Gene therapy for hemophilia A using a lentivirus
Part of Blood & lymphatic, Genetic & congenital clinical trials.
This trial tests a one-time gene therapy that uses a modified virus (lentivirus) to deliver a working copy of the factor VIII gene to your cells. The goal is to help your body produce factor VIII on its own, reducing or eliminating the need for factor VIII infusions.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You are male and at least 2 years old.
- You have hemophilia A with very low factor VIII (2% or less of normal).
- You have on average at least 4 bleeding episodes a year, or you take factor VIII regularly to prevent bleeds.
- You have never had a factor VIII inhibitor (an antibody that blocks factor VIII treatment).
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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