JAG201 Gene Therapy for SHANK3 Haploinsufficiency
This study is testing a gene therapy called JAG201 for children and adults with SHANK3 haploinsufficiency, including those with Phelan-McDermid Syndrome. JAG201 is a special virus (Adeno-associated virus 2/9) that delivers a miniature version of the human SHANK3 gene. The main goal of this study is to see if JAG201 is safe and well-tolerated. Researchers will also look for any early signs that the treatment is working. You may be able to join if you are between 2 and 9 years old and have a confirmed SHANK3 genetic change and developmental delays. The study is currently enrolling a small number of participants, with a planned enrollment of 6 children first.
- Study design
- This is a Phase 1/2, first-in-human, open-label study, meaning everyone knows what treatment is being given. It aims to enroll a target of 6 pediatric participants aged 2 to 9 years.
- What's involved
- You would undergo pre-screening and screening for up to 90 days, receive a one-time injection of JAG201, and then have regular follow-up visits for safety and clinical activity assessments.
- Compensation
- Not stated in the trial record.
- Follow-up
- Participants will be followed for safety and tolerability for at least 5 years after receiving the JAG201 treatment.
AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.
JAG201 Gene Therapy Study in Children & Adults With SHANK3 Haploinsufficiency
At a glance
Conditions
NCT06662188
Where you'd take part
This study runs at 3 sites. They're the same protocol — you choose where, and that choice sets who your contact draft is addressed to.
Boston Children's Hospital
Boston, Massachusettsstudy coordinator listed
Recruiting
Rush University
Chicago, Illinoisstudy coordinator listed
Recruiting
Seaver Autism Center at Mount Sinai
New York, New Yorkstudy coordinator listed
Recruiting
Sites open and close at different times, so the status above is per site — it can differ from the study's overall status.
Study leadership
- Dan Gallo, PhD · STUDY_DIRECTOR · Jaguar Gene Therapy
Who to contact
Opens a ready-to-send draft in your own email app — review before sending.
What this trial measures
- Incidence of Adverse Events (AEs)Enrollment to Month 60
Incidence, type, severity, and frequency of AEs
- Incidence of Serious Adverse Events (SAEs)Enrollment to Month 60
Incidence, type, severity, and frequency of SAEs
- Clinically significant abnormalities in laboratory valuesEnrollment to Month 60
Changes in clinically significant abnormalities in laboratory values
- Incidence of immunogenicity response abnormalitiesEnrollment to Month 60
Incidence of anti-AAV9 antibodies, anti-transgene antibodies, and T-cell reactivity to transgene over time