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.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for PHELAN-MCDERMID SYNDROME are added.
By submitting, you agree to our Terms of use
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
Show contact details
Enter your email to view the contact information for this study.
By submitting, you agree to our Terms of use
Study contacts
-
Contact
Email: •••••@•••••
Locations
-
Boston Children's Hospital
RECRUITINGBoston, Massachusetts, 02115, United States
-
Rush University
RECRUITINGChicago, Illinois, 60612, United States
-
Seaver Autism Center at Mount Sinai
RECRUITINGNew York, New York, 10029, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Can tracking a rare genetic syndrome unlock its mysteries?
- Can early parent coaching help infants with rare genetic disorders thrive?
- Gene therapy for rare genetic syndrome moves to Long-Term safety check
- Gene therapy injection into the brain aims to treat rare genetic disorder
- New drug shows promise for rare genetic disorder in kids
- Hope for rare genetic disorder: new drug trial targets core symptoms