NCT07173153View on ClinicalTrials.gov

Gene Therapy for SLC6A1 Neurodevelopmental Disorder

PHASE1/PHASE2Enrolling by invitationINTERVENTIONAL
Enrollment

1

Participants

Timeline

Start Date

August 25, 2025

Primary Completion Date

August 25, 2028

Study Completion Date

August 25, 2030

Conditions
SLC6A1
Interventions
BIOLOGICAL

AAV9.SLC6A1 Gene Therapy

This is an open-label, single injection study of an AAV9 vector carrying the SLCA1 coding sequence delivered one time through an intrathecal injection.

Trial Locations (1)

43205

Nationwide Children's Hospital, Columbus

All Listed Sponsors
lead

Emily de los Reyes

OTHER