drugset

drugset / Trial / NCT07173153

Gene Therapy for SLC6A1 Neurodevelopmental Disorder

NCT07173153 ↗

Phase 1/2 Enrolling by invitation 1 enrolled Emily de los Reyes

NaSingle-groupOpen-labelTreatment

Summary

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

Timeline

Start: 2025-08-25
Primary completion: 2028-08-25
Completion: 2030-08-25

Publications

Samanta D. SLC6A1-Related Neurodevelopmental Disorder: A Scoping Review of Clinical Features and Emerging Therapeutic Strategies. Pediatr Neurol. 2026 May 5;180:155-170. doi: 10.1016/j.pediatrneurol.2026.04.014. Online ahead of print.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	AAV9.SLC6A1	Gene therapy (AAV / viral vector)	—	Intrathecal

Indications