NCT04370054

Study to Evaluate the Efficacy and Safety of PF-07055480 / Giroctocogene Fitelparvovec Gene Therapy in Moderately Severe to Severe Hemophilia A Adults

Phase 3, Open-Label, Single-Arm Study to Evaluate the Efficacy and Safety of PF-07055480 (Recombinant AAV2/6 Human Factor VIII Gene Therapy) in Adult Male Participants With Moderately Severe to Severe Hemophilia A(FVIII:C≤1%)

• Status: Active Not Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 77 (actual)
• Sponsor: Pfizer · Industry
• Sex: Male
• Age: 18 Years – 64 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Hemophilia A

Interventions

Type	Name	Description
BIOLOGICAL	PF-07055480 (giroctocogene fitelparovec): Recombinant AAV2/6 Human Factor VIII Gene Therapy	Single IV infusion

Timeline

• Start date: 2020-08-18
• Primary completion: 2024-06-17
• Completion: 2028-10-25
• First posted: 2020-04-30
• Last updated: 2025-08-17
• Results posted: 2025-08-17

Locations

36 sites across 16 countries: United States, Australia, Brazil, Canada, France, Germany, Greece, Italy, Japan, Saudi Arabia, South Korea, Spain, Sweden, Taiwan, Turkey (Türkiye), United Kingdom

Regulatory

• FDA-regulated drug study