Intrathecal Gene Therapy For SLC13A5 Citrate Transporter Disorder

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

First listed

Aug 3, 2025

Start date

Jul 15, 2026

Status verified

Jun 2026

Primary completion

Dec 1, 2030

Completion

Jun 1, 2031

Study Design

Enrollment

8 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

• Alexandria Silver, RN
  214-456-0876
  [email protected]
• Holly Lawrence
  [email protected]

Locations (1)

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