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

NCT04370054 · tracked via the Priya Life Science South Korea tracker

Sponsor

Pfizer

Phase

Phase 3

Started

2020-08-18

Last updated

2026-08-19

Condition(s) studied

Hemophilia A

Investigational drug(s) / intervention(s)

PF-07055480 (giroctocogene fitelparovec): Recombinant AAV2/6 Human Factor VIII Gene Therapy

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

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.

Eligibility

Sex

MALE

Min age

18 Years

Max age

64 Years

Healthy volunteers

Main inclusion Criteria * Males who have been followed on routine Factor VIII prophylaxis therapy during the lead-in study (C0371004) and have \> = 150 documented exposure days to a Factor VIII protein product * Moderately severe to severe hemophilia A (Factor VIII activity \< =1%) * Suspension of FVIII prophylaxis therapy post study drug infusion Main exclusion Criteria * Anti-AAV6 neutralizing antibodies * History of inhibitor to Factor VIII * Laboratory values at screening visit that are abnormal or outside acceptable study limits * Significant and/or unstable liver disease, biliary disease, significant liver fibrosis * Conditions associated with increased thromboembolic risk such as inherited or acquired thrombophilia, or a history of thrombotic events * Planned surgical procedure requiring Factor VIII surgical prophylactic factor treatment 12 months from screening visit * Active hepatitis B or C * Serological evidence of human immunodeficiency virus HIV-1 or HIV-2 with Cluster of Differentiation 4 positive (CD4+) cell count ≤200 mm3 and/or viral load \>20 copies/mL

Primary outcome measure(s)

Total Annualized Bleeding Rate (ABR) — FVIII Prophylaxis arm: a minimum of 7.4 months, up to maximum of 32.3 months (Pre-infusion period); PF-07055480 arm: Week 12 through at least 15 months of follow-up, maximum follow up was of 44.4 months (Post-infusion period)
Bleeds for evaluation included both treated and untreated bleeds. Treated bleed: bleeding event necessitated administration of coagulation factor within 72 hours of signs or symptoms of bleeding. Untreated bleed: bleeding event did not necessitate administration of coagulation factor within 72 hours of signs and symptoms of bleeding. In this outcome measure total ABR following infusion of PF-07055480 in current study (post-infusion) and on prior FVIII prophylaxis regimen prior to infusion of PF-07055480 (pre-infusion, data collected from lead-in study C0371004) were reported. Total ABR for FVIII prophylaxis= \[(Number of total bleeding episodes during pre-infusion period)\*365.25\]/ \[(Number of days of follow-up in pre-infusion period)\]. Total ABR for PF-07055480: \[(Number of total bleeding episodes during post-infusion period)\*365.25\] / \[(last date of post-infusion period - date of PF-07055480 infusion) - 77 days + 1\].

Trial sites (45)

Facility	City	Region
NOW Physical Therapy	Mountain View	California
Regents of The University of California	Oakland	California
Clinical and Translational Research Unit (CTRU)	Palo Alto	California
Lucile Packard Childrens Hospital	Palo Alto	California
Imaging Clinic at Stanford Medicine Outpatient Center in Redwood City	Redwood City	California
The Board of Trustees of the Leland Stanford Junior University	Redwood City	California
UCSF Outpatient Radiology.	San Francisco	California
UCSF IDS Pharmacy	San Francisco	California
UCSF lnvestigational Drug Service	San Francisco	California
UCSF Outpatient Radiology (alternate location)	San Francisco	California
UCSF Outpatient Radiology	San Francisco	California
University of California, San Francisco - Clinical Research Center	San Francisco	California
University of California, San Francisco - Moffitt/Long Inpatient Hematology	San Francisco	California
University of California, San Francisco - Outpatient Hematology Clinic	San Francisco	California
Access Sourcecorp Deliverex (off-site storage)	San Jose	California
Stanford Health Care (hospital and IDS pharmacy)	Stanford	California
Stanford Health Care	Stanford	California
Washington Institute for Coagulation	Seattle	Washington
University of Washington Medical Center - Translational Research Unit (TRU)	Seattle	Washington
The Alfred Hospital	Melbourne	Victoria
Universidade Estadual de Campina	Campina	Brazil
Hamilton Health Sciences Corporation	Hamilton	Ontario
McMaster University Medical Centre - Hamilton Health Sciences	Hamilton	Ontario
Juravinski Hospital - Hamilton Health Sciences	Hamilton	Ontario
Hopital Necker	Paris	France
Vivantes Klinikum im Friedrichshain	Berlin	Germany
Klinikum der Johann Wolfgang Goethe-Universitaet, Medizinische Klinik II	Frankfurt am Main	Germany
General Hospital of Athens ''Laiko''	Athens	Attikí
General Hospital of Athens "Hippokration"	Athens	Greece
Università degli studi di Roma "La Sapienza"- Policlinico Umberto I	Roma	RM
Azienda Ospedaliero Universitaria Careggi SODc Malattie Emorragiche e della Coagulazione	Florence	Italy
Dip. di Medicina Clinica e Chirurgia, Università degli Studi di Napoli Federico II - UOC di Medicina	Naples	Italy
Nagoya University Hospital - Transfusion Medicine	Nagoya	Aichi-ken
Saitama Medical University Hospital	Iruma-gun	Saitama
King Faisal Specialist Hospital & Research Center	Riyadh	Saudi Arabia
Kyung Hee University Hospital at Gangdong	Seoul	South Korea
Hospital Universitari Vall d'Hebron	Barcelona	Spain
H.U. Rio Hortega	Valladolid	Spain
Skåne University Hospital	Malmö	Skåne County
National Taiwan University Hospital	Taipei	Taiwan

+ 5 more sites — see the full list on the official registry below.

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Other trials for the same condition

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Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT04370054 on ClinicalTrials.gov ↗ ← All trials in South Korea