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)
Development Status	Active
Indication	Hemophilia A
Disease Ontology Term	DOID:12134
Compound Name	Giroctogogene fitelparvovec
Sponsor	Pfizer
Funder Type	Industry
Recruitment Status	Active not recruiting
Enrollment Count	76
Results Posted	Not Available

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	none
Dose 1	3E13 vg/kg
Dose 2
Dose 3
Dose 4
Dose 5

No.of Trial Sites	37
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
FDA Designations
Recent Updates	Pfizer terminated this program due to marketing considerations, Development rights are being returned to Sangamo