the following 3 emicizumab SC maintenance dose regimens: 1.5 mg/kg QW, 3 mg/kg Q2W, or 6 mg/kg Q4W. |
| |
| Secondary | Median Calculated Annualized Bleed Rate for Treated Spontaneous Bleeds | The number of treated spontaneous bleeds over the efficacy period is presented here as a calculated annualized bleed 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. | The Treated Population comprises all participants who received at least one dose of emicizumab. | Posted | | Median | Inter-Quartile Range | Treated spontaneous bleeds | | From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks) | | | | ID | Title | Description |
|---|
| OG000 | Emicizumab | Participants with mild and moderate hemophilia A without factor VIII (FVIII) inhibitors were enrolled to receive the emicizumab loading dose regimen (emicizumab 3 mg/kg administered subcutaneously [SC] QW for 4 weeks) followed by participant preference of one of the following 3 emicizumab SC maintenance dose regimens: 1.5 mg/kg QW, 3 mg/kg Q2W, or 6 mg/kg Q4W. |
| |
| Secondary | CATCH Questionnaire for Adult Participants: Change From Baseline in the Daily Activity Risk Perception and Impact Domain Scores Over Time | CATCH = Comprehensive Assessment Tool of Challenges in Hemophilia | | Not Posted | Dec 2026 | | | | | Baseline (Week 1), Weeks 13, 25, 37 and 49, and every 12 weeks thereafter until Study Completion/Discontinuation Visit (up to approximately 48 months) | | Participants | | | | |
| Secondary | CATCH Questionnaire for Adult Participants: Change From Baseline in the Social Activity Risk Perception and Impact Domain Scores Over Time | CATCH = Comprehensive Assessment Tool of Challenges in Hemophilia | | Not Posted | Dec 2026 | | | | | Baseline (Week 1), Weeks 13, 25, 37 and 49, and every 12 weeks thereafter until Study Completion/Discontinuation Visit (up to approximately 48 months) | | Participants | | | | |
| Secondary | CATCH Questionnaire for Adult Participants: Change From Baseline in the Recreational Activity Risk Perception and Impact Domain Scores Over Time | CATCH = Comprehensive Assessment Tool of Challenges in Hemophilia | | Not Posted | Dec 2026 | | | | | Baseline (Week 1), Weeks 13, 25, 37 and 49, and every 12 weeks thereafter until Study Completion/Discontinuation Visit (up to approximately 48 months) | | Participants | | | | |
| Secondary | CATCH Questionnaire for Adult Participants: Change From Baseline in the Work Impact Domain Score Over Time | CATCH = Comprehensive Assessment Tool of Challenges in Hemophilia | | Not Posted | Dec 2026 | | | | | Baseline (Week 1), Weeks 13, 25, 37 and 49, and every 12 weeks thereafter until Study Completion/Discontinuation Visit (up to approximately 48 months) | | Participants | | | | |
| Secondary | CATCH Questionnaire for Adult Participants: Change From Baseline in the Preoccupation Domain Score Over Time | CATCH = Comprehensive Assessment Tool of Challenges in Hemophilia | | Not Posted | Dec 2026 | | | | | Baseline (Week 1), Weeks 13, 25, 37 and 49, and every 12 weeks thereafter until Study Completion/Discontinuation Visit (up to approximately 48 months) | | Participants | | | | |
| Secondary | CATCH Questionnaire for Adult Participants: Change From Baseline in the Treatment Burden Domain Score Over Time | CATCH = Comprehensive Assessment Tool of Challenges in Hemophilia | | Not Posted | Dec 2026 | | | | | Baseline (Week 1), Weeks 13, 25, 37 and 49, and every 12 weeks thereafter until Study Completion/Discontinuation Visit (up to approximately 48 months) | | Participants | | | | |
| Secondary | CATCH Questionnaire for Adult Participants: Number of Participants by Responses to Their Level of Pain Associated With a Bleed Over Time | CATCH = Comprehensive Assessment Tool of Challenges in Hemophilia | | Not Posted | Dec 2026 | | | | | Baseline (Week 1), Weeks 13, 25, 37 and 49, and every 12 weeks thereafter until Study Completion/Discontinuation Visit (up to approximately 48 months) | | Participants | | | | |
