NCT06662188View on ClinicalTrials.gov

JAG201 Gene Therapy Study in Children & Adults With SHANK3 Haploinsufficiency

PHASE1/PHASE2RecruitingINTERVENTIONAL
Enrollment

6

Participants

Timeline

Start Date

January 7, 2024

Primary Completion Date

June 30, 2028

Study Completion Date

June 30, 2031

Conditions
SHANK3 HaploinsufficiencyPhelan-McDermid Syndrome
Interventions
GENETIC

JAG201

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

GENETIC

JAG201

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

Trial Locations (3)

10029

RECRUITING

Seaver Autism Center at Mount Sinai, New York

60612

RECRUITING

Rush University, Chicago

02115

RECRUITING

Boston Children's Hospital, Boston

Sponsors
All Listed Sponsors
lead

Jaguar Gene Therapy, LLC

INDUSTRY