NCT07102524

Intrathecal Gene Therapy For SLC13A5 Citrate Transporter Disorder

A Phase 1/2 Open-Label Intrathecal Administration of TSHA-105 to Determine the Safety and Efficacy in 2 Subjects With SLC13A5 Citrate Transporter Disorder Caused by a Mutation in the SLC13A5 Gene

• Status: Not Yet Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 2 (estimated)
• Sponsor: TESS Research Foundation · Academic / Other
• Sex: All
• Age: 2 Years – 20 Years
• Healthy volunteers: Not accepted

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

Detailed description

TSHA-105 is an AAV9-based gene therapy vector that expresses the fully functional form of SLC13A5 under the control of a synthetic promoter. TSHA-105 will be delivered intrathecally and is designed to achieve stable, potentially life-long expression of SLC13A5 protein in non-dividing cells. This clinical study is a pivotal open-label phase 1/2 study designed to assess safety and efficacy of TSHA-105 in individuals with SLC13A5 Citrate Transporter Disorder.

Conditions

• SLC13A5 Citrate Transporter Disorder

Interventions

Type	Name	Description
DRUG	TSHA-105	AAV9/SLC13A5

Timeline

• Start date: 2025-12-01
• Primary completion: 2030-12-01
• Completion: 2031-06-01
• First posted: 2025-08-03
• Last updated: 2025-08-14

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study