A Global Prospective Observational Registry of Patients With Pompe Disease

ClinicalTrials.gov ID: NCT06121011

Public ClinicalTrials.gov record NCT06121011. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.

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ClinicalTrials.gov public records Last synced May 20, 2026, 1:05 AM EDT

Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.

Study identification

NCT ID: NCT06121011
Recruitment status: Recruiting
Study type: Observational
Phase: Not listed
Lead sponsor: Amicus Therapeutics; Industry
Enrollment: 500 participants

Conditions and interventions

Conditions

Pompe Disease

Interventions

Alglucosidase alfa or Avalglucosidase alfa Biological
Cipaglucosidase alfa Biological
Miglustat Drug
Untreated Other

Biological · Drug · Other

Eligibility (public fields only)

Age range: Not listed
Sex: All
Healthy volunteers: Healthy volunteers not accepted

This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.

Study timeline

Start date: Feb 15, 2024
Primary completion: Dec 19, 2034
Completion: Dec 19, 2034
Last update posted: Mar 9, 2026

2024 – 2034

United States locations

U.S. sites: 15
U.S. states: 12
U.S. cities: 13

Facility	City	State	ZIP	Site status
University of Arkansas Medical Science	Little Rock	Arkansas	72205	Recruiting
University of California Irvine	Irvine	California	92697	Recruiting
Wolfson Children's Hospital	Jacksonville	Florida	32207	Recruiting
Emory University	Atlanta	Georgia	30322	Recruiting
Indiana University, IU Health Physicians Neurology	Indianapolis	Indiana	46202	Recruiting
Washington University School of Medicine	St Louis	Missouri	63110	Recruiting
NYU Neurogenetics, NYU Langone Medical Center	New York	New York	10017	Recruiting
Duke University Medical Center	Durham	North Carolina	27710	Recruiting
University of Cincinnati Medical Center	Cincinnati	Ohio	45219	Recruiting
Cincinnati Children's Hospital	Cincinnati	Ohio	45229	Recruiting
University of Pennsylvania Perelman Center for Advanced Medicine	Philadelphia	Pennsylvania	19104	Recruiting
University of Pittsburgh	Pittsburgh	Pennsylvania	15219	Recruiting
UPMC Children's Hospital of Pittsburgh	Pittsburgh	Pennsylvania	15224	Recruiting
University of Utah	Salt Lake City	Utah	84108	Recruiting
Lysosomal and Rare Disorders Research and Treatment Center, Inc.	Fairfax	Virginia	22030	Recruiting

Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.

Non-U.S. locations

This page focuses on the U.S. directory. The official record also lists 26 non-U.S. sites.

About this trial record page

What this page shows: Public field values for ClinicalTrials.gov record NCT06121011, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
What this page does not do: No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
Where the data comes from: Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
Last refresh: Last update posted Mar 9, 2026 · Synced May 20, 2026

Open the official record

The complete protocol, eligibility criteria, and contact information for NCT06121011 live on ClinicalTrials.gov.

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