Actively Recruiting
JAG201 Gene Therapy Study in Children & Adults With SHANK3 Haploinsufficiency
Led by Jaguar Gene Therapy, LLC · Updated on 2026-02-09
6
Participants Needed
3
Research Sites
386 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
This is a Phase 1/2, first in human, open-label, dose-escalation study to evaluate the safety, tolerability, and clinical activity of a single dose of JAG201 administered via intracerebroventricular (ICV) injection in pediatric and adult participants with SHANK3 haploinsufficiency resulting from SHANK3 loss of function mutations and chromosomal deletions encompassing the SHANK3 gene. Clinical data will be evaluated for safety, tolerability, and preliminary clinical activity of JAG201 in pediatric and adult participants with SHANK3 haploinsufficiency. The pediatric cohorts will start enrolling first and the enrollment for adult cohorts may be initiated at a later timepoint in the study.
CONDITIONS
Official Title
JAG201 Gene Therapy Study in Children & Adults With SHANK3 Haploinsufficiency
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Male or female aged 2 to 9 years at time of JAG201 administration
- Molecular confirmation of SHANK3 loss of function mutation or 22q13.3 Class I deletion
- Evidence of developmental or cognitive delay with IQ or Developmental Quotient score of 70 or below
- Phelan-McDermid Syndrome Assessment of Severity score of 3 or higher at screening
- Willingness to start and continue structured therapies (physical, occupational, speech, behavior) during study
- Stable medication regimen for at least 3 months before planned treatment
- Stable behavioral or therapeutic intervention level for at least 3 months before planned treatment
- Permanent legal resident of the continental United States
You will not qualify if you...
- History of developmental regression with loss of skills persisting at least 3 months
- Known or suspected prion disease such as Creutzfeldt-Jakob Disease
- Poorly-controlled epilepsy or seizure-related hospitalizations within past 12 months
- History of acute cerebrovascular episodes
- Active autoimmune disease or immunomodulatory treatment within 3 months prior to enrollment
- Infection requiring treatment within 6 weeks before JAG201 administration
- Medical illness or condition interfering with study procedures or data interpretation
- Known allergy or hypersensitivity to prednisolone, glucocorticosteroids, or their excipients
- Vaccination within 6 weeks before JAG201 administration
- Prior gene therapy treatment
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 3 locations
1
Rush University
Chicago, Illinois, United States, 60612
Actively Recruiting
2
Boston Children's Hospital
Boston, Massachusetts, United States, 02115
Actively Recruiting
3
Seaver Autism Center at Mount Sinai
New York, New York, United States, 10029
Actively Recruiting
Research Team
J
Jaguar Gene Therapy
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
2
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