Could a diabetes drug help kids with rare PMM2-CDG?
NCT ID NCT04925960
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tested an oral drug called epalrestat, originally used for diabetic nerve pain, in children with PMM2-CDG, a rare genetic disorder that causes nerve problems and other symptoms. The trial aimed to see if the drug could safely improve nerve function and metabolic markers. However, the study was terminated early, so we have limited data on its effectiveness.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
Epalrestat (a drug used for diabetic neuropathy in Japan)
What this could lead to
If successful, epalrestat could offer a way to manage neuropathy symptoms and improve quality of life for children with PMM2-CDG.
What could go wrong
The trial was terminated early, so results are limited. Epalrestat is repurposed from diabetes and may not work for this rare genetic condition.
Disclaimer
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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.
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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Locations
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Mayo Clinic
Rochester, Minnesota, 55905, United States
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