Gene therapy for FHL type 3 (UNC13D mutation)
Part of Blood & lymphatic, Genetic & congenital, Immune system & allergy clinical trials.
This trial tests a one-time gene therapy for FHL type 3, a rare immune disorder. Doctors take your own blood stem cells, add a working copy of the UNC13D gene, and then put them back into your body. The goal is to fix the immune system so you don't need a transplant from a donor.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must be between 3 months and 45 years old.
- You must have a confirmed diagnosis of FHL type 3 caused by a mutation in the UNC13D gene.
- Your HLH symptoms must be completely under control (no fever, spleen nearly normal, blood counts improved, and other lab values stable).
- You must be eligible for a stem cell transplant, but not have a perfect matched brother or sister donor.
- You must be willing to use effective birth control during the study and for 12 months after treatment.
- You cannot be pregnant, breastfeeding, or have active HIV/HTLV infections.
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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