Gene Therapy for SLC6A1 Neurodevelopmental Disorder

Part of paid clinical trials in Columbus, Ohio.

Sponsor
Emily de los Reyes
Study ID
NCT07173153
Phase
PHASE1/PHASE2
Status
Enrolling By Invitation

Conditions

  • SLC6A1

Eligibility Criteria

Sex
ALL
Age
N/A - N/A
Healthy Volunteers
Not accepted

Interventions

  • AAV9.SLC6A1 Gene Therapy — BIOLOGICAL
    This is an open-label, single injection study of an AAV9 vector carrying the SLCA1 coding sequence delivered one time through an intrathecal injection.

Study Details

This is gene therapy study of an AAV9 vector carrying the SLCA1 gene for SLC6A1 neurodevelopmental disorder.

Key Dates

Start date
Aug 25, 2025
Status verified
Sep 2025
Primary completion
Aug 25, 2028
Completion
Aug 25, 2030

Study Design

Enrollment
1 participants (estimated)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: AAV9.SLC6A1 Gene Therapy

Primary Outcome Measure

Monitoring for the development of unacceptable toxicity. [ Time Frame: 3 years ]

Locations (1)

FacilityCityStateZIPSite coordinators
Nationwide Children's HospitalColumbusOhio43205-

Find similar trials in Columbus, OH

By research site
Nationwide Children's Hospital· Columbus, OH

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