Gene correction using your own stem cells to improve sickle disease
Part of Blood & lymphatic, Genetic & congenital clinical trials.
This trial tests a gene-correction treatment that changes sickle hemoglobin (HbS) into normal hemoglobin (HbA) using your own stem cells. It may help reduce severe sickle cell complications for people with severe disease who meet specific safety and medical criteria.
Summary written for real people, not researchers, by Clin2.
Who can take part
- Age 12 to 40 years
- Have severe sickle cell disease despite standard supportive care (many painful crises and/or lung complications)
- Have a good functional status (Lansky/Karnofsky score 80 or higher)
- No available 10/10 HLA-matched sibling donor
- No prior stem cell transplant or prior gene therapy
- No active serious infection; not pregnant or breastfeeding; and no certain cancer-related or high-risk blood/chromosome problems
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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