JAG201 Gene Therapy Study in Children & Adults With SHANK3 Haploinsufficiency

Part of paid clinical trials in Chicago, Illinois.

Sponsor
Jaguar Gene Therapy, LLC
Study ID
NCT06662188
Phase
PHASE1/PHASE2
Status
Recruiting
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Conditions

  • Phelan-McDermid Syndrome
  • SHANK3 Haploinsufficiency

Eligibility Criteria

Sex
ALL
Age
2 Years - 9 Years
Healthy Volunteers
Not accepted

Interventions

  • JAG201 — GENETIC
    Adeno-associated virus 2/9 expressing a miniature version of the human SHANK3 gene (AAV2/9-miniSHANK3)
  • JAG201 — GENETIC
    Adeno-associated virus 2/9 expressing a miniature version of the human SHANK3 gene (AAV2/9-miniSHANK3)

Study Details

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 Dates

Start date
Jan 7, 2024
Status verified
Feb 2026
Primary completion
Jun 30, 2028
Completion
Jun 30, 2031

Study Design

Enrollment
6 participants (estimated)
Allocation
NON_RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT

Arms

  • Experimental: Pediatric Cohort 1
    Starting Dose
  • Experimental: Pediatric Cohort 2
    Escalated Dose

Primary Outcome Measure

Incidence of Adverse Events (AEs) [ Time Frame: Enrollment to Month 60 ]

Central Contacts

Locations (3)

FacilityCityStateZIPSite coordinators
Rush UniversityChicagoIllinois60612
Giulia DeMarco
[email protected]
312-942-9841
Elizabeth B Kravis, MD, PhD (PRINCIPAL_INVESTIGATOR)
Boston Children's HospitalBostonMassachusetts02115
Anna Cronin
[email protected]
(617)-919-3499
Siddharth Srivastava, MD (PRINCIPAL_INVESTIGATOR)
Seaver Autism Center at Mount SinaiNew YorkNew York10029
Serena Cai
[email protected]
212-241-6231
Alex Kolevzon, MD (PRINCIPAL_INVESTIGATOR)

Find similar trials in Chicago, IL

By research site
Rush University· Chicago, ILBoston Children's Hospital· Boston, MASeaver Autism Center at Mount Sinai· New York, NY

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