Bone marrow transplant with lighter chemo for non-cancer illnesses
Part of Blood & lymphatic, Genetic & congenital, Hormones & metabolism, Immune system & allergy clinical trials.
This early-phase study tests a safer “lighter” conditioning chemo plan before a bone marrow transplant for people with non-cancer blood and immune disorders. It aims to reduce transplant risk while using donor marrow that does not match perfectly in the HLA type.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You need a bone marrow transplant for a non-cancer problem (like bone marrow failure, immune problems, metabolic disorders, or blood disorders).
- If you have sickle cell disease, you must have severe symptoms (such as stroke/TCD issues, repeat lung episodes, kidney problems, frequent painful crises, ongoing transfusions, or repeated bone/joint injury).
- If you have sickle cell disease, your hemoglobin S level must be under 30% within 30 days before starting the study drug alemtuzumab.
- You must be 20 years old or younger at enrollment, and your heart and lung tests must be strong enough (echocardiogram and oxygen/lung function limits).
- Your liver and kidney blood tests must be within allowed ranges, and you must not have known cirrhosis, bridging liver scarring, or active hepatitis.
- You must not have an HLA-matched sibling donor available, and you must not have had a prior stem cell transplant.
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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