Stem cell transplant using T-cell cleanup for immune and metabolism disorders
Part of Genetic & congenital, Hormones & metabolism, Immune system & allergy clinical trials.
This Phase 2 study tests a specialized stem cell transplant that removes certain “T-cells” (a type of immune cell) to treat children with serious immune system or inherited metabolic problems. The goal is to improve or stabilize disease by resetting the immune system and supporting the body’s function.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You (or your child) must have a serious inherited immune disorder, related immune overactivity, or certain childhood blood/immune cell conditions, or some inherited metabolic disorders.
- There must be a donor-recipient genetic match: a 5-out-of-10 matching donor with the required shared HLA markers.
- Heart, lung, kidney, and liver tests must be good enough for transplant (specific cutoff values are required).
- The child must be well enough to participate in study visits (age-based activity score must be at least 50).
- A parent/guardian must be able to sign informed consent (and the patient if appropriate).
- The child must not be currently pregnant/breastfeeding, and must not have uncontrolled infections like HIV or major ongoing viral/fungal/bacterial illness.
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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