Summary

Eligibility
for males ages 18-64 (full criteria)
Location
at San Francisco, California and other locations
Dates
study started
completion around

Description

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.

Official Title

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%)

Keywords

Hemophilia A, PF-07055480, SB-525, Hemophilia, Factor VIII, FVIII, Gene Therapy, AAV, AAV6, Annualized bleeding rate, ABR, giroctocogene fitelparvovec, AFFINE, PF-07055480 (giroctocogene fitelparovec): Recombinant AAV2/6 Human Factor VIII Gene Therapy, PF-07055480 (giroctocogene fitelparvovec)

Eligibility

Locations

Details

Status
in progress, not accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Pfizer
ID
NCT04370054
Phase
Phase 3 Hemophilia Research Study
Study Type
Interventional
Participants
About 76 people participating
Last Updated