Factor IX Gene Therapy for Hemophilia B
(BENEGENE-2 Trial)
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial tests a new gene therapy treatment for adult men with severe hemophilia B. The treatment aims to help their bodies produce a crucial blood-clotting protein. Participants will receive one dose and be monitored over several years to see if it reduces their bleeding episodes. Gene therapy for hemophilia B has shown promising results in recent studies, suggesting potential long-term therapeutic production of the coagulant protein.
Will I have to stop taking my current medications?
The trial requires participants to stop their current Factor IX prophylaxis therapy after receiving the study drug. The protocol does not specify about other medications, but certain therapies like blood products, aspirin, ibuprofen, and other investigational therapies are restricted.
Is Factor IX gene therapy for hemophilia B safe for humans?
Factor IX gene therapy for hemophilia B has shown a positive safety profile in several studies. In trials with humans and animals, there were no significant side effects, no evidence of toxicity, and no immune responses against the treatment. Some participants experienced mild, temporary liver enzyme changes, which were managed with medication.12345
How is the treatment PF-06838435 for hemophilia B different from other treatments?
What data supports the effectiveness of the treatment PF-06838435 for hemophilia B?
Research shows that gene therapy using modified versions of factor IX can significantly increase its activity, leading to better blood clotting in hemophilia B. Studies in animals and humans have demonstrated that these gene therapies can produce stable and long-term expression of factor IX, improving clotting times without significant side effects.1681112
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 B (Factor IX activity <=2%) who have completed at least 6 months of Factor IX prophylaxis therapy. Participants must not have antibodies against the gene therapy vector, no history of Factor IX inhibitors, and agree to contraception post-treatment.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Pre-infusion
Participants receive at least 6 months of routine Factor IX prophylaxis therapy
Treatment
Participants receive a single intravenous dose of the gene therapy drug PF-06838435
Post-infusion Monitoring
Participants are monitored for safety and effectiveness, with assessments from Week 12 to Month 15
Long-term Follow-up
Participants are monitored for long-term safety and efficacy outcomes
What Are the Treatments Tested in This Trial?
Interventions
- PF-06838435
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