Gene therapy hope for pompe patients in early safety trial
NCT ID NCT03533673
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This early-phase study tested a new gene therapy called ACTUS-101 in 7 adults with late-onset Pompe disease. The main goal was to check safety and side effects. Participants received a single intravenous dose, and researchers monitored their health over time.
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
-
Duke University
Durham, North Carolina, 27705, 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