Immune-suppressing treatment before stem cell transplant for blood disorders
Treatments studied
Part of Blood & lymphatic, Genetic & congenital clinical trials.
This Phase 2 study tests an immune-suppressing treatment for people with sickle cell disease or beta-thalassemia who are preparing for a stem cell transplant from a donor. It aims to help the transplant work better by changing the immune response.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must be between ages 2 and 50
- You need a suitable “half-matching” (haploidentical) donor for the transplant
- Your heart, lungs, kidneys, and liver tests must be strong enough for transplant (specific numbers will be checked)
- For sickle cell: you must have certain serious past complications (like frequent painful episodes or acute chest syndrome) or specific blood/brain/lung circulation findings
- For thalassemia: you must meet specific criteria about early, frequent transfusions and related complications (like enlarged liver/spleen)
- You must be able to consent (and minors may need assent) and follow the study plan; if sexually active, you must use effective birth control
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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