| Secondary | CATCH Questionnaire for Adult Participants: Number of Participants by Responses to Their Level of Pain in Target Joints Over Time | CATCH = Comprehensive Assessment Tool of Challenges in Hemophilia | | Not Posted | Dec 2026 | | | | | Baseline (Week 1), Weeks 13, 25, 37 and 49, and every 12 weeks thereafter until Study Completion/Discontinuation Visit (up to approximately 48 months) | | Participants | | | | |
| Secondary | CATCH Questionnaire for Adult Participants: Number of Participants by Responses to Their Level of Pain at Its Worst Over Time | CATCH = Comprehensive Assessment Tool of Challenges in Hemophilia | | Not Posted | Dec 2026 | | | | | Baseline (Week 1), Weeks 13, 25, 37 and 49, and every 12 weeks thereafter until Study Completion/Discontinuation Visit (up to approximately 48 months) | | Participants | | | | |
| Secondary | CATCH Questionnaire for Adult Participants: Number of Participants by Responses to Their Level of Pain at Its Least Over Time | CATCH = Comprehensive Assessment Tool of Challenges in Hemophilia | | Not Posted | Dec 2026 | | | | | Baseline (Week 1), Weeks 13, 25, 37 and 49, and every 12 weeks thereafter until Study Completion/Discontinuation Visit (up to approximately 48 months) | | Participants | | | | |
| Secondary | CATCH Questionnaire for Adult Participants: Number of Participants by Responses to Their Level of Pain on Average Over Time | CATCH = Comprehensive Assessment Tool of Challenges in Hemophilia | | Not Posted | Dec 2026 | | | | | Baseline (Week 1), Weeks 13, 25, 37 and 49, and every 12 weeks thereafter until Study Completion/Discontinuation Visit (up to approximately 48 months) | | Participants | | | | |
| Secondary | CATCH Questionnaire for Pediatric Participants: Change From Baseline in the Daily Activity Risk Perception and Impact Domain Score Over Time | CATCH = Comprehensive Assessment Tool of Challenges in Hemophilia | | Not Posted | Dec 2026 | | | | | Baseline (Week 1), Weeks 13, 25, 37 and 49, and every 12 weeks thereafter until Study Completion/Discontinuation Visit (up to approximately 48 months) | | Participants | | | | |
| Secondary | CATCH Questionnaire for Pediatric Participants: Change From Baseline in the Social Activity Risk Perception and Impact Domain Score Over Time | CATCH = Comprehensive Assessment Tool of Challenges in Hemophilia | | Not Posted | Dec 2026 | | | | | Baseline (Week 1), Weeks 13, 25, 37 and 49, and every 12 weeks thereafter until Study Completion/Discontinuation Visit (up to approximately 48 months) | | Participants | | | | |
| Secondary | CATCH Questionnaire for Pediatric Participants: Change From Baseline in the Recreational Activity Risk Perception and Impact Domain Score Over Time | CATCH = Comprehensive Assessment Tool of Challenges in Hemophilia | | Not Posted | Dec 2026 | | | | | Baseline (Week 1), Weeks 13, 25, 37 and 49, and every 12 weeks thereafter until Study Completion/Discontinuation Visit (up to approximately 48 months) | | Participants | | | | |
| Secondary | CATCH Questionnaire for Pediatric Participants: Change From Baseline in the School Impact Domain Score Over Time | CATCH = Comprehensive Assessment Tool of Challenges in Hemophilia | | Not Posted | Dec 2026 | | | | | Baseline (Week 1), Weeks 13, 25, 37 and 49, and every 12 weeks thereafter until Study Completion/Discontinuation Visit (up to approximately 48 months) | | Participants | | | | |
| Secondary | CATCH Questionnaire for Pediatric Participants: Change From Baseline in the Preoccupation Domain Score Over Time | CATCH = Comprehensive Assessment Tool of Challenges in Hemophilia | | Not Posted | Dec 2026 | | | | | Baseline (Week 1), Weeks 13, 25, 37 and 49, and every 12 weeks thereafter until Study Completion/Discontinuation Visit (up to approximately 48 months) | | Participants | | | | |
| Secondary | CATCH Questionnaire for Pediatric Participants: Change From Baseline in the Treatment Burden Domain Score Over Time | CATCH = Comprehensive Assessment Tool of Challenges in Hemophilia | | Not Posted | Dec 2026 | | | | | Baseline (Week 1), Weeks 13, 25, 37 and 49, and every 12 weeks thereafter until Study Completion/Discontinuation Visit (up to approximately 48 months) | | Participants | | | | |
