One-time gene therapy for severe sickle cell and transfusion anemia
Part of Blood & lymphatic, Genetic & congenital clinical trials.
This trial tests a single dose of gene therapy (CTX001) to improve outcomes for people with severe sickle cell disease or transfusion-dependent beta-thalassemia. It aims to see how well it works and whether it is safe, especially after a planned stem cell transplant process.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You have either transfusion-dependent beta-thalassemia or severe sickle cell disease
- You have a history of needing frequent red blood transfusions (for beta-thalassemia)
- For sickle cell disease, you’ve had frequent severe pain crises (VOC) in the past two years
- Your medical team believes you’re eligible for an autologous stem cell transplant (using your own stem cells)
- You do not have a fully matched sibling or relative donor available (as decided by your team)
- You have not had a prior stem cell transplant, and you don’t have a serious active infection or certain specific genetic/injury conditions
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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