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NCT06662188 · ClinicalTrials.gov registry record · Phase 1
JAG201 Gene Therapy Study in Children & Adults With SHANK3 Haploinsufficiency
A Phase 1 study of Phelan-McDermid Syndrome and SHANK3 Haploinsufficiency, sponsored by Jaguar Gene Therapy.
- Recruiting
- Registry status
- Phase 1
- Development phase
- 6
- Enrollment target
- 3
- Study locations
NCT06662188 is a Phase 1 study of Phelan-McDermid Syndrome and SHANK3 Haploinsufficiency that is actively recruiting participants, run by Jaguar Gene Therapy. The registered enrollment target is 6 participants, below the 110-participant average among 4 other Phelan-McDermid Syndrome trials with a reported enrollment target (95% lower). The trial reports 3 study locations across 3 states.
The verdict
NCT06662188, a Phase 1 study of Phelan-McDermid Syndrome and SHANK3 Haploinsufficiency, is actively recruiting participants, sponsored by Jaguar Gene Therapy.
- RECRUITING
- Registry status
- Phase 1
- Development phase
- 6 participants
- Enrollment target
- 3
- Study locations
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 Studied
Interventions
- GENETIC JAG201
Study Locations (3)
Illinois
- Rush University - Chicago
Massachusetts
- Boston Children's Hospital - Boston
New York
- Seaver Autism Center at Mount Sinai - New York
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 6 participants |
| Start Date | 2024-01-07 |
| Est. Completion | 2031-06 |
| Phase | Phase 1 |
Interested in This Trial?
Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT06662188
The ClinicalTrials.gov registry entry for NCT06662188 describes a study currently listed as recruiting, categorized as Phase 1. The registered enrollment target is 6 participants, a figure that helps gauge the scale of data the investigators plan to collect, below the 110-participant average among 4 other Phelan-McDermid Syndrome trials with a reported enrollment target (95% lower). The listed sponsor is Jaguar Gene Therapy, which has 1 total studies on file at ClinicalTrials.gov.
The record links to 2 conditions, with Phelan-McDermid Syndrome appearing as the primary indexed condition, and to 1 intervention - of which JAG201 is the first listed.
NCT06662188 reports 3 study locations spanning 3 distinct geographic areas - top geographies include Illinois, Massachusetts, New York.
Frequently Asked Questions
What is clinical trial NCT06662188 about?
NCT06662188 is a clinical study titled "JAG201 Gene Therapy Study in Children & Adults With SHANK3 Haploinsufficiency". 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 SHA...
What is the current status of trial NCT06662188?
This trial is currently recruiting. It is a Phase 1 study. The enrollment target is 6 participants. The study started on 2024-01-07. Estimated completion is 2031-06.
What conditions does trial NCT06662188 study?
This clinical trial studies the following conditions: Phelan-McDermid Syndrome, SHANK3 Haploinsufficiency.
What interventions are being tested in trial NCT06662188?
The interventions under investigation include: JAG201 (GENETIC).
Who is sponsoring clinical trial NCT06662188?
This trial is sponsored by Jaguar Gene Therapy, which has 1 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT06662188 being conducted?
This trial has 3 study locations across Illinois, Massachusetts, New York. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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