Gene therapy for beta thalassemia
Part of Blood & lymphatic, Genetic & congenital clinical trials.
This trial tests a new gene therapy for beta thalassemia. A modified virus is used to deliver a working gene to your blood stem cells, which may help your body produce normal hemoglobin and reduce the need for blood transfusions.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must have a confirmed diagnosis of beta thalassemia, a blood disorder that causes low hemoglobin.
- You must be at least 4 years old.
- Your overall health must be fairly good (able to do most daily activities) – this is measured by a score of 80% or more on a standard scale.
- Your heart and lungs should be working well – specifically, your heart's pumping ability should be above 50%, and your lung function should be at least 60% of normal.
- Your kidney function should be within normal limits (blood creatinine level no more than twice the upper limit).
- You should not have iron overload in your heart or liver, and no severe liver scarring (cirrhosis).
- You must not have had a bone marrow transplant before, and you must not have a family member who is a perfect tissue match (HLA-identical) and available to donate marrow.
- You must not have a history of cancer (except for certain skin or cervical conditions), epilepsy, or active infections like HIV, syphilis, or 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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