Gene therapy for severe sickle cell disease in teens and adults
Part of Blood & lymphatic, Genetic & congenital clinical trials.
This trial tests a new gene therapy for sickle cell disease. It uses your own blood stem cells (collected from bone marrow) that are modified in a lab to help produce healthy red blood cells, then given back to you after a short chemotherapy. The goal is to reduce or stop severe complications like pain crises and lung problems.
Summary written for real people, not researchers, by Clin2.
Who can take part
- Be between 12 and 35 years old
- Have severe sickle cell disease (HbSS type) with complications like frequent pain crises or acute chest syndrome
- Have tried hydroxyurea for at least 3 months without enough benefit
- Be willing to undergo a bone marrow aspiration (myelogram)
- Not have a fully matched sibling donor available
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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