Gene therapy using your own stem cells for severe hemophilia a
Part of Blood & lymphatic, Genetic & congenital clinical trials.
This trial tests a new gene therapy approach: your own blood stem cells are modified to produce factor VIII, which helps blood clot. It is for adults with severe hemophilia A who have had frequent bleeding or joint damage.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must be a male aged 18 or older.
- You must have severe hemophilia A (factor VIII level less than 1%).
- You must have had factor VIII treatment for at least 150 days in your life.
- You must have had frequent bleeding episodes or joint damage due to hemophilia.
- You must be in generally good health, with no major organ problems or bleeding inhibitor history.
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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