Gene therapy for GM1 disease with IV delivery
Treatments studied
Part of Brain & nervous system, Genetic & congenital, Hormones & metabolism clinical trials.
This early-stage study tests an IV gene therapy that delivers a working copy of an enzyme gene to help the body make beta-galactosidase in GM1 gangliosidosis. It may help slow or improve disease symptoms and looks closely at safety and how well the treatment works.
Summary written for real people, not researchers, by Clin2.
Who can take part
- Your child must have GM1 gangliosidosis type I (age 6–12 months) or type II (age >6 months to <12 years, with onset after age 1).
- Genetic testing must show two disease-causing (biallelic) changes in the GLB1 gene.
- Testing must confirm beta-galactosidase enzyme deficiency.
- Blood test must show AAV9 antibody levels at or below 1:50.
- You must be able to live within 50 miles of the study site for at least 1 month after treatment.
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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