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

NCT04370054

Active Not RecruitingPhase 3

Sponsor: Pfizer

Conditions: Hemophilia A

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

Countries: United States, Australia, Brazil, Canada, France, Germany, Greece, Italy

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 overview

Sex: MALE

Age: 18 Years to 64 Years

Healthy volunteers: No

Study type: INTERVENTIONAL

Eligibility criteria
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
Locations (45)
  • Mountain View, California, United States
  • Oakland, California, United States
  • Palo Alto, California, United States
  • Palo Alto, California, United States
  • Redwood City, California, United States
  • Redwood City, California, United States
  • San Francisco, California, United States
  • San Francisco, California, United States
  • San Francisco, California, United States
  • San Francisco, California, United States
  • San Francisco, California, United States
  • San Francisco, California, United States
  • San Francisco, California, United States
  • San Francisco, California, United States
  • San Jose, California, United States
  • Stanford, California, United States
  • Stanford, California, United States
  • Seattle, Washington, United States
  • Seattle, Washington, United States
  • Melbourne, Victoria, Australia
  • Campina, Brazil
  • Hamilton, Ontario, Canada
  • Hamilton, Ontario, Canada
  • Hamilton, Ontario, Canada
  • Paris, France
  • Berlin, Germany
  • Frankfurt am Main, Germany
  • Athens, Attikí, Greece
  • Athens, Greece
  • Roma, RM, Italy
  • + 15 more on CT.gov