Intrathecal Gene Therapy For SLC13A5 Citrate Transporter Disorder

Part of paid clinical trials in Dallas, Texas.

Sponsor
TESS Research Foundation
Study ID
NCT07102524
Phase
PHASE1/PHASE2
Status
Not Yet Recruiting

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Conditions

  • SLC13A5 Citrate Transporter Disorder

Eligibility Criteria

Sex
ALL
Age
2 Years - 20 Years
Healthy Volunteers
Not accepted

Interventions

  • TSHA-105 — DRUG
    AAV9/SLC13A5

Study Details

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

Key Dates

Start date
Aug 1, 2026
Status verified
Jul 2025
Primary completion
Dec 1, 2030
Completion
Jun 1, 2031

Study Design

Enrollment
2 participants (estimated)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: TSHA-105 treament
    TSHA-105 Treatment

Primary Outcome Measure

Safety and Tolerability [ Time Frame: 5 years ]

Central Contacts

Locations (1)

FacilityCityStateZIPSite coordinators
University of Texas Southwestern Medical CenterDallasTexas75390-7208
Anne-Marie Van-Jones, RN
[email protected]
214) 645-7000
Dallas Armstrong, MD (PRINCIPAL_INVESTIGATOR)

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University of Texas Southwestern Medical Center· Dallas, TX