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JAG201 Gene Therapy Study in Children & Adults With SHANK3 Haploinsufficiency

NCT06662188 · Status: RECRUITING · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 6

Last updated 2026-02-09

No results posted yet for this study

Summary

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

  • SHANK3 Haploinsufficiency
  • Phelan-McDermid Syndrome

Interventions

GENETIC

JAG201

Adeno-associated virus 2/9 expressing a miniature version of the human SHANK3 gene (AAV2/9-miniSHANK3)

GENETIC

JAG201

Adeno-associated virus 2/9 expressing a miniature version of the human SHANK3 gene (AAV2/9-miniSHANK3)

Sponsors & Collaborators

  • Jaguar Gene Therapy, LLC

    lead INDUSTRY

Principal Investigators

  • Dan Gallo, PhD · Jaguar Gene Therapy

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SEQUENTIAL

Eligibility

Min Age
2 Years
Max Age
9 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2024-01-07
Primary Completion
2028-06-30
Completion
2031-06-30
FDA Drug
Yes

Countries

  • United States

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT06662188 on ClinicalTrials.gov