| Secondary | CATCH Questionnaire for Pediatric Participants: Number of Participants by Responses to Their Level of Pain Associated With a Bleed Over Time | CATCH = Comprehensive Assessment Tool of Challenges in Hemophilia | | Not Posted | Dec 2026 | | | | | Baseline (Week 1), Weeks 13, 25, 37 and 49, and every 12 weeks thereafter until Study Completion/Discontinuation Visit (up to approximately 48 months) | | Participants | | | | |
| Secondary | CATCH Questionnaire for Pediatric Participants: Number of Participants by Responses to Their Level of Pain at Its Worst Over Time | CATCH = Comprehensive Assessment Tool of Challenges in Hemophilia | | Not Posted | Dec 2026 | | | | | Baseline (Week 1), Weeks 13, 25, 37 and 49, and every 12 weeks thereafter until Study Completion/Discontinuation Visit (up to approximately 48 months) | | Participants | | | | |
| Secondary | CATCH Questionnaire for Caregivers: Change From Baseline in the Preoccupation Domain Score Over Time | CATCH = Comprehensive Assessment Tool of Challenges in Hemophilia | | Not Posted | Dec 2026 | | | | | Baseline (Week 1), Weeks 13, 25, 37 and 49, and every 12 weeks thereafter until Study Completion/Discontinuation Visit (up to approximately 48 months) | | Participants | | | | |
| Secondary | CATCH Questionnaire for Caregivers: Change From Baseline in the Treatment Burden Domain Score Over Time | CATCH = Comprehensive Assessment Tool of Challenges in Hemophilia | | Not Posted | Dec 2026 | | | | | Baseline (Week 1), Weeks 13, 25, 37 and 49, and every 12 weeks thereafter until Study Completion/Discontinuation Visit (up to approximately 48 months) | | Participants | | | | |
| Secondary | Percentage of Participants Who Prefer Emicizumab SC Treatment, Their Previous Hemophilia IV Treatment, or Have No Preference, as Assessed Through Use of the Emicizumab Preference Survey at Week 17 | The Emicizumab Preference Survey is a fit-for-purpose questionnaire developed by the sponsor to record the participant's preference for treatment with subcutaneous (SC) emicizumab, intravenous (IV) factor VIIII (FVIII), or no preference. The 95% confidence intervals were calculated using the Pearson-Clopper method. | The analysis population included all treated participants 12 years of age or older who provided a response to the survey at Week 17. | Posted | | Number | 95% Confidence Interval | Percentage of participants | | Week 17 | | | | ID | Title | Description |
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| OG000 | Emicizumab | Participants with mild and moderate hemophilia A without factor VIII (FVIII) inhibitors were enrolled to receive the emicizumab loading dose regimen (emicizumab 3 mg/kg administered subcutaneously [SC] QW for 4 weeks) followed by participant preference of one of the following 3 emicizumab SC maintenance dose regimens: 1.5 mg/kg QW, 3 mg/kg Q2W, or 6 mg/kg Q4W. |
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| Secondary | Percentage of Caregivers Who Prefer Emicizumab SC Treatment, Their Child's Previous Hemophilia IV Treatment, or Have No Preference, as Assessed Through Use of the Emicizumab Preference Survey at Week 17 | The Emicizumab Preference Survey is a fit-for-purpose questionnaire developed by the sponsor to record the caregiver's preference for their child's treatment with subcutaneous (SC) emicizumab, intravenous (IV) factor VIIII (FVIII), or no preference. The 95% confidence intervals were calculated using the Pearson-Clopper method. | The analysis population included all caregivers of treated participants less than 18 years of age and who provided a response to the survey at Week 17. | Posted | | Number | 95% Confidence Interval | Percentage of caregivers | | Week 17 | | | | ID | Title | Description |
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| OG000 | Emicizumab | Participants with mild and moderate hemophilia A without factor VIII (FVIII) inhibitors were enrolled to receive the emicizumab loading dose regimen (emicizumab 3 mg/kg administered subcutaneously [SC] QW for 4 weeks) followed by participant preference of one of the following 3 emicizumab SC maintenance dose regimens: 1.5 mg/kg QW, 3 mg/kg Q2W, or 6 mg/kg Q4W. |
