Early-Stop trial tests pill for rare muscle disease
NCT ID NCT00688597
Summary
This study tested the safety and effects of three different dosing schedules of an oral drug called duvoglustat (AT2220) in adults with Pompe disease, a rare genetic disorder that weakens muscles. The goal was to see if the drug was well-tolerated and if it could improve muscle function and walking ability. The trial was terminated early after enrolling only three participants.
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.
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
Contacts and locations
Show contact details
Enter your email to view the contact information for this study.
By submitting, you agree to our Terms of use
Locations
-
Study site
Decatur, Georgia, 30033, United States
Conditions
Explore the condition pages connected to this study.