Gene therapy for beta thalassemia using a modified virus
Part of Blood & lymphatic, Genetic & congenital clinical trials.
This trial tests a new gene therapy that uses a modified virus to add a working copy of the gene for beta globin, which may help your body make healthy red blood cells. It is designed for people with beta thalassemia who have not had a bone marrow transplant and do not have a matching sibling donor.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You have been diagnosed with beta thalassemia.
- You are at least 4 years old.
- You are in generally good health (able to do most daily activities).
- Your heart and lungs are functioning well (heart pump strength >50%, good breathing capacity).
- You do not have severe iron buildup in your heart or liver (based on MRI).
- You have not had a bone marrow transplant, and you do not have a sibling with a perfect tissue match.
- You are not pregnant or breastfeeding, and you have no active infections like HIV, syphilis, or active tuberculosis.
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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