Gene therapy transplant for severe hemophilia A
Part of Blood & lymphatic, Genetic & congenital clinical trials.
This Phase 1 trial tests a gene therapy given with an “own blood stem cell” transplant to improve hemophilia A. It’s mainly to check safety and early signs of how well the therapy works in people with severe bleeding problems.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You can legally give informed consent for the study.
- You must be a man age 18 or older.
- You have severe hemophilia A (very low factor VIII activity).
- You have had lots of bleeding in the past and/or joint damage from bleeding.
- You must be well enough for treatment (Karnofsky score 70 or higher).
- You must be able to follow birth-control rules for 12 weeks after transplant if applicable.
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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