Gene therapy for HLH type 3 caused by UNC13D mutations
Part of Blood & lymphatic, Genetic & congenital, Immune system & allergy clinical trials.
This trial tests a new gene therapy for a rare immune disorder called familial hemophagocytic lymphohistiocytosis (HLH) type 3, caused by mutations in the UNC13D gene. Your own blood stem cells are modified in the lab to carry a working copy of the gene, then given back to you after a mild chemotherapy, aiming to fix the immune problem without needing a bone marrow transplant.
Summary written for real people, not researchers, by Clin2.
Who can take part
- Age between 3 months and 45 years
- Diagnosed with familial hemophagocytic lymphohistiocytosis (HLH) type 3 due to a mutation in the UNC13D gene
- HLH is in remission (fever resolved, spleen not enlarged or only slightly, blood counts improved, and other lab values normal or close to normal)
- Eligible for a bone marrow transplant but no fully matched sibling donor is available
- Willing to use effective contraception if of childbearing age
- Must be affiliated with French Social Security or have an exemption
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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