Gene therapy for metachromatic leukodystrophy using a patient's own stem cells
Part of Brain & nervous system, Genetic & congenital, Hormones & metabolism clinical trials.
This trial tests a new gene therapy for metachromatic leukodystrophy (MLD), a rare genetic disease that damages the nervous system. The therapy uses a patient's own blood stem cells, modified to help the body produce a missing enzyme, to potentially slow or stop the disease.
Summary written for real people, not researchers, by Clin2.
Who can take part
- Must be at least 1 month old
- Must have a confirmed genetic test showing MLD (metachromatic leukodystrophy)
- Must be able to have an MRI (imaging test) and have brain images taken
- A parent or guardian must sign a consent form and be willing to take part fully
- Must not have HIV, uncontrolled infections, cancer, serious heart/liver/kidney problems, or certain other neurological conditions
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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