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| Secondary | Hemophilia Joint Health Scores Over Time | | | Not Posted | Dec 2026 | | | | | Days -7 to -1, Weeks 25, 49, and every 24 weeks thereafter until Study Completion/Discontinuation Visit (up to approximately 48 months) | | Participants | | | | |
| Secondary | Change From Baseline in Mean Daily Peak Activity Duration Over Time | For physical activity assessment, participants ≥5 years of age were instructed to wear the study accelerometry device on the wrist continuously (24 hours/day) every day for the designated 2-week periods during the study. A participant was considered to be compliant with the physical activity assessments if they wore the study device continuously (≥8 hours/day) every day for at least 8 days of each of the designated 2-week periods during the study. If this compliance criterion was not reached at a specific timepoint the participant was not included in the analysis of the designated 2-week periods where compliance was not reached. Daily measurements were averaged over the 14-days timepoint. Activity count was a measure of the acceleration measured by the device. The daily peak activity duration was defined as the sum of moderate to vigorous activity per day (in minutes). | The analysis population is treated participants ≥5 years old, further subdivided into analysis groups of ≥5 to <18 years old and ≥18 years old. The number analyzed for change from baseline (BL) are participants with data that met the compliance criterion at BL and the specified timepoint (i.e., wearing device ≥8 hours/day for ≥8 days per period); if it was not met, they were excluded for each noncompliant timepoint. The overall number analyzed had valid data for at least the BL timepoint. | Posted | | Mean | Standard Deviation | Minutes per day | | Baseline (Weeks 1-2) and Weeks 13 (Weeks 12-13 period), 25 (Weeks 24-25 period), 37 (Weeks 36-37 period), and 49 (Weeks 48-49 period) | | | | ID | Title | Description |
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| OG000 | Participants ≥5 Years Old: Emicizumab | Participants with mild and moderate hemophilia A without factor VIII (FVIII) inhibitors were enrolled and received the emicizumab loading dose regimen (emicizumab 3 mg/kg administered subcutaneously QW for 4 weeks) followed by participant preference of one of the following 3 emicizumab maintenance dose regimens: 1.5 mg/kg QW, 3 mg/kg Q2W, or 6 mg/kg Q4W. |
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| Secondary | Change From Baseline in Mean Daily Step Count Over Time | For physical activity assessment, participants ≥5 years of age were instructed to wear the study accelerometry device on the wrist continuously (24 hours/day) every day for the designated 2-week periods during the study. A participant was considered to be compliant with the physical activity assessments if they wore the study device continuously (≥8 hours/day) every day for at least 8 days of each of the designated 2-week periods during the study. If this compliance criterion was not reached at a specific timepoint the participant was not included in the analysis of the designated 2-week periods where compliance was not reached. Daily measurements were averaged over the 14-days timepoint. Activity count was a measure of the acceleration measured by the device. | The analysis population is treated participants ≥5 years old, further subdivided into analysis groups of ≥5 to <18 years old and ≥18 years old. The number analyzed for change from baseline (BL) are participants with data that met the compliance criterion at BL and the specified timepoint (i.e., wearing device ≥8 hours/day for ≥8 days per period); if it was not met, they were excluded for each noncompliant timepoint. The overall number analyzed had valid data for at least the BL timepoint. | Posted | | Mean | Standard Deviation | Step count per day | | Baseline (Weeks 1-2) and Weeks 13 (Weeks 12-13), 25 (Weeks 24-25), 37 (Weeks 36-37), and 49 (Weeks 48-49) | | | | ID | Title | Description |
