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| ID | Type | Description | Link |
|---|---|---|---|
| 2022-004149-11 | EudraCT Number |
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| Name | Class |
|---|---|
| Takeda Development Center Americas, Inc. | INDUSTRY |
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The main aim of this study is to learn more about side effects of Advate when given as standard treatment to people with hemophilia A who have already been treated.
The study sponsor will not be involved in how participants are treated but will provide instructions on how the clinics will record what happens during the study. Participants will need to visit the study doctor 5 times in total during the study. During these visits, study data will be collected by the study doctor.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Hemophilia A Group | Experimental | Participants with hemophilia A were treated with ADVATE according to a regimen determined by the treating physician at the study site and in accordance with the national product label under standard clinical practice for 6.7 months. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| ADVATE | Biological | Antihemophilic factor (AHF) activity expressed in international units (IU) per vial. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants With Serious Adverse Events (SAE) at Least Possibly Related to ADVATE | An adverse event (AE) was defined as any untoward medical occurrence in a participant administered a pharmaceutical product and that does not necessarily have a causal relationship with this investigational product or medicinal product. An SAE was defined as any untoward medical occurrence that resulted in death; was life-threatening; required inpatient hospitalization or prolongation of present hospitalization; resulted in persistent or significant disability/incapacity; was a congenital anomaly/birth defect or was a medically important event. Number of participants with SAEs (including FVIII inhibitor formation) that were at least possibly related to ADVATE were reported. | Baseline (Day 0) up to end of study (up to 12.9 months) |
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants With Non-serious Adverse Events (AEs) at Least Possibly Related to ADVATE | An AE was any untoward medical occurrence in a participant administered a pharmaceutical product and that does not necessarily have a causal relationship with this investigational product or medicinal product. Number of participants with non-serious AEs that were at least possibly related to ADVATE were reported. |
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Inclusion Criteria:
The participant or legally authorized representative (in case of study participants less than (<) 18 years of age) gave written informed consent to participate in the study.
Participant of any age with hemophilia A.
Participant defined as a previously treated patient (PTP):
Participant as negative history of FVIII inhibitors and negative inhibitor at screening defined as less than 0.6 Bethesda units (BU) per milliliter (Nijmegen-modified Bethesda assay).
Participant is human immunodeficiency virus negative (HIV-); or human immunodeficiency virus positive (HIV+) with stable disease and cluster of differentiation 4 (CD4+) count >=200 cells per cubic millimeter (mm^3), as confirmed by central laboratory at screening.
Participant is hepatitis C virus negative (HCV-) by antibody or polymerase chain reaction (PCR) testing (if positive, anti-body titer will be confirmed by PCR), as confirmed by central laboratory at screening; or hepatitis C virus positive (HCV+) with chronic stable hepatitis.
Participant is willing and able to comply with the requirements of the protocol.
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Medical Director | Takeda | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Amrita Institute of Medical Science & Research Centre | Ernākulam | Kerala | 682041 | India | ||
| St. John's Medical College |
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| Label | URL |
|---|---|
| To obtain more information on the study, click here/on this link | View source |
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Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.
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IPD from eligible studies will be shared with qualified researchers according to the criteria and process described on https://vivli.org/ourmember/takeda/. For approved requests, the researchers will be provided access to anonymized data (to respect patient privacy in line with applicable laws and regulations) and with information necessary to address the research objectives under the terms of a data sharing agreement.
Participants previously treated for hemophilia A who met eligibility criteria were enrolled to receive ADVATE according to a dosing regimen determined by the treating physician and in accordance with the national product label.
Participants took part in the study at 4 investigative sites in India from 14 January 2022 to 10 February 2023.
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| ID | Title | Description |
|---|---|---|
| FG000 | Hemophilia A Group | Participants with hemophilia A were treated with ADVATE according to a regimen determined by the treating physician at the study site and in accordance with the national product label under standard clinical practice for 6.7 months. |
| Title | Milestones | Reasons Not Completed | ||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
|
Effectiveness Full Analysis Set (EFAS) comprised of all participants for whom all inclusion and none of the exclusion criteria were met.
