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 UCSF
Dates
study started
completion 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, 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 IDS Pharmacy
    San Francisco California 94143 United States
  • University of California, San Francisco - Clinical Research Center
    San Francisco California 94143 United States
  • University of California, San Francisco - Moffitt/Long Inpatient Hematology
    San Francisco California 94143 United States
  • University of California, San Francisco - Outpatient Hematology Clinic
    San Francisco California 94143 United States
  • Imaging Clinic at Stanford Medicine Outpatient Center in Redwood City
    Redwood City California 94063 United States
  • Clinical and Translational Research Unit (CTRU)
    Palo Alto California 94304 United States
  • Lucile Packard Childrens Hospital
    Palo Alto California 94304 United States
  • Stanford Health Care
    Stanford California 94305 United States

Details

Status
in progress, not accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Pfizer
Links
To obtain contact information for a study center near you, click here.
ID
NCT04370054
Phase
Phase 3 Hemophilia Research Study
Study Type
Interventional
Participants
About 76 people participating
Last Updated