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Gene Therapy for SLC6A1 Neurodevelopmental Disorder

NCT07173153 · Status: ENROLLING_BY_INVITATION · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 1

Last updated 2025-09-15

No results posted yet for this study

Summary

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

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.

Sponsors & Collaborators

  • Emily de los Reyes

    lead OTHER

Principal Investigators

  • Emily de los Reyes, MD · Nationwide Children's Hospital

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2025-08-25
Primary Completion
2028-08-25
Completion
2030-08-25
FDA Drug
Yes

Countries

  • United States

Study Locations

More Related Trials

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT07173153 on ClinicalTrials.gov