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| ID | Title | Description |
|---|---|---|
| BG000 | Hemophilia A Group | Participants with hemophilia A were treated with ADVATE according to a regimen determined by the treating physician at the study site and in accordance with the national product label under standard clinical practice for 6.7 months. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean |
| 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 | |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Number of Participants With Serious Adverse Events (SAE) at Least Possibly Related to ADVATE | An adverse event (AE) was defined as any untoward medical occurrence in a participant administered a pharmaceutical product and that does not necessarily have a causal relationship with this investigational product or medicinal product. An SAE was defined as any untoward medical occurrence that resulted in death; was life-threatening; required inpatient hospitalization or prolongation of present hospitalization; resulted in persistent or significant disability/incapacity; was a congenital anomaly/birth defect or was a medically important event. Number of participants with SAEs (including FVIII inhibitor formation) that were at least possibly related to ADVATE were reported. | Safety Analysis Set (SAS) included all participants who received ADVATE at any time during the study. | Posted | Count of Participants | Participants | Baseline (Day 0) up to end of study (up to 12.9 months) |
|
Baseline (Day 0) up to end of study (up to 12.9 months)
SAS included all participants who received ADVATE at any time during the study.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Hemophilia A Group | Participants with hemophilia A were treated with ADVATE according to a regimen determined by the treating physician at the study site and in accordance with the national product label under standard clinical practice for 6.7 months. |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Pyrexia | General disorders | MedDRA25.0 | Systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Study Director | Takeda | +1-877-825-3327 | TrialDisclosures@takeda.com |
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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 | Dec 2, 2022 | Aug 7, 2023 | Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | May 9, 2023 | Aug 7, 2023 | SAP_001.pdf |
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| ID | Term |
|---|---|
| D006467 | Hemophilia A |
| ID | Term |
|---|---|
| D025861 | Blood Coagulation Disorders, Inherited |
| D001778 | Blood Coagulation Disorders |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
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| ID | Term |
|---|---|
| D005169 | Factor VIII |
| ID | Term |
|---|---|
| D001779 | Blood Coagulation Factors |
| D001798 | Blood Proteins |
| D011506 | Proteins |
| D000602 | Amino Acids, Peptides, and Proteins |
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| Baseline (Day 0) up to end of study (up to 12.9 months) |
| Number of Participants With Clinically Significant Changes in Clinical Laboratory Parameters | Clinical laboratory parameters included hematology, clinical chemistry, viral serology, factor VIII (FVIII) antigen, FVIII activity, incremental recovery, and FVIII inhibitor. Clinical significance was judged as per Investigator's assessment. | Baseline (Day 0) up to end of study (up to 12.9 months) |
| Total Annualized Bleeding Rate (ABR) With Prophylactic Treatment of ADVATE | ABR was defined as number of bleeding episodes during the study period divided by total number of study period days multiplied by 365.25. The mean ABR and standard error was estimated using a generalized linear model (GLM). The total ABR is reported in this outcome measure. | Baseline (Day 0) up to end of study (up to 12.9 months) |
| ABR With Prophylactic Treatment of ADVATE Categorized Based on Location of Bleed | ABR was defined as number of bleeding episodes during the study period divided by total number of study period days multiplied by 365.25. The mean ABR and standard error were estimated using a GLM. The ABR by bleed sites (example, joint, soft tissue, muscle, other [mouth, gums or nose] are reported in this outcome measure. | Baseline (Day 0) up to end of study (up to 12.9 months) |
