Pompe drug study halted after just 3 patients
NCT ID NCT00688597
First seen Nov 01, 2025 · Last updated May 21, 2026 · Updated 31 times
Summary
This study tested the safety of three different doses of an oral drug called duvoglustat in adults with Pompe disease, a rare genetic disorder that causes muscle weakness. Only 3 people took part, and the study was stopped early. The main goal was to see if the drug caused severe side effects.
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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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Study site
Decatur, Georgia, 30033, United States
Conditions
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