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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
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