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.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for POMPE DISEASE are added.
By submitting, you agree to our Terms of use
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
-
Study site
Decatur, Georgia, 30033, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Can an oral pill boost muscle strength in pompe disease?
- Hope for pompe patients: could a switch in enzyme therapy slow decline?
- Pompe disease: new study probes hidden nerve damage behind breathing problems
- Heart condition costs under the microscope in massive hospital study
- Pompe disease study aims to unlock immune secrets for better treatment
- Pompe disease study peers into the brain for hidden clues