Gene therapy for metachromatic leukodystrophy
Part of Brain & nervous system, Genetic & congenital, Hormones & metabolism clinical trials.
This trial tests a new gene therapy for metachromatic leukodystrophy (MLD), a rare inherited condition that damages the nervous system. The treatment involves injecting a modified virus directly into the body to deliver a working copy of the gene that is faulty in MLD. This may help slow or stop the disease from getting worse.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must be at least 1 month old
- Must have a confirmed diagnosis of MLD through a genetic test
- Must have brain MRI changes typical of MLD
- Must be willing to sign a consent form (parent or guardian if under 18)
- Must be generally healthy except for MLD – no serious heart, liver, or kidney disease, and no active infections or other brain disorders
- Must be able to have an MRI scan
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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