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Clinical Trial Report
Gene Therapy Trial Report
Summary
JAG201 Gene Therapy Study in Children & Adults with SHANK3 Haploinsufficiency
NCTID
NCT06662188
(View at clinicaltrials.gov)
Description
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.
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Development Status
Active
Indication
SHANK3 Haploinsufficiency, Phelan-McDermid Syndrome
Disease Ontology Term
DOID:0080354
Compound Name
JAG201
Compound Description
AAV2/9-miniSHANK3
Sponsor
Jaguar Gene Therapy, LLC
Funder Type
Industry
Recruitment Status
Recruiting
Enrollment Count
6
Results Posted
Not Available
Therapy Information
Target Gene/Variant
SHANK3
Therapy Type
Gene transfer
Therapy Route
In-vivo
Mechanism of Action
Functional gene replacement
Route of Administration
Intracerebroventricular
Drug Product Type
Viral vector
Target Tissue/Cell
Delivery System
Viral transduction
Vector Type
AAV2/9
Editor Type
none
Dose 1
Undisclosed dose escalation
Dose 2
Dose 3
Dose 4
Dose 5
Study Record Dates
Current Stage
Phase1, Phase2
Submit Date
2024-10-16
Completion Date
2031-06
Last Update
2025-03-05
Participation Criteria
Eligible Age
2 Years - 9 Years
Standard Ages
Child
Sexes Eligible for Study
ALL
Locations
No.of Trial Sites
2
Locations
United States
Regulatory Information
Has US IND
True
FDA Designations
Recent Updates
Clinical sites are now open
Resources/Links
News and Press Releases
JAG201 Clinical Study Enrollment is Now Open
Jaguar Gene Therapy Announces FDA Clearance of IND to Study JAG201 in a Genetic Form of Autism Spectrum Disorder and Phelan-McDermid Syndrome
Jaguar Gene Therapy to Initiate Inaugural Pediatric Clinical Trial Targeting a Genetic Form of Autism Spectrum Disorder and Phelan-McDermid Syndrome
Jaguar Gene Therapy Community Webinar - July 11, 2024
Preclinical Publications
(Poster) Biodistribution Assessment in Non-Human Primates of JAG201, a SHANK3 AAV9 Vector Delivered via ICV Injection for ASD, Phelan McDermid Syndrome, and Other SHANK3 Mutation or Deletion Related Conditions - ASGCT 2023
(Poster) Preclinical Assessment of JAG201, a Clinical Stage Gene Therapy for Severe Neurodevelopmental Disorders Caused by Mutations or Deletions in SHANK3 Including Phelan-McDermid Syndrome (PMS) and Autism Spectrum Disorder (ASD) - ASGCT 2024