Pompe Disease (Late-onset) Trials in the USA, 2026
Every industry-sponsored study on the ClinicalTrials.gov registry that lists a US site and is open, about to open, active-but-closed, or enrolling by invitation. This deliberately excludes academic and other non-industry sponsors — the full US registry across all sponsors runs to over 22,000 recruiting studies alone.
About this register
This register is built from ClinicalTrials.gov, the trial registry run by the U.S. National Library of Medicine. It includes every industry-sponsored study on the registry that lists at least one investigative site in the US and is recruiting, not yet recruiting, active but no longer recruiting, or enrolling by invitation: 9,144 studies in total at the last sync on 16 Oct 2026, 03:16 UTC. Academic and other non-industry sponsors are out of scope by design. The full list can be downloaded as CSV or queried as JSON through the register API.
This page shows the 6 of those studies listing the condition Pompe Disease (Late-onset).
How many Pompe Disease (Late-onset) trials are recruiting in the US?
2 of the 6 studies on this page are recruiting new participants as of the 16 Oct 2026, 03:16 UTC sync. A further 0 have not yet opened recruitment, 4 are active but closed to new participants, and 0 are enrolling by invitation only. Eligibility for any study is decided by its study team.
Summary by phase
| Phase | Studies | Recruiting | Share |
|---|---|---|---|
| Phase 1/2 | 3 | 1 | 50% |
| Not applicable | 1 | 0 | 16.7% |
| Observational | 1 | 1 | 16.7% |
| Phase 3 | 1 | 0 | 16.7% |
Most active sponsors
| Lead sponsor | Studies | Recruiting |
|---|---|---|
| Astellas Gene Therapies | 2 | 0 |
| Amicus Therapeutics | 1 | 0 |
| AskBio Inc | 1 | 1 |
| Genzyme, a Sanofi Company | 1 | 1 |
| Spark Therapeutics, Inc. | 1 | 0 |
Top states
| State | Studies |
|---|---|
| Georgia | 4 |
| Pennsylvania | 4 |
| Utah | 4 |
| Arizona | 3 |
| California | 3 |
| Florida | 2 |
| Kansas | 2 |
| Michigan | 2 |
| Minnesota | 2 |
| Missouri | 2 |