Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Recombinant Antihemophilic Factor Manufactured and Formulated without Added Human or Animal Proteins · 1 trial · 1 indication
Percentage of treated participants who developed factor VIII inhibitors
| Arm | Type | Description |
|---|---|---|
| Single Arm - All Participants | EXPERIMENTAL | All subjects enrolled in the study who meet the eligibility criteria. |
| Name | Type | Description |
|---|---|---|
| Recombinant Antihemophilic Factor Manufactured and Formulated without Added Human or Animal Proteins (rAHF-PFM) | BIOLOGICAL | Treatment regimens were determined by the investigator, and may have been any combination of standard prophylaxis (25 to 50 IU/kg body weight, 3 to 4 times per week), investigator-determined prophylaxis, and/or on-demand treatment (dose selected by investigator). The treatment of bleeding episodes and perioperative management was at the discretion of the investigator and consistent with the institution's standard of care. For incremental recovery assessments, a single infusion at 50 +/- 5 IU/kg was to be given. Immune tolerance induction (ITI) therapy for subjects who developed factor VIII inhibitors was at the discretion of the investigator, based on the institution's guidelines or described in peer-reviewed literature, and was to be approved by the sponsor's medical director. rAHF-PFM was to be administered intravenously via bolus infusion, except for perioperative management when it may have been given either by continuous or bolus infusion. |
Inclusion Criteria: * The subject has severe or moderately severe hemophilia A as defined by a baseline factor VIII level \<= 2% of normal, as documented at screening * The subject is \< 6 years of age * The subject's legally authorized representative has provided written informed consent Exclusio...