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| OG000 | Participants ≥5 Years Old: Emicizumab | Participants with mild and moderate hemophilia A without factor VIII (FVIII) inhibitors were enrolled and received the emicizumab loading dose regimen (emicizumab 3 mg/kg administered subcutaneously QW for 4 weeks) followed by participant preference of one of the following 3 emicizumab maintenance dose regimens: 1.5 mg/kg QW, 3 mg/kg Q2W, or 6 mg/kg Q4W. |
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| Secondary | Menstrual Bleed Questionnaire (MBQ) for Female Participants of Childbearing Potential: Change From Baseline in the MBQ Total Score Over Time | | | Not Posted | Dec 2026 | | | | | Baseline (Week 1), Weeks 5, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, 49, and every 4 weeks thereafter until Study Completion (up to approximately 48 months) | | Participants | | | | |
| Secondary | Menstrual Bleed Questionnaire (MBQ) for Female Participants of Childbearing Potential: Change From Baseline in the Heaviness Subscale Score Over Time | | | Not Posted | Dec 2026 | | | | | Baseline (Week 1), Weeks 5, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, 49, and every 4 weeks thereafter until Study Completion (up to approximately 48 months) | | Participants | | | | |
| Secondary | Menstrual Bleed Questionnaire (MBQ) for Female Participants of Childbearing Potential: Change From Baseline in the Quality of Life Subscale Score Over Time | | | Not Posted | Dec 2026 | | | | | Baseline (Week 1), Weeks 5, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, 49, and every 4 weeks thereafter until Study Completion (up to approximately 48 months) | | Participants | | | | |
| Secondary | Menstrual Bleed Questionnaire (MBQ) for Female Participants of Childbearing Potential: Change From Baseline in the Irregularity Subscale Score Over Time | | | Not Posted | Dec 2026 | | | | | Baseline (Week 1), Weeks 5, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, 49, and every 4 weeks thereafter until Study Completion (up to approximately 48 months) | | Participants | | | | |
| Secondary | Menstrual Bleed Questionnaire (MBQ) for Female Participants of Childbearing Potential: Change From Baseline in the Pain Subscale Score Over Time | | | Not Posted | Dec 2026 | | | | | Baseline (Week 1), Weeks 5, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, 49, and every 4 weeks thereafter until Study Completion (up to approximately 48 months) | | Participants | | | | |
| Secondary | Menstruation Diary With the Pictorial Blood Assessment Chart (PBAC) for Female Participants of Childbearing Potential: PBAC Scores Over Time | | | Not Posted | Dec 2026 | | | | | Baseline (Day 1) and monthly (on days of menstruation) until Study Completion (up to approximately 48 months) | | Participants | | | | |
| Secondary | Number of Participants With at Least One Adverse Event by Severity, According to the World Health Organization (WHO) Toxicity Grading Scale | | | Not Posted | Dec 2026 | | | | | From Screening (Day -28 to -1) to Study Completion/Discontinuation Visit (up to approximately 48 months) | | Participants | | | | |
| Secondary | Number of Participants With Adverse Events Leading to Study Drug Discontinuation | | | Not Posted | Dec 2026 | | | | | From Screening (Day -28 to -1) to Study Completion/Discontinuation Visit (up to approximately 48 months) | | Participants | | | | |
| Secondary | Number of Participants With at Least One Thromboembolic Event | | | Not Posted | Dec 2026 | | | | | From Screening (Day -28 to -1) to Study Completion/Discontinuation Visit (up to approximately 48 months) | | Participants | | | | |
| Secondary | Number of Participants With at Least One Event of Thrombotic Microangiopathy | | | Not Posted | Dec 2026 | | | | | From Screening (Day -28 to -1) to Study Completion/Discontinuation Visit (up to approximately 48 months) | | Participants | | | | |
| Secondary | Number of Participants With at Least One Injection-Site Reaction by Severity, According to the WHO Toxicity Grading Scale | | | Not Posted | Dec 2026 | | | | | From Screening (Day -28 to -1) to Study Completion/Discontinuation Visit (up to approximately 48 months) | | Participants | | | | |