| ABR With Prophylactic Treatment of ADVATE Categorized Based on Type of Bleed | ABR was defined as number of bleeding episodes during the study period divided by total number of study period days multiplied by 365.25. The mean ABR and standard error were estimated using a GLM. The ABR by bleed cause (example, spontaneous, injury, and unknown) are reported in this outcome measure. | Baseline (Day 0) up to end of study (up to 12.9 months) |
| Total Number of ADVATE Infusions Required During Prophylactic Treatment | Baseline (Day 0) up to end of study (up to 12.9 months) |
| Average Number of ADVATE Infusions Required Per Week During Prophylactic Treatment | Baseline (Day 0) up to end of study (up to 12.9 months) |
| Average Number of ADVATE Infusions Required Per Month During Prophylactic Treatment of Bleeding Episode | Baseline (Day 0) up to end of study (up to 12.9 months) |
| Total Body Mass Adjusted Consumption of ADVATE During Prophylactic Treatment | Body mass adjusted consumption international units per kilograms (IU/kg) was derived as the total units infused (IU) divided by the last available body weight (kg) prior to the infusion. | Baseline (Day 0) up to end of study (up to 12.9 months) |
| Average Body Mass Adjusted Consumption of ADVATE Per Week During Prophylactic Treatment | Body mass adjusted consumption (IU/kg) was derived as the total units infused (IU) divided by the last available body weight (kg) prior to the infusion. | Baseline (Day 0) up to end of study (up to 12.9 months) |
| Average Body Mass Adjusted Consumption of ADVATE Per Month During Prophylactic Treatment | Body mass adjusted consumption (IU/kg) was derived as the total units infused (IU) divided by the last available body weight (kg) prior to the infusion. | Baseline (Day 0) up to end of study (up to 12.9 months) |
| Overall Hemostatic Efficacy Rating of ADVATE for Treatment of Bleeding Episodes | Overall hemostatic efficacy for treatment of bleeding episodes was rated on 4-point Likert scale as: excellent=full relief of pain and cessation of objective signs of bleeding after a single infusion, no additional infusion is required for the control of bleeding and administration of further infusion to maintain hemostasis would not affect the scoring; good=definite pain relief and/or improvement in signs of bleeding after a single infusion, possibly requires more than 2 infusions for complete resolution and administration of further infusion to maintain hemostasis would not affect the scoring; moderate=probable and/or slight relief of pain and slight improvement in signs of bleeding after a single infusion, required multiple infusions for complete resolution; none=no improvement of signs or symptoms or conditions worsen. | Baseline (Day 0) up to end of study (up to 12.9 months) |
| Number of ADVATE Infusions Required to Achieve Resolution of Bleeding Episodes | Baseline (Day 0) up to end of study (up to 12.9 months) |
| Total Body Mass Adjusted Consumption of ADVATE Per Bleeding Episode | Body mass adjusted consumption (IU/kg) was derived as the total units infused (IU) divided by the last available body weight (kg) prior to the infusion. | Baseline (Day 0) up to end of study (up to 12.9 months) |
| Bengaluru |
| 560034 |
| India |
| K J Somaiya Hospital & Research Centre | Mumbai | 400022 | India |
| All India Institute of Medical Sciences (AIIMS) | New Delhi | 110029 | India |
| Unique Children's Hospital Pvt. Ltd. | Pune | 411019 | India |
| years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
|
| Race (NIH/OMB) | Count of Participants | Participants |
|
| Region of Enrollment | Count of Participants | Participants |
|
| Weight | Mean | Standard Deviation | kilograms (kg) |
|
| Height | Mean | Standard Deviation | centimeters (cm) |
|
| Body Mass Index (BMI) | BMI = Weight (kg)/Height (m)^2 | Mean | Standard Deviation | kilograms per meter square (kg/m^2) |
|
| Hemophilia A Group |
Participants with hemophilia A were treated with ADVATE according to a regimen determined by the treating physician at the study site and in accordance with the national product label under standard clinical practice for 6.7 months. |
|
|
