Gene therapy for teenagers with sickle cell disease
Part of Blood & lymphatic, Genetic & congenital clinical trials.
This study tests an experimental gene therapy called CS-206 for teenagers with sickle cell disease. It aims to help reduce severe complications by modifying the patient's own blood stem cells.
Summary written for real people, not researchers, by Clin2.
Who can take part
- Age between 12 and 18 years (inclusive)
- Confirmed sickle cell disease (HbSS, HbSβ0, or HbSβ+)
- At least 2 serious sickle cell complications (like severe pain, acute chest syndrome, priapism, or stroke) in the last 2 years, OR need for ongoing blood transfusions due to antibody problems, OR failed/refused standard treatment
- Adequate liver, kidney, and overall health (functional status score ≥70)
- Not pregnant, not breastfeeding, and willing to use contraception during the study
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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