Gene editing for sickle cell disease
Part of Blood & lymphatic, Genetic & congenital clinical trials.
This trial tests a new gene therapy that may help people with severe sickle cell disease by editing their own blood stem cells to produce healthy red blood cells. It aims to reduce painful crises and other complications.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must be between 13 and 40 years old.
- You must have a severe form of sickle cell disease (like HbSS, HbS/B0 thalassemia, HbSD, or HbSO).
- You must have had at least 2 episodes of acute chest syndrome or 3 or more severe pain crises in the last 2 years.
- You must have tried hydroxyurea (a standard medicine) and it didn't work well enough or you couldn't tolerate it.
- You must not have a matched bone marrow donor available.
- You must be willing to come back for check-ups 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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