Pompe Disease — Trials, FDA Actions & Pivotal Research

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

Synced daily from openFDA and ClinicalTrials.gov. Last sync: July 6, 2026.

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.

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

Apply to this trial

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

Apply to this trial

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

FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases

for Alpha-Thalassemia

1 location in Los Altos

Sponsor: xCures

Sex: All·Age: All ages

Code: NCT06539169·Recruiting

Apply to this trial

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

Apply to this trial

Avalglucosidase Alfa Pregnancy Study

for Pompe Disease

1 location in Bridgewater

Sponsor: Sanofi

Sex: Female·Age: All ages

Code: NCT05734521·Recruiting

Apply to this trial

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

Apply to this trial

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

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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

Apply to this trial

Pompe Disease Registry Protocol

for Glycogen Storage Disease Type II

92 locations incl. Birmingham

Sponsor: Genzyme, a Sanofi Company

Sex: All·Age: All ages

Code: NCT00231400·Recruiting

Apply to this trial