JAG201 Gene Therapy Study in Children & Adults With SHANK3 Haploinsufficiency
Recruiting now · Phase 1/Phase 2
Conditions studied: SHANK3 Haploinsufficiency, Phelan-McDermid Syndrome
In brief
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.
Key facts
- Study ID
- NCT06662188
- Run by
- Jaguar Gene Therapy, LLC
- People needed
- 6
- Starts
- 2024-01-07
- Expected to finish
- 2031-06-01
- Last updated by the study team
- 2026-02-09
Who can join
Age: 2 and older, up to 9. Sex: any. Healthy volunteers: not accepted.
Where it is running
- Rush University — Chicago, Illinois, United States (enrolling)
- Boston Children's Hospital — Boston, Massachusetts, United States (enrolling)
- Seaver Autism Center at Mount Sinai — New York, New York, United States (enrolling)
Full record on ClinicalTrials.gov
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