drugset
drugset / Trial / NCT07102524

Intrathecal Gene Therapy For SLC13A5 Citrate Transporter Disorder

NCT07102524 ↗

Phase 1/2 Recruiting 8 enrolled TESS Research Foundation
NaSingle-groupOpen-labelTreatment

Summary

Phase 1/2, open-label study to assess the efficacy and safety of a single lumbar intrathecal administration of TSHA-105 in individuals with SLC13A5 Citrate Transporter Disorder

Timeline

Start
2026-07-15
Primary completion
2030-12-01
Completion
2031-06-01

Drugs

EvaluationDrugModalityDoseRoute
Subject TSHA-105 Gene therapy (AAV / viral vector) — Intrathecal

Indications

developmental and epileptic encephalopathy, 25