NCT04370054|Active Not RecruitingPhase 3ResultsDMCFDA Drug

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

AFFINE

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%)

Pfizer ClinicalTrials.gov

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2 other identifiers

C37310032024-512075-12-00

Study Type

interventional

Target

Locations

16 countries

Sites

Timeline

RegisteredApr 2020

StartedAug 2020

CompletionOct 2026

Brief 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.

Trial Health

On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment

participants targeted

Target at below P25 for phase_3

Timeline

5mo left

Started Aug 2020

Longer than P75 for phase_3

Geographic Reach

16 countries

45 active sites

Status

active not recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

6.2 years study duration

Study Progress93%

Aug 2020Oct 2026

First Submitted

Initial submission to the registry

April 21, 2020

Completed

9 days until next milestone

First Posted

Study publicly available on registry

April 30, 2020

Completed

4 months until next milestone

Study Start

First participant enrolled

August 18, 2020

Completed

3.8 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

June 17, 2024

Completed

1.2 years until next milestone

Results Posted

Study results publicly available

August 17, 2025

Completed

1.2 years until next milestone

Study Completion

Last participant's last visit for all outcomes

October 25, 2026

Expected

Last Updated

May 18, 2026

Status Verified

May 1, 2026

Enrollment Period

3.8 years

First QC Date

April 21, 2020

Results QC Date

June 9, 2025

Last Update Submit

May 4, 2026

Conditions

Hemophilia A

Keywords

PF-07055480SB-525HemophiliaFactor VIIIFVIIIGene TherapyAAVAAV6Annualized bleeding rateABRgiroctocogene fitelparvovecAFFINE

Outcome Measures

Primary Outcomes (1)

Total Annualized Bleeding Rate (ABR)
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: \[(Number of total bleeding episodes during post-infusion period)\*365.25\] / \[(last date of post-infusion period - date of PF-07055480 infusion) - 77 days + 1\].
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)

Secondary Outcomes (42)

Percentage of Participants With Coagulation FVIII Activity Levels Greater Than (>)5 Percent (%) at 15 Months
At 15 months following infusion of PF-07055480
Treated 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)
Annualized Infusion Rate (AIR) of Exogenous FVIII Activity
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)
FVIII Activity Levels From Week 12 Through 15 Months Following PF-07055480 Infusion
Week 12 through Month 15 following PF-07055480 Infusion
Annualized FVIII Consumption
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)
+37 more secondary outcomes

Study Arms (1)

PF-07055480 (giroctocogene fitelparvovec)

EXPERIMENTAL

Single administration of PF-07055480

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

Interventions

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

Single IV infusion

Also known as: Gene Therapy

PF-07055480 (giroctocogene fitelparvovec)

Eligibility Criteria

Age18 Years - 64 Years

Sexmale(Gender-based eligibility)

Healthy VolunteersNo

Age GroupsAdult (18-64)

You may qualify if:

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

You may not qualify if:

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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Pfizerlead

Study Sites (45)

NOW Physical Therapy

Mountain View, California, 94040, United States

Location

Regents of The University of California

Oakland, California, 94607, United States

Location

Clinical and Translational Research Unit (CTRU)

Palo Alto, California, 94304, United States

Location

Lucile Packard Childrens Hospital

Palo Alto, California, 94304, United States

Location

Imaging Clinic at Stanford Medicine Outpatient Center in Redwood City

Redwood City, California, 94063, United States

Location

The Board of Trustees of the Leland Stanford Junior University

Redwood City, California, 94063, United States

Location

UCSF Outpatient Radiology.

San Francisco, California, 94115, United States

Location

UCSF IDS Pharmacy