Stem cell transplant to rebuild immunity in genetic immune disorders
Part of Blood & lymphatic, Genetic & congenital, Hormones & metabolism, Immune system & allergy, Skin clinical trials.
This Phase 2 trial tests a stem cell (bone marrow) transplant approach to restore bone marrow function and immune function in people with certain inherited immune problems. If you have one of these conditions and a suitable donor, the study may offer a path to rebuild blood and immune systems.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You have a specific inherited immune disorder or inherited bone marrow failure (the trial lists many, like SCID, CGD, WAS, CVID, IPEX, HLH, Fanconi anemia).
- You need a transplant (BMT/HCT) for your condition—this is part of the trial’s starting point.
- You must have a suitable donor: a close family member match or a well-matched unrelated donor (or a partial family match meeting minimum HLA overlap).
- Your organs must work well enough for transplant (heart, liver, kidneys, and lungs have required thresholds).
- You (or your legal guardian) must sign informed consent for the transplant.
- You should not have certain excluded issues, like uncontrolled infections, HIV, active hepatitis B/C, or prior allogeneic 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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