Trials / Enrolling By Invitation
Enrolling By InvitationNCT07173153
Gene Therapy for SLC6A1 Neurodevelopmental Disorder
Phase I/II Intrathecal Gene Delivery Clinical Trial of scAAV9.P546.SLC6A1 for SLC6A1 Neurodevelopmental Disorder
- Status
- Enrolling By Invitation
- Phase
- Phase 1 / Phase 2
- Study type
- Interventional
- Enrollment
- 1 (estimated)
- Sponsor
- Emily de los Reyes · Academic / Other
- Sex
- All
- Age
- —
- Healthy volunteers
- Not accepted
Summary
This is gene therapy study of an AAV9 vector carrying the SLCA1 gene for SLC6A1 neurodevelopmental disorder.
Conditions
Interventions
| Type | Name | Description |
|---|---|---|
| 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. |
Timeline
- Start date
- 2025-08-25
- Primary completion
- 2028-08-25
- Completion
- 2030-08-25
- First posted
- 2025-09-15
- Last updated
- 2025-09-15
Locations
1 site across 1 country: United States
Regulatory
- FDA-regulated drug study
Source: ClinicalTrials.gov record NCT07173153. Inclusion in this directory is not an endorsement.