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

NCTID	NCT04370054 (View at clinicaltrials.gov)
Description	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. (Show More)
Indication	Hemophilia A
Compound Name	Giroctogogene fitelparvovec
Sponsor	Pfizer
Funder Type	Industry
Status	Active not recruiting
Enrollment Count	76

Target Gene/Variant	F8
Therapy Type	Gene transfer
Therapy Route	In-vivo
Mechanism of Action	Functional gene replacement
Route of Administration	Intravenous
Drug Product Type	Viral vector
Target Tissue/Cell	Liver
Delivery System	Viral transduction
Vector Type	AAV2/6
Editor Type
Dose 1	3 × 10^13 vg/kg
Dose 2
Dose 3
Dose 4
Dose 5

No.of Trial Sites	36
Locations	United States,Japan,United Kingdom,Spain,Greece,Saudi Arabia,Canada,Sweden,Turkey,Taiwan,Brazil,Korea, Republic of,Italy,Australia,France,Germany

Has US IND	True
Recent Updates	BLA and MAA submissions expected early 2025