Gene therapy hope for kids with rare citrate disorder
NCT ID NCT07102524
First seen Jun 25, 2026 · Last updated Jul 01, 2026 · Updated 3 times
Summary
This early-stage trial tests a single dose of TSHA-105, a gene therapy injected into the spinal fluid, in 8 people aged 2 to 20 with SLC13A5 citrate transporter disorder. The goal is to see if it is safe and whether it can improve motor and thinking skills. Because the trial is very small and open-label, results will be preliminary.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- TSHA-105 (a gene therapy given as a single injection into the spinal fluid)
- What this could lead to
- If it works, this could point toward a treatment that improves motor and cognitive skills in children with SLC13A5 disorder.
- What could go wrong
- This is a very early, small trial with only 8 participants. It is designed mainly to check safety, so it may not show clear benefit. Gene therapies can have side effects like immune reactions.
This is an AI summary of the original study and may miss details. Read our disclaimer.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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University of Texas Southwestern Medical Center
RECRUITINGDallas, Texas, 75390-7208, United States