Gene therapy trial for severe sickle cell disease: standard care comparison
Part of Blood & lymphatic, Genetic & congenital clinical trials.
This trial is part of a gene therapy study for severe sickle cell disease. It compares the usual care (like hydroxyurea or transfusions) to help understand if gene therapy is better. You might join if you have had serious complications and other treatments haven't helped enough.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You are between 12 and 35 years old.
- You have sickle cell disease (type HbSS).
- You have had severe problems like repeated pain crises, lung issues, or priapism.
- You have tried hydroxyurea but it didn't work well or you couldn't take it.
- You do not have a matched brother or sister for a bone marrow transplant.
- You have not had gene therapy or a bone marrow transplant before.
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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