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.
Disclaimer
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This is a summary of
the original study
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Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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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
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
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Contact
Email: •••••@•••••
Locations
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University of Texas Southwestern Medical Center
RECRUITINGDallas, Texas, 75390-7208, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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