New pompe disease drug moves to first human tests
NCT ID NCT06109948
First seen Jan 06, 2026 · Last updated May 08, 2026 · Updated 18 times
Summary
This early-phase study tested a new drug called ABX1100 in 46 healthy volunteers and people with late-onset Pompe disease. The main goal was to check safety and how the drug moves through the body. Researchers gave single or multiple doses by IV and watched for side effects over several weeks.
Disclaimer
Read more
Show less
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.
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for HEALTHY 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
-
Lysosomal and Rare Disorders Research and Treatment Center, Inc
Fairfax, Virginia, 22030, United States
-
MAGIC clinic
Calgary, Alberta, Canada
-
McMaster University
Hamilton, Ontario, Canada
-
UCI
Orange, California, 92868, United States
-
Washington University School of Medicine
St Louis, Missouri, 63110, United States
Conditions
Explore the condition pages connected to this study.