Gene therapy for children with SHANK3-related condition
Part of Brain & nervous system, Genetic & congenital clinical trials.
This trial tests a gene therapy called JAG201 for children with a SHANK3 mutation or 22q13.3 deletion, which causes Phelan-McDermid syndrome. The goal is to see if it can help with development and daily function.
Summary written for real people, not researchers, by Clin2.
Who can take part
- A child between 2 and 9 years old at the time of treatment
- Genetic test confirmed a loss-of-function mutation in SHANK3 or a certain type of 22q13.3 deletion
- Significant developmental or cognitive delay (IQ or score 70 or below)
- Commit to ongoing therapies like physical, speech, or behavioral therapy during the study
- Be a permanent legal resident of the continental U.S.
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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