This study is in progress, not accepting new patients

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

a study on Hemophilia

Summary

Eligibility
for males ages 18-64 (full criteria)
Location
at San Francisco, California and other locations
Dates
study started
study ends around

Description

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.

Official Title

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%)

Keywords

Hemophilia A, PF-07055480, SB-525, Hemophilia, Factor VIII, FVIII, Gene Therapy, AAV, AAV6, Annualized bleeding rate, ABR, giroctocogene fitelparvovec, AFFINE, Genetic Therapy, PF-07055480 (giroctocogene fitelparovec): Recombinant AAV2/6 Human Factor VIII Gene Therapy, PF-07055480 (giroctocogene fitelparvovec)

Eligibility

You can join if…

Open to males ages 18-64

  • 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 CAN'T join 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

Locations

  • UCSF Outpatient Radiology.
    San Francisco California 94115 United States
  • UCSF IDS Pharmacy
    San Francisco California 94143 United States
  • UCSF lnvestigational Drug Service
    San Francisco California 94143 United States
  • UCSF Outpatient Radiology (alternate location)
    San Francisco California 94143 United States
  • UCSF Outpatient Radiology
    San Francisco California 94143 United States
  • UCSF - Clinical Research Center
    San Francisco California 94143 United States
  • UCSF - Moffitt/Long Inpatient Hematology
    San Francisco California 94143 United States
  • UCSF - Outpatient Hematology Clinic
    San Francisco California 94143 United States
  • Regents of The University of California
    Oakland California 94607 United States
  • Imaging Clinic at Stanford Medicine Outpatient Center in Redwood City
    Redwood City California 94063 United States

Details

Status
in progress, not accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Pfizer
ID
NCT04370054
Phase
Phase 3 Hemophilia Research Study
Study Type
Interventional
Participants
About 77 people participating
Last Updated