Pompe Disease — Trials, FDA Actions & Pivotal Research

12 active Pompe Disease trials currently recruiting in the US, sourced from ClinicalTrials.gov.

Recruiting trials by city

Cities where Pompe Disease trials in our archive had recruiting sites. Each link lists every currently recruiting paid clinical trial at that location.

Birmingham, AL Irvine, CA Little Rock, AR

Active Pompe Disease trials

Currently recruiting US trials matching Pompe Disease, sourced live from ClinicalTrials.gov. We’ll add per-event articles here as those trials post results or hit primary completion.

A Study of S-606001 as Monotherapy in Participants With Late-onset Pompe Disease (LOPD)

for Pompe Disease

Sponsor: Shionogi

Sex: All·Age: 18+

Code: NCT07787650·Phase 1·Not Yet Recruiting·Listed Aug 26, 2026

An Extension Study of S-606001 in Participants With Late-onset Pompe Disease (LOPD)

for Pompe Disease

Sponsor: Shionogi

Sex: All·Age: 18+

Code: NCT07750990·Phase 2·Not Yet Recruiting·Listed Aug 2026

Phrenic Nerve and Diaphragm Electrophysiology in Pompe Disease

for Pompe Disease

Sponsor: IRCCS National Neurological Institute "C. Mondino" Foundation

Sex: All·Age: 18+

Code: NCT07664930·Recruiting·Listed Jun 2026

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Study of S-606001 as an Add-on to Enzyme Replacement Therapy (ERT) in Participants With Late-onset Pompe Disease (LOPD)

for Pompe Disease

9 locations incl. Irvine

Sponsor: Shionogi

Sex: All·Age: 18+

Code: NCT07123155·Phase 2·Recruiting·Listed Aug 2025

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Development and Validation of the FBIndex to Determine the Risk of Falls for Patients With Neuromuscular Disorders

for Inclusion Body Myositis

Sponsor: LMU Klinikum

Sex: All·Age: 18 - 65

Code: NCT06605612·Enrolling By Invitation·Listed Sep 2024

A Global Prospective Observational Registry of Patients With Pompe Disease

for Pompe Disease

15 locations incl. Little Rock

Sponsor: Amicus Therapeutics

Sex: All·Age: All ages

Code: NCT06121011·Recruiting·Listed Nov 2023

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Pompe Disease RWE Long-term Monitoring Program

for Pompe Disease

Sponsor: National Taiwan University Hospital

Sex: All·Age: All ages

Code: NCT07834320·Recruiting·Listed Sep 22, 2026

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Avalglucosidase Alfa Pregnancy Study

for Pompe Disease

1 location in Bridgewater

Sponsor: Sanofi

Sex: Female·Age: All ages

Code: NCT05734521·Recruiting·Listed Feb 2023

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Cognitive and Neurological Pathologies in Pompe Disease

for Pompe Disease

1 location in Durham

Sponsor: Duke University

Sex: All·Age: 0 - 18

Code: NCT04639336·Recruiting·Listed Nov 2020

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Determination of CRIM Status and Longitudinal Follow-up of Individuals With Pompe Disease

for Pompe Disease

1 location in Durham

Sponsor: Duke University

Sex: All·Age: All ages

Code: NCT01665326·Recruiting·Listed Aug 2012

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A Long-term Study for the Outcome of Pompe Disease

for Pompe Disease

Sponsor: National Taiwan University Hospital

Sex: All·Age: All ages

Code: NCT02399748·Recruiting·Listed Mar 2015

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Pompe Disease Registry Protocol

for Glycogen Storage Disease Type II

74 locations incl. Birmingham

Sponsor: Genzyme, a Sanofi Company

Sex: All·Age: All ages

Code: NCT00231400·Recruiting·Listed Oct 2005

Apply to this trial