RecruitingPhase 1Phase 2NCT07102524

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 Subjects With SLC13A5 Citrate Transporter Disorder Caused by a Mutation in the SLC13A5 Gene.

Sponsor

TESS Research Foundation

Enrollment

8 participants

Start Date

Jul 15, 2026

Study Type

INTERVENTIONAL

Conditions

SLC13A5 Citrate Transporter Disorder

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

Eligibility

Min Age: 2 YearsMax Age: 20 Years

Plain Language Summary

Simplified for easier understanding

This clinical trial is studying a drug called TSHA-105 for people with slc13a5 citrate transporter disorder. The study is currently recruiting participants at 1 location. People eligible for this study include aged 2 Years to 20 Years.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

DRUGTSHA-105

AAV9/SLC13A5

Locations(1)

University of Texas Southwestern Medical Center

Dallas, Texas, United States

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NCT07102524