Pompe disease drug trial halted after just 3 patients
NCT ID NCT00688597
First seen Jun 26, 2026 · Last updated Jun 26, 2026
Summary
This study tested 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. Researchers looked at safety and whether the drug could improve walking distance and other functions.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
duvoglustat (AT2220)
What this could lead to
If successful, duvoglustat could offer a new oral treatment option for Pompe disease, potentially improving muscle function and daily activity.
What could go wrong
This was a very small, early-phase study with only 3 participants, and it was terminated early. The results may not apply to a broader population, and the drug's safety and effectiveness are not yet proven.
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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Study site
Decatur, Georgia, 30033, United States