Gene therapy for transfusion-dependent beta-thalassemia
Part of Blood & lymphatic, Genetic & congenital clinical trials.
This trial tests a gene therapy to help people with transfusion-dependent beta-thalassemia (a severe type of thalassemia) stop needing regular blood transfusions. It uses your own stem cells, which are modified to produce healthy red blood cells.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must be between 12 and 45 years old.
- You have been diagnosed with transfusion-dependent beta-thalassemia (also called beta-thalassemia major).
- In the past 2 years, you needed at least 8 red blood cell transfusions each year.
- Your iron levels are not too high (ferritin under 5000 ng/mL and MRI shows moderate or less iron in heart/liver).
- You have no active serious infections, bleeding problems, or cancer, and you are healthy enough for a stem cell transplant.
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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