Gene therapy trial hopes to tackle rare Autism-Linked condition
NCT ID NCT06662188
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This early-stage trial tests a single dose of JAG201 gene therapy, given directly into the brain fluid, for people with SHANK3 haploinsufficiency (Phelan-McDermid syndrome), a rare genetic condition causing developmental delays and autism-like symptoms. The study includes children aged 2-9 and may later include adults. The main goal is to check safety and tolerability, while also looking for signs of improvement in sensory and developmental function.
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the original study
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Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Boston Children's Hospital
RECRUITINGBoston, Massachusetts, 02115, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact
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Rush University
RECRUITINGChicago, Illinois, 60612, United States
Contact
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Seaver Autism Center at Mount Sinai
RECRUITINGNew York, New York, 10029, United States
Contact
Contact Phone: •••-•••-•••• Email: •••••@•••••