| Secondary | Number of Participants With at Least One Severe Hypersensitivity, Anaphylaxis, and Anaphylactoid Event | | | Not Posted | Dec 2026 | | | | | From Screening (Day -28 to -1) to Study Completion/Discontinuation Visit (up to approximately 48 months) | | Participants | | | | |
| Secondary | Number of Participants With at Least One Laboratory Abnormality | Laboratory parameters for hematology and blood chemistry will be measured and compared with a standard reference range. Values outside the standard reference range are considered abnormalities. Not every laboratory abnormality qualifies as an adverse event. A laboratory test result will 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 or a medical intervention; or is clinically significant in the investigator's judgment. | | Not Posted | Dec 2026 | | | | | From Screening (Day -28 to -1) to Study Completion/Discontinuation Visit (up to approximately 48 months) | | Participants | | | | |
| Secondary | Change From Baseline in Respiratory Rate Over Time | | | Not Posted | Dec 2026 | | | | | Baseline, Weeks 1, 2, 3, 4, 5, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, and 49, and every 12 weeks thereafter until Study Completion/Discontinuation Visit (up to approximately 48 months) | | Participants | | | | |
| Secondary | Change From Baseline in Pulse Rate Over Time | | | Not Posted | Dec 2026 | | | | | Baseline, Weeks 1, 2, 3, 4, 5, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, and 49, and every 12 weeks thereafter until Study Completion/Discontinuation Visit (up to approximately 48 months) | | Participants | | | | |
| Secondary | Change From Baseline in Body Temperature Over Time | | | Not Posted | Dec 2026 | | | | | Baseline, Weeks 1, 2, 3, 4, 5, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, and 49, and every 12 weeks thereafter until Study Completion/Discontinuation Visit (up to approximately 48 months) | | Participants | | | | |
| Secondary | Change From Baseline in Systolic Blood Pressure Over Time | | | Not Posted | Dec 2026 | | | | | Baseline, Weeks 1, 2, 3, 4, 5, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, and 49, and every 12 weeks thereafter until Study Completion/Discontinuation Visit (up to approximately 48 months) | | Participants | | | | |
| Secondary | Change From Baseline in Diastolic Blood Pressure Over Time | | | Not Posted | Dec 2026 | | | | | Baseline, Weeks 1, 2, 3, 4, 5, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, and 49, and every 12 weeks thereafter until Study Completion/Discontinuation Visit (up to approximately 48 months) | | Participants | | | | |
| Secondary | Change From Baseline in Electrocardiogram (ECG) Parameters Over Time: QT, QTcB, QTcF, RR, PR, and QRS Intervals | | | Not Posted | Dec 2026 | | | | | Baseline, Weeks 5, 25, and 49, and every 12 weeks thereafter until Study Completion/Discontinuation Visit (up to approximately 48 months) | | Participants | | | | |
| Secondary | Change From Baseline in Heart Rate Over Time, as Measured by Electrocardiogram (ECG) | | | Not Posted | Dec 2026 | | | | | Baseline, Weeks 5, 25, and 49, and every 12 weeks thereafter until Study Completion/Discontinuation Visit (up to approximately 48 months) | | Participants | | | | |
| Secondary | Plasma Trough Concentration (Ctrough) of Emicizumab Over Time | | | Not Posted | Dec 2026 | | | | | Pre-dose at Weeks 1, 2, 3, 4, 5, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, and 49, and every 12 weeks thereafter until study completion/discontinuation (up to approximately 48 months) | | Participants | | | | |
| Secondary | Number of Participants With Anti-Drug Antibodies Against Emicizumab at Baseline and Post-Baseline | | | Not Posted | Dec 2026 | | | | | Pre-dose at Baseline (Week 1) and Weeks 5, 13, 25, 33, 41, and 49, and every 12 weeks thereafter until study completion/discontinuation (up to approximately 48 months) | | Participants | | | | |
| Secondary | Number of Participants Who Develop Anti-FVIII Inhibitors Over Time | | | Not Posted | Dec 2026 | | | | | Screening (Day -28 to -1) and Weeks 1, 13, 25, 37, and 49, and every 12 weeks thereafter until study completion/discontinuation (up to approximately 48 months) | | Participants | | | | |