Gene therapy for adults with severe hemophilia A
Part of Blood & lymphatic, Genetic & congenital clinical trials.
This early-phase study tests an FVIII gene therapy to help your body make more of the missing clotting factor in hemophilia A. It’s aimed at adults with severe low FVIII levels who have already used FVIII replacement products for a long time and have not developed an immune blocker (an inhibitor).
Summary written for real people, not researchers, by Clin2.
Who can take part
- You’re a male age 18 or older and can understand the study and sign consent
- You have hemophilia A with very low FVIII levels (2% or less, about 2 IU/dL or less)
- You’ve had at least 150 prior days of FVIII replacement (from recombinant or blood/plasma sources)
- You had bleeding and/or FVIII infusions in the last 12 weeks, documented in your records
- You do not have an FVIII inhibitor (an immune response that blocks FVIII), and you’ve not had signs of reduced response
- You agree to use barrier contraception from consent until 52 weeks after the gene therapy dose
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
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