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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: Recruiting Phase 1 study of Phelan-McDermid Syndrome and SHANK3 Haploinsufficiency, sponsored by Jaguar Gene Therapy.

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. According to ClinicalTrials.gov, the official US trial registry.

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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.

Primary Outcome

Incidence, type, severity, and frequency of AEs

Conditions Studied

Phelan-McDermid Syndrome SHANK3 Haploinsufficiency

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

FieldValue
Enrollment Target 6 participants
Start Date 2024-01-07
Est. Completion 2031-06
Phase Phase 1
Jaguar Gene Therapy

1 total trials

What NCT06662188 shows while recruiting

NCT06662188 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 6 participants, a relatively small participant target, below the 110-participant average among 4 other Phelan-McDermid Syndrome trials with a reported enrollment target (95% lower).

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 a single indexed study location in 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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More recruiting context for NCT06662188

Source: ClinicalTrials.gov NCT06662188, the US trial registry maintained by the National Library of Medicine. NCT06662188 (small enrollment · single site footprint · recruiting) retrieved and formatted by PlainTrial, see methodology.