| Secondary | Number of Participants With Non-serious Adverse Events (AEs) at Least Possibly Related to ADVATE | An AE was any untoward medical occurrence in a participant administered a pharmaceutical product and that does not necessarily have a causal relationship with this investigational product or medicinal product. Number of participants with non-serious AEs that were at least possibly related to ADVATE were reported. | SAS included all participants who received ADVATE at any time during the study. | Posted | Count of Participants | Participants | Baseline (Day 0) up to end of study (up to 12.9 months) |
|
|
|
| Secondary | Number of Participants With Clinically Significant Changes in Clinical Laboratory Parameters | Clinical laboratory parameters included hematology, clinical chemistry, viral serology, factor VIII (FVIII) antigen, FVIII activity, incremental recovery, and FVIII inhibitor. Clinical significance was judged as per Investigator's assessment. | SAS included all participants who received ADVATE at any time during the study. | Posted | Count of Participants | Participants | Baseline (Day 0) up to end of study (up to 12.9 months) |
|
|
|
| Secondary | Total Annualized Bleeding Rate (ABR) With Prophylactic Treatment of ADVATE | ABR was defined as number of bleeding episodes during the study period divided by total number of study period days multiplied by 365.25. The mean ABR and standard error was estimated using a generalized linear model (GLM). The total ABR is reported in this outcome measure. | EFAS comprised of all participants for whom all inclusion and none of the exclusion criteria were met. | Posted | Mean | Standard Error | bleeds per year | Baseline (Day 0) up to end of study (up to 12.9 months) |
|
|
|
| Secondary | ABR With Prophylactic Treatment of ADVATE Categorized Based on Location of Bleed | ABR was defined as number of bleeding episodes during the study period divided by total number of study period days multiplied by 365.25. The mean ABR and standard error were estimated using a GLM. The ABR by bleed sites (example, joint, soft tissue, muscle, other [mouth, gums or nose] are reported in this outcome measure. | EFAS comprised of all participants for whom all inclusion and none of the exclusion criteria were met. | Posted | Mean | Standard Error | bleeds per year | Baseline (Day 0) up to end of study (up to 12.9 months) |
|
|
|
| Secondary | ABR With Prophylactic Treatment of ADVATE Categorized Based on Type of Bleed | ABR was defined as number of bleeding episodes during the study period divided by total number of study period days multiplied by 365.25. The mean ABR and standard error were estimated using a GLM. The ABR by bleed cause (example, spontaneous, injury, and unknown) are reported in this outcome measure. | EFAS comprised of all participants for whom all inclusion and none of the exclusion criteria were met. | Posted | Mean | Standard Error | bleeds per year | Baseline (Day 0) up to end of study (up to 12.9 months) |
|
|
|
| Secondary | Total Number of ADVATE Infusions Required During Prophylactic Treatment | EFAS comprised of all participants for whom all inclusion and none of the exclusion criteria were met. | Posted | Mean | Standard Deviation | infusions | Baseline (Day 0) up to end of study (up to 12.9 months) |
|
|
|
| Secondary | Average Number of ADVATE Infusions Required Per Week During Prophylactic Treatment | EFAS comprised of all participants for whom all inclusion and none of the exclusion criteria were met. | Posted | Mean | Standard Deviation | infusions per week | Baseline (Day 0) up to end of study (up to 12.9 months) |
|
|
|
| Secondary | Average Number of ADVATE Infusions Required Per Month During Prophylactic Treatment of Bleeding Episode | EFAS comprised of all participants for whom all inclusion and none of the exclusion criteria were met. | Posted | Mean | Standard Deviation | infusions per month | Baseline (Day 0) up to end of study (up to 12.9 months) |
|
|
|
| Secondary | Total Body Mass Adjusted Consumption of ADVATE During Prophylactic Treatment | Body mass adjusted consumption international units per kilograms (IU/kg) was derived as the total units infused (IU) divided by the last available body weight (kg) prior to the infusion. | EFAS comprised of all participants for whom all inclusion and none of the exclusion criteria were met. | Posted | Mean | Standard Deviation | IU/kg | Baseline (Day 0) up to end of study (up to 12.9 months) |
|
|
|
