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NCT04370054 · ClinicalTrials.gov registry record · Phase 3
Study to Evaluate the Efficacy and Safety of PF-07055480 / Giroctocogene Fitelparvovec Gene Therapy in Moderately Severe to Severe Hemophilia A Adults
A Phase 3 study of Hemophilia A, sponsored by Pfizer.
- Active
- Registry status
- Phase 3
- Development phase
- 77
- Enrollment target
- 20
- Study locations
NCT04370054: Active Phase 3 study of Hemophilia A, sponsored by Pfizer.
NCT04370054 is a Phase 3 study of Hemophilia A that is active but no longer recruiting, run by Pfizer. The registered enrollment target is 77 participants, below the 90-participant average among 42 other Hemophilia A trials with a reported enrollment target (14% lower). The trial reports 20 study locations across 7 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT04370054, a Phase 3 study of Hemophilia A, is active but no longer recruiting, sponsored by Pfizer.
- ACTIVE NOT RECRUITING
- Registry status
- Phase 3
- Development phase
- 77 participants
- Enrollment target
- 20
- Study locations
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.
Primary Outcome
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-
Conditions Studied
Interventions
- BIOLOGICAL PF-07055480 (giroctocogene fitelparovec): Recombinant AAV2/6 Human Factor VIII Gene Therapy
Study Locations (20)
California
- NOW Physical Therapy - Mountain View
- Clinical and Translational Research Unit (CTRU) - Palo Alto
- Lucile Packard Childrens Hospital - Palo Alto
- Imaging Clinic at Stanford Medicine Outpatient Center in Redwood City - Redwood City
- UCSF IDS Pharmacy - San Francisco
- University of California, San Francisco - Clinical Research Center - San Francisco
- University of California, San Francisco - Moffitt/Long Inpatient Hematology - San Francisco
- University of California, San Francisco - Outpatient Hematology Clinic - San Francisco
- Stanford Health Care - Stanford
Other
- Hopital Necker - Paris
- Vivantes Klinikum im Friedrichshain - Berlin
- Klinikum der Johann Wolfgang Goethe-Universitaet, Medizinische Klinik II - Frankfurt am Main
- General Hospital of Athens "Hippokration" - Athens
Washington
- Washington Institute for Coagulation - Seattle
- University of Washington Medical Center - Translational Research Unit (TRU) - Seattle
Ontario
- McMaster University Medical Centre - Hamilton Health Sciences - Hamilton
- Juravinski Hospital - Hamilton Health Sciences - Hamilton
Victoria
- The Alfred Hospital - Melbourne
São Paulo
- Centro de Hematologia e Hemoterapia de Campinas - Hemocentro UNICAMP - Campinas
Attikí
- General Hospital of Athens ''Laiko'' - Athens
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 77 participants |
| Start Date | 2020-08-18 |
| Est. Completion | 2028-10-25 |
| Phase | Phase 3 |
What the registry record for NCT04370054 still lists
NCT04370054 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 77 participants, a relatively small participant target, below the 90-participant average among 42 other Hemophilia A trials with a reported enrollment target (14% lower).
The record links to 1 condition, with Hemophilia A appearing as the primary indexed condition, and to 1 intervention - of which PF-07055480 (giroctocogene fitelparovec): Recombinant AAV2/6 Human Factor VIII Gene Therapy is the first listed.
NCT04370054 names 20 study sites across 7 states, led by California, Other, Washington.
Frequently Asked Questions
What is clinical trial NCT04370054 about?
NCT04370054 is a clinical study titled "Study to Evaluate the Efficacy and Safety of PF-07055480 / Giroctocogene Fitelparvovec Gene Therapy in Moderately Severe to Severe Hemophilia A Adults". 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 stud...
What is the current status of trial NCT04370054?
This trial is currently active not recruiting. It is a Phase 3 study. The enrollment target is 77 participants. The study started on 2020-08-18. Estimated completion is 2028-10-25.
What conditions does trial NCT04370054 study?
This clinical trial studies the following conditions: Hemophilia A.
What interventions are being tested in trial NCT04370054?
The interventions under investigation include: PF-07055480 (giroctocogene fitelparovec): Recombinant AAV2/6 Human Factor VIII Gene Therapy (BIOLOGICAL).
Who is sponsoring clinical trial NCT04370054?
This trial is sponsored by Pfizer, which has 1,845 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT04370054 being conducted?
This trial has 20 study locations across California, Washington, Victoria, São Paulo, Ontario. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
Similar trials for Hemophilia A
Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.
Where NCT04370054's enrollment target sits among peer trials
77 18th of 42 higher than 25 of 42 other Hemophilia A trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Hemophilia A trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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