Intrathecal Gene Therapy For SLC13A5 Citrate Transporter Disorder
NCT07102524 · Status: NOT_YET_RECRUITING · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 2
Last updated 2025-08-14
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
Conditions
- SLC13A5 Citrate Transporter Disorder
Interventions
- DRUG
-
TSHA-105
AAV9/SLC13A5
Sponsors & Collaborators
-
TESS Research Foundation
lead OTHER
Study Design
- Allocation
- NA
- Purpose
- TREATMENT
- Masking
- NONE
- Model
- SINGLE_GROUP
Eligibility
- Min Age
- 2 Years
- Max Age
- 20 Years
- Sex
- ALL
- Healthy Volunteers
- No
Timeline & Regulatory
- Start
- 2025-12-01
- Primary Completion
- 2030-12-01
- Completion
- 2031-06-01
- FDA Drug
- Yes
Countries
- United States
Study Locations
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