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

COMPLETEDPHASE3

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

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)

NCT04138277•Amicus Therapeutics

View on ClinicalTrials.gov

Summary

This is a multicenter, international open-label extension study of ATB200/AT2221 in adult subjects with late-onset Pompe disease (LOPD) who completed Study ATB200-03.

Detailed Description

This is an open-label extension study for subjects who completed the ATB200-03 study. The subjects will stay in this study until regulatory approval or marketing authorization and/or commercialization in the participating subject's country.

Eligibility

Age

18 - No limit years

Sex

ALL

Healthy Volunteers

Inclusion Criteria

•1\. Subject must have completed Study ATB200-03.
•Note: Subjects who were forced to withdraw from Study ATB200-03 for a logistical reason not related to the efficacy or safety of cipaglucosidase alfa/miglustat (eg, hospitalization for a car accident, COVID-19 pandemic, or emergency surgery) that resulted in several consecutive missed doses may have been eligible to participate in this study upon approval by the Amicus medical monitor.

Exclusion Criteria

•1. Subject plans to receive gene therapy or participate in another interventional study for Pompe disease.
•2. Subject, if female, is pregnant or breastfeeding.
•3. Subject, whether male or female, is planning to conceive a child during the study.
•4. Subject had a hypersensitivity to any of the excipients in cipaglucosidase alfa or miglustat, or had a medical condition or any other extenuating circumstance that may have, in the opinion of the investigator or medical monitor, posed an undue safety risk to the subject or