Gene editing for sickle cell disease
Part of Blood & lymphatic, Genetic & congenital clinical trials.
This trial tests a gene editing treatment for sickle cell disease. It aims to modify your own blood stem cells to produce more fetal hemoglobin, which can reduce pain episodes and other complications.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You have a severe type of sickle cell disease (like HbSS, HbS/B0 thalassemia, HbSD, or HbSO).
- You are between 13 and 40 years old.
- You have had at least 2 episodes of acute chest syndrome or 3 severe pain crises in the last 2 years.
- You have tried hydroxyurea but it did not work well or caused side effects, or you cannot take it for other reasons.
- You have an available related bone marrow donor that is not a good match.
- You are willing to come for follow-up visits for 15 years after treatment.
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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