Study to Evaluate the Efficacy and Safety of PF-07055480 / Giroctocogene Fitelparvovec Gene Therapy in Moderately Severe to Severe Hemophilia A Adults
Summary
C3731003 is a pivotal Phase 3 study to evaluate the clinical efficacy and safety of a single IV infusion of PF-07055480 / giroctocogene fitelparvovec (Recombinant AAV2/6 Human Factor VIII Gene Therapy) in adult male participants with moderately severe or severe hemophilia A (FVIII:C≤1%) for the study duration of 5 years. The study will enroll eligible participants who have been followed on routine prophylaxis with FVIII products in the Lead-In study C0371004.
Timeline
- Start
- 2020-08-18
- Primary completion
- 2024-06-17
- Completion
- 2026-10-25
Outcome
Met primary endpoint
registry analysis (non-inferiority test); FVIII Prophylaxis vs PF-07055480; p =0.0040; Mean Difference (Final Values) -3.49 (95% CI -6.06 to -0.91) NCT04370054 ↗
release “The AFFINE study achieved its primary objective of non-inferiority, as well as superiority, of total annualized bleeding rate (ABR)” pfizer.com ↗
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | giroctocogene fitelparvovec | Gene therapy (AAV / viral vector) | — | Intravenous |