Gene therapy for Fanconi anemia type A
Part of Blood & lymphatic, Genetic & congenital, Hormones & metabolism clinical trials.
This trial tests a new gene therapy for people with Fanconi anemia type A. A modified virus carries a working copy of the FANCA gene into your blood stem cells, which may help your bone marrow produce healthy blood cells.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must have Fanconi anemia type A, confirmed by genetic testing.
- You must be at least 4 years old.
- Your blood counts (ANC, platelets, hemoglobin) must be above certain safe levels.
- Your kidney and liver function must be within a safe range.
- You must not have any active cancer, leukemia, or a serious active infection.
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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