A Study to Assess the Long-term Safety and Efficacy of ATB200/AT2221 in Adult Subjects With Late-Onset Pompe Disease (LOPD)

ClinicalTrials.gov ID: NCT04138277

Public ClinicalTrials.gov record NCT04138277. 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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Official title

A Phase 3 Open-label Extension Study to Assess the Long-term Safety and Efficacy of Intravenous ATB200 Co-administered With Oral AT2221 in Adult Subjects With Late-onset Pompe Disease (LOPD)

Study identification

NCT ID: NCT04138277
Recruitment status: Completed
Study type: Interventional
Phase: Phase 3
Lead sponsor: Amicus Therapeutics; Industry
Enrollment: 119 participants

Conditions and interventions

Conditions

Pompe Disease (Late-onset)

Interventions

AT2221 Drug
ATB200 Biological

Drug · Biological

Eligibility (public fields only)

Age range: 18 Years and older
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: Dec 17, 2019
Primary completion: Dec 30, 2024
Completion: Dec 30, 2024
Last update posted: Mar 4, 2026

2019 – 2024

United States locations

U.S. sites: 18
U.S. states: 14
U.S. cities: 17

Facility	City	State	ZIP	Site status
University of Arkansas for Medical Sciences	Little Rock	Arkansas	72205	—
University of California, Irvine	Irvine	California	92868	—
University of Florida Clinical Research Center	Gainesville	Florida	32610	—
Emory Clinic	Atlanta	Georgia	30322	—
IU Health Neuroscience Center	Indianapolis	Indiana	46202	—
Washington University School of Medicine	St Louis	Missouri	63110	—
Billings Clinic	Billings	Montana	59101	—
Hackensack University Medical Center	Hackensack	New Jersey	07061	—
Northwell Health	Great Neck	New York	11021	—
NYU School of Medicine	New York	New York	10017	—
Duke University Medical Center	Durham	North Carolina	27710	—
University of Cincinnati Gardner Neuroscience Institute	Cincinnati	Ohio	45219	—
Cincinnati Children's Hospital	Cincinnati	Ohio	45229	—
The Ohio State University Wexner Medical Center	Columbus	Ohio	43210	—
University of Pennsylvania	Philadelphia	Pennsylvania	19104	—
UPMC Montefiore Clinical and Translational Research Center	Pittsburgh	Pennsylvania	15213	—
University of Utah	Salt Lake City	Utah	84108	—
Lysosomal and Rare Disorders Research and Treatment Center, Inc.	Fairfax	Virginia	22030	—

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 42 non-U.S. sites.

About this trial record page

What this page shows: Public field values for ClinicalTrials.gov record NCT04138277, 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 4, 2026 · Synced May 20, 2026

Open the official record

The complete protocol, eligibility criteria, and contact information for NCT04138277 live on ClinicalTrials.gov.

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