Growth and development after gene therapy for beta-thalassemia in children
Part of Blood & lymphatic clinical trials.
This study looks at how children with transfusion-dependent beta-thalassemia grow and develop after gene therapy. It compares children who have had gene therapy with those who have not, and also with healthy children of the same age and gender.
Summary written for real people, not researchers, by Clin2.
Who can take part
- Your child must be between 3 and 14 years old.
- Your child must have transfusion-dependent beta-thalassemia (needs regular blood transfusions).
- If your child has already had gene therapy, they can join the gene therapy group.
- If your child has never had gene therapy or a stem cell transplant, they can join the supportive therapy group.
- Your child cannot have another type of thalassemia called compound alpha-thalassemia.
- Your child cannot have an uncontrolled serious infection or a history of cancer, immune disorders, or mental illness.
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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