Gene therapy trial for Canavan disease in young children
Part of Brain & nervous system, Genetic & congenital, Hormones & metabolism clinical trials.
This Phase 1/2 trial tests an AAV9 gene therapy to treat Canavan disease. It aims to improve disease-related findings in children whose genetic test confirms ASPA gene changes.
Summary written for real people, not researchers, by Clin2.
Who can take part
- Your child must be 30 months old or younger
- Your child must have Canavan disease confirmed by urine NAA levels, genetic ASPA testing, and symptoms
- Your child’s overall health should be stable, with no other major blood, kidney, liver, immune, or nerve disorders
- Your child must be up to date on immunizations per local guidelines
- Blood testing must show no prior antibodies against AAV9 (gene therapy delivery vehicle)
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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