Gene Therapy for Hemophilia A
(AFFINE Trial)
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial tests a single treatment for adult males with severe hemophilia A. The treatment aims to help their bodies produce a necessary protein, reducing the need for regular preventive treatments. Research has been ongoing for a long time to enable the body to produce this protein and reduce bleeding episodes.
Will I have to stop taking my current medications?
Yes, participants will need to stop their routine Factor VIII prophylaxis therapy after receiving the study drug infusion.
Is gene therapy for hemophilia A generally safe for humans?
How is the treatment giroctocogene fitelparovec unique for hemophilia A?
Giroctocogene fitelparovec is a gene therapy that uses an adeno-associated virus (AAV) vector to deliver a functional gene for factor VIII, allowing the body to produce its own factor VIII and potentially reducing the need for regular factor replacement therapy. This approach aims to provide a more sustained and stable increase in factor VIII levels, which could lead to fewer bleeding episodes compared to traditional treatments.14678
What data supports the effectiveness of the treatment PF-07055480 (giroctocogene fitelparovec) for hemophilia A?
Gene therapy for hemophilia, using similar adeno-associated viral (AAV) vectors, has shown promising results in increasing clotting factor levels and reducing bleeding episodes in patients. Other treatments like Roctavian and Hemgenix, which use AAV vectors, have been approved based on their effectiveness in treating hemophilia A and B, suggesting potential for similar therapies like PF-07055480.12489
Who Is on the Research Team?
Pfizer CT.gov Call Center
Principal Investigator
Pfizer
Are You a Good Fit for This Trial?
Adult males with moderately severe to severe hemophilia A (Factor VIII activity ≤1%) who have been on routine Factor VIII prophylaxis and participated in the lead-in study C0371004 with ≥150 exposure days to a Factor VIII product. Excluded are those with anti-AAV6 antibodies, history of Factor VIII inhibitors, abnormal lab values, thrombosis risk, planned surgeries requiring prophylaxis within 12 months, active hepatitis B or C, significant HIV infection or liver disease.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Single IV infusion of PF-07055480 / giroctocogene fitelparvovec gene therapy
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- PF-07055480 (giroctocogene fitelparovec)
Find a Clinic Near You
Who Is Running the Clinical Trial?
Pfizer
Lead Sponsor
Albert Bourla
Pfizer
Chief Executive Officer since 2019
PhD in Biotechnology of Reproduction, Aristotle University of Thessaloniki
Patrizia Cavazzoni
Pfizer
Chief Medical Officer
MD from McGill University