| Secondary | Average Body Mass Adjusted Consumption of ADVATE Per Week During Prophylactic Treatment | Body mass adjusted consumption (IU/kg) was derived as the total units infused (IU) divided by the last available body weight (kg) prior to the infusion. | EFAS comprised of all participants for whom all inclusion and none of the exclusion criteria were met. | Posted | Mean | Standard Deviation | IU/kg per week | Baseline (Day 0) up to end of study (up to 12.9 months) |
|
|
|
| Secondary | Average Body Mass Adjusted Consumption of ADVATE Per Month During Prophylactic Treatment | Body mass adjusted consumption (IU/kg) was derived as the total units infused (IU) divided by the last available body weight (kg) prior to the infusion. | EFAS comprised of all participants for whom all inclusion and none of the exclusion criteria were met. | Posted | Mean | Standard Deviation | IU/kg per month | Baseline (Day 0) up to end of study (up to 12.9 months) |
|
|
|
| Secondary | Overall Hemostatic Efficacy Rating of ADVATE for Treatment of Bleeding Episodes | Overall hemostatic efficacy for treatment of bleeding episodes was rated on 4-point Likert scale as: excellent=full relief of pain and cessation of objective signs of bleeding after a single infusion, no additional infusion is required for the control of bleeding and administration of further infusion to maintain hemostasis would not affect the scoring; good=definite pain relief and/or improvement in signs of bleeding after a single infusion, possibly requires more than 2 infusions for complete resolution and administration of further infusion to maintain hemostasis would not affect the scoring; moderate=probable and/or slight relief of pain and slight improvement in signs of bleeding after a single infusion, required multiple infusions for complete resolution; none=no improvement of signs or symptoms or conditions worsen. | EFAS comprised of all participants for whom all inclusion and none of the exclusion criteria were met. Overall number of participants analyzed are the number of participants with bleeds who required ADVATE infusion for management of the bleeding episode. | Posted | Number | bleeding episodes | Baseline (Day 0) up to end of study (up to 12.9 months) | Treated Bleeds | Treated Bleeds |
|
|
|
| Secondary | Number of ADVATE Infusions Required to Achieve Resolution of Bleeding Episodes | EFAS comprised of all participants for whom all inclusion and none of the exclusion criteria were met. Overall number of participants analyzed are the number of participants with bleeds who required ADVATE infusion for management of the bleeding episode. | Posted | Mean | Standard Deviation | infusions | Baseline (Day 0) up to end of study (up to 12.9 months) |
|
|
|
| Secondary | Total Body Mass Adjusted Consumption of ADVATE Per Bleeding Episode | Body mass adjusted consumption (IU/kg) was derived as the total units infused (IU) divided by the last available body weight (kg) prior to the infusion. | EFAS comprised of all participants for whom all inclusion and none of the exclusion criteria were met. Overall number of participants analyzed are the number of participants with bleeds who required ADVATE infusion for management of the bleeding episode. | Posted | Mean | Standard Deviation | IU/kg | Baseline (Day 0) up to end of study (up to 12.9 months) |
|
|
|
| 0 |
| 50 |
| 0 |
| 50 |
| 6 |
| 50 |
| Varicella | Infections and infestations | MedDRA25.0 | Systematic Assessment |
|
| Animal bite | Injury, poisoning and procedural complications | MedDRA25.0 | Systematic Assessment |
|
| Joint injury | Injury, poisoning and procedural complications | MedDRA25.0 | Systematic Assessment |
|
| Skin laceration | Injury, poisoning and procedural complications | MedDRA25.0 | Systematic Assessment |
|
The first study related publication will be a multi-center publication submitted within 24 months after conclusion or termination of a study at all sites. After such multi site publication, all proposed site publications and presentations will be submitted to sponsor for review 60 days in advance of publication. Site will remove Sponsor confidential information unrelated to study results. Sponsor can delay a proposed publication for another 60 days to preserve intellectual property.
| D020147 | Coagulation Protein Disorders |
| D006474 | Hemorrhagic Disorders |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D011498 |
| Protein Precursors |
| D001685 | Biological Factors |
| Title | Measurements |
|---|---|
|
| Bleed Location: Other (Mouth, Gums or Nose) |
|
| Title | Measurements |
|---|---|
|
| Title | Measurements |
|---|
|
| None |
|