Gene Therapy for Hemophilia A
(GENEr8-INH Trial)
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial is testing a gene therapy called valoctocogene roxaparvovec in patients with severe haemophilia A who have developed antibodies against standard treatments. The therapy aims to provide a working version of the gene needed for proper blood clotting.
Will I have to stop taking my current medications?
The trial does not specify if you need to stop taking your current medications. However, if you are currently undergoing immune tolerance induction therapy or prophylaxis with FVIII, you cannot participate in Part A of the study.
Is Valoctocogene Roxaparvovec safe for humans?
Valoctocogene Roxaparvovec, a gene therapy for severe hemophilia A, has been studied for safety in clinical trials. It has received conditional approval in the EU, indicating that it has been deemed safe enough for use in adults with specific conditions, although ongoing monitoring and further studies are likely required.12345
How does the treatment Valoctocogene Roxaparvovec for Hemophilia A differ from other treatments?
Valoctocogene Roxaparvovec is unique because it is a gene therapy designed to provide a long-term solution by delivering a functional copy of the gene responsible for producing factor VIII, a protein essential for blood clotting, potentially reducing or eliminating the need for regular factor VIII infusions.678910
What data supports the effectiveness of the treatment Valoctocogene Roxaparvovec for Hemophilia A?
Valoctocogene Roxaparvovec has shown sustained clinical benefits in people with severe Hemophilia A, with significant increases in factor VIII levels and a reduction in bleeding episodes, as demonstrated in phase III clinical trials. It has been approved by the European Commission and FDA, indicating its effectiveness and safety for treating Hemophilia A.111121314
Who Is on the Research Team?
Medical Monitor, MD
Principal Investigator
BioMarin Pharmaceutical
Are You a Good Fit for This Trial?
This trial is for adult males with severe Hemophilia A who have had inhibitors to Factor VIII. They must not drink alcohol for a year post-treatment, use effective contraception, and cannot have other bleeding disorders or significant organ dysfunction. Part A includes those currently with inhibitors; Part B is for those with a past history of inhibitors.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Single administration of valoctocogene roxaparvovec at a dose of 6E13 vg/kg
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- Valoctocogene Roxaparvovec
Find a Clinic Near You
Who Is Running the Clinical Trial?
BioMarin Pharmaceutical
Lead Sponsor
Alexander Hardy
BioMarin Pharmaceutical
Chief Executive Officer since 2023
MBA from INSEAD
Greg Friberg
BioMarin Pharmaceutical
Chief Medical Officer
MD from New York Medical College