At a glance
ClinicalIndex Comparison Record- ✓Severe or moderately severe hemophilia A with baseline factor VIII level ≤2% of normal
- ✓Age <6 years
- ✓Previously untreated (except for limited prior ADVATE exposure)
- ✕Prior factor VIII exposure other than ≤3 ADVATE infusions in 28 days; any factor VIII prior to 28-day window excludes participation
- ✕More than 3 rAHF-PFM infusions between screening and initial recovery infusion
- ✕Detectable factor VIII inhibitor (Nijmegen assay) or prior inhibitor history
- ✕History of factor VIII inhibitor at any time
Standardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.
Recombinant Antihemophilic Factor Manufactured and Formulated Without Added Human or Animal Proteins (rAHF-PFM): Evaluation of Immunogenicity, Efficacy, and Safety in Previously Untreated Patients With Hemophilia A
In Brief
A Phase 3 clinical trial evaluating Recombinant Antihemophilic Factor Manufactured and Formulated without Added Human or Animal Proteins (rAHF-PFM) for Hemophilia A. Completed, enrolled 66 participants across 35 sites in 10 countries.
Detailed Summary
The purpose of this study is to evaluate whether Antihemophilic factor, recombinant, manufactured protein-free (rAHF-PFM) is effective and safe in the treatment of hemophilia A patients who have not been treated with factor VIII (FVIII) before.
Study Details
Timeline
Interventions
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.