Gene editing therapy for beta-thalassemia major
Part of Blood & lymphatic clinical trials.
This trial tests a new gene-editing treatment called CS-101 for people with beta-thalassemia major who need regular blood transfusions. It aims to see if the treatment is safe and can reduce or eliminate the need for transfusions.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must be between 12 and 35 years old.
- You must have beta-thalassemia major that requires regular blood transfusions.
- You must have received at least 8 units of blood transfusions in the past year and had low hemoglobin before transfusions.
- You must be in generally good health (no serious heart, liver, or kidney problems, and no active infections or cancer).
- You cannot have had a previous stem cell transplant, gene therapy, or gene-editing 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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