Promising PMM2-CDG drug trial halted early
NCT ID NCT04925960
First seen Nov 01, 2025 · Last updated Apr 24, 2026 · Updated 24 times
Summary
This study tested an oral drug called epalrestat in children aged 2 to 17 with PMM2-CDG, a rare genetic disorder that affects how the body makes certain proteins. The goal was to see if the drug could improve symptoms and lab markers compared to a placebo. The trial was terminated early, so results are limited.
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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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Locations
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Mayo Clinic
Rochester, Minnesota, 55905, United States
Conditions
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