Stem cell transplant with donor T-cells for severe bone marrow failure
Part of Blood & lymphatic clinical trials.
This Phase 2 study tests a specific type of donor stem cell transplant for severe bone marrow failure conditions, aiming to improve cure rates while reducing transplant risks. It includes people with diseases like severe aplastic anemia and certain bone marrow disorders, especially when standard transplant plans feel too risky.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You have one of these diagnoses: severe aplastic anemia (or pure red cell aplasia), paroxysmal nocturnal hemoglobinuria (PNH), or certain myelodysplastic syndromes (MDS) with blood counts issues
- Your illness should be considered potentially curable with a donor stem cell transplant, and your team is concerned standard transplant may be too risky
- You are between ages 4 and 80, and you weigh more than 15 kg
- A suitable donor is available: either a close family donor with matching tissue markers (HLA) or a well-matched unrelated donor
- Your heart, kidney, liver, and lung function meet safety cutoffs (including heart pumping strength, kidney tests, liver tests, and lung breathing test)
- You can do the consent process (including age-appropriate understanding), and you are not pregnant or breastfeeding
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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