Condition
Pompe Disease (Late-onset) clinical trials and research
As of November 12, 2026, Find a Trial lists 4 Pompe Disease (Late-onset) clinical trials that are recruiting, at sites in 10 countries (most in United States (3), Australia (1), Belgium (1)). By phase: Phase 1/2 (3). Every study is explained in plain English. Data from ClinicalTrials.gov and NIH RePORTER, updated November 12, 2026.
- 4Clinical trials
- 10Countries
Newest Pompe Disease (Late-onset) clinical trials
| Study | Phase | Where | Status | Registered |
|---|---|---|---|---|
| AB-1009 gene therapy for adults with late-onset Pompe disease | Phase 1/2 | Phoenix, Arizona and 8 other locations | Recruiting | Dec 15, 2025 |
| AB-1009 gene therapy for adults with late-onset Pompe disease | Phase 1/2 | Phoenix, Arizona and 8 other locations | Recruiting | Dec 15, 2025 |
| Gene therapy for late-onset Pompe disease | Phase 1/2 | Beijing | Recruiting | Apr 30, 2024 |
| Tracking pregnancy outcomes in women with Pompe disease | — | Phoenix, Arizona and 34 other locations | Recruiting | Dec 4, 2007 |
All recruiting trials (4)
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AB-1009 gene therapy for adults with late-onset Pompe disease
This study tests a one-time IV gene therapy (AB-1009) to try to restore GAA enzyme activity and improve muscle and breathing function in adults with late-onset Pompe disease who are already on enzyme replacement therapy.
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AB-1009 gene therapy for adults with late-onset Pompe disease
This study tests a one-time IV gene therapy (AB-1009) to try to restore GAA enzyme activity and improve muscle and breathing function in adults with late-onset Pompe disease who are already on enzyme replacement therapy.
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Gene therapy for late-onset Pompe disease
This study is testing a new gene therapy called GC301 to see if it can improve breathing and overall health in kids and adults with late-onset Pompe disease.
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Tracking pregnancy outcomes in women with Pompe disease
This study looks at the pregnancy experiences and outcomes for women with Pompe disease to see how it affects both them and their babies.
Quick answers
How many Pompe Disease (Late-onset) clinical trials are recruiting right now?
4, as of November 12, 2026. We update this list each time we refresh our data from ClinicalTrials.gov.
Where are these trials running?
In 10 countries. The most sites are in United States (3 trials), Australia (1 trial), Belgium (1 trial), Brazil (1 trial), Croatia (1 trial). Each study page lists its sites and which ones are recruiting.
What phases are they in?
Phase 1/2: 3. Trials with no phase listed are usually device, behavioural or observational studies.
How do I join one?
Open a study, check the "Is this trial a fit for you?" section, then contact the study team listed on the page. They will ask a few screening questions. Asking is free and does not commit you to anything. Talk it over with your doctor first.
Find the studies that fit you
A list is a starting point. Tell us your situation in your own words and we'll rank the studies that match and explain why. It's free and needs no account.