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Priya Life Science Clinical trial register
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Trackers› Clinical trials› Pompe Disease (Late-onset)
Clinical trial register · United States · industry-sponsored

Pompe Disease (Late-onset) Trials in the USA

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.

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Source: ClinicalTrials.gov. Complete coverage of the industry-sponsored slice — all 9094 matching records are in this view (non-industry sponsors are out of scope by design, not truncated). Synced 17 Sep 2026, 08:23 UTC
9094
Studies in scope
industry-sponsored, all four open statuses
4935
Recruiting now
open to new participants
2188
Lead sponsors
across recruiting studies
Top states
Pennsylvania 4 Utah 4 Georgia 4 Arizona 3 California 3
Narrow the registerReset
Recruitment status
Phase
Lead sponsor
Astellas Gene Therapies2 AskBio Inc1 Genzyme, a Sanofi Company1 Spark Therapeutics, Inc.1 Amicus Therapeutics1
State
Pennsylvania4 Utah4 Georgia4 Arizona3 California3
All 19 states
Target enrolment
to
6 of 9094 studies
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Status
Study
Lead sponsor
Phase
Enrol
US states
Start
Recruiting
A Study to Evaluate Safety, Tolerability, and Efficacy of AB-1009 Gene Therapy (GAA Gene) in Adult Participants With Late-Onset Pompe Disease (PROGRESS-GT LOPD)
NCT07282847Pompe Disease (Late-onset), Pompe Disease Late-Onset, LOPD
AskBio Inc
Phase 1/2
12
Arizona, California, New York, North Carolina, Pennsylvania, Texas, Virginia
2026-04-15
Active, closed
A Study About Antibody Levels and Biomarkers in the Blood in People With Late-onset Pompe Disease
NCT06150820Pompe Disease (Late-onset)
Astellas Gene Therapies
Not applicable
119
Georgia, Kansas, Michigan, Minnesota, New Jersey, Ohio, Pennsylvania, Utah
2024-02-01
Active, closed
Gene Transfer Study in Patients With Late Onset Pompe Disease
NCT04174105Pompe Disease (Late-onset)
Astellas Gene Therapies
Phase 1/2
11
California, Utah
2020-10-27
Active, closed
A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE)
NCT04093349Pompe Disease, Pompe Disease (Late-onset), Glycogen Storage Disease Type 2, Glycogen Storage Disease Type II
Spark Therapeutics, Inc.
Phase 1/2
4
Arizona, California, Georgia, Kansas, Minnesota, Oregon, Pennsylvania, Utah
2020-10-01
Active, closed
ZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y.o. w/LOPD
NCT03911505Pompe Disease (Late-onset)
Amicus Therapeutics
Phase 3
21
Florida, Georgia, Missouri, North Carolina, Ohio, Pennsylvania, Utah, Virginia
2020-02-13
Recruiting
Pompe Pregnancy Sub-Registry
NCT00567073Glycogen Storage Disease Type II (GSD-II), Pompe Disease (Late-onset), Glycogenesis 2 Acid Maltase Deficiency
Genzyme, a Sanofi Company
Observational
20
Arizona, Florida, Georgia, Indiana, Iowa, Michigan, Missouri, New York
2007-06-18
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1
Cite this view

Priya Life Science (2026). Clinical trial register: industry-sponsored studies with a US investigative site, filtered. Derived from ClinicalTrials.gov. Retrieved 18 September 2026. https://priyalifescience.com/clinical-trials-usa/condition/pompe-disease-late-onset

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Listing here is informational and not medical advice; eligibility is determined by each study team. Compare pay for the teams running these studies in the US Pharma Salary Guide, see who's behind them in the US Company Directory, or track approvals on the FDA Approvals Tracker. For Irish, UK and EU sites, see the Ireland, UK and EU registers.