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NCT04370054

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%)

Study 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.

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Interventions

PF-07055480 (giroctocogene fitelparovec): Recombinant AAV2/6 Human Factor VIII Gene TherapyBIOLOGICAL
Single IV infusion

Study Locations

FacilityCityStateCountry
NOW Physical TherapyMountain ViewCaliforniaUnited States
Clinical and Translational Research Unit (CTRU)Palo AltoCaliforniaUnited States
Lucile Packard Childrens HospitalPalo AltoCaliforniaUnited States
Imaging Clinic at Stanford Medicine Outpatient Center in Redwood CityRedwood CityCaliforniaUnited States
UCSF IDS PharmacySan FranciscoCaliforniaUnited States
University of California, San Francisco - Clinical Research CenterSan FranciscoCaliforniaUnited States
University of California, San Francisco - Moffitt/Long Inpatient HematologySan FranciscoCaliforniaUnited States
University of California, San Francisco - Outpatient Hematology ClinicSan FranciscoCaliforniaUnited States
Stanford Health CareStanfordCaliforniaUnited States
Washington Institute for CoagulationSeattleWashingtonUnited States
University of Washington Medical Center - Translational Research Unit (TRU)SeattleWashingtonUnited States
The Alfred HospitalMelbourneVictoriaAustralia
Centro de Hematologia e Hemoterapia de Campinas - Hemocentro UNICAMPCampinasSão PauloBrazil
McMaster University Medical Centre - Hamilton Health SciencesHamiltonOntarioCanada
Juravinski Hospital - Hamilton Health SciencesHamiltonOntarioCanada
Hopital NeckerParisFrance
Vivantes Klinikum im FriedrichshainBerlinGermany
Klinikum der Johann Wolfgang Goethe-Universitaet, Medizinische Klinik IIFrankfurt am MainGermany
General Hospital of Athens ''Laiko''AthensAttikíGreece
General Hospital of Athens "Hippokration"AthensGreece
Università degli studi di Roma "La Sapienza"- Policlinico Umberto IRomaRMItaly
Azienda Ospedaliero Universitaria Careggi SODc Malattie Emorragiche e della CoagulazioneFlorenceItaly
Dip. di Medicina Clinica e Chirurgia, Università degli Studi di Napoli Federico II - UOC di MedicinaNaplesItaly
Nagoya University Hospital - Transfusion MedicineNagoyaAichi-kenJapan
Saitama Medical University HospitalIruma-gunSaitamaJapan
King Faisal Specialist Hospital & Research CenterRiyadhSaudi Arabia
Kyung Hee University Hospital at GangdongSeoulSouth Korea
Hospital Universitari Vall d'HebronBarcelonaSpain
H.U. Rio HortegaValladolidSpain
Skåne University HospitalMalmoSkåne CountySweden
National Taiwan University HospitalTaipeiTaiwan
Adana Acibadem HospitalAdanaTurkey (Türkiye)
Gaziantep University Sahinbey Research and Training HospitalGaziantepTurkey (Türkiye)
Ege University Medical Faculty, Pediatric HematologyIzmirTurkey (Türkiye)
Ege University Medical FacultyIzmirTurkey (Türkiye)
Guy's and St. Thomas' NHS Foundation TrustLondonUnited Kingdom

Official Trial Information

View on ClinicalTrials.gov

Data sourced from ClinicalTrials.gov. Last updated: April 14, 2026