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.
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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Mayo Clinic
Rochester, Minnesota, 55905, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.