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JAG201 Gene Therapy Study in Children & Adults With SHANK3 Haploinsufficiency
NCT06662188 · View on ClinicalTrials.gov ↗
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 |
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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. It is categorized as Phase 1, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 6 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Jaguar Gene Therapy, which has 1 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.
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. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.
Geographic footprint matters for practical reasons: NCT06662188 reports 3 study locations spanning 3 distinct geographic areas — top geographies include Illinois, Massachusetts, New York. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.
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. These conditions were identified from the trial registry and reflect the primary focus areas of the research.
What interventions are being tested in trial NCT06662188?
The interventions under investigation include: JAG201 (GENETIC). Each intervention is being evaluated for safety and efficacy as part of this clinical study.
Who is sponsoring clinical trial NCT06662188?
This trial is sponsored by Jaguar Gene Therapy, which has 1 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.
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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