Gene therapy for transfusion-dependent beta thalassemia
Part of Blood & lymphatic, Genetic & congenital clinical trials.
This trial tests a gene therapy that may help people with beta thalassemia who need regular blood transfusions. It aims to reduce or eliminate the need for transfusions by fixing the blood cell production problem.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You are between 12 and 45 years old.
- You have beta thalassemia major (also called transfusion-dependent thalassemia) and need regular blood transfusions.
- You have had at least 8 blood transfusions per year for the past 2 years.
- Your iron levels are not too high, and your heart and liver are not severely overloaded with iron.
- You are healthy enough to undergo a stem cell transplant (using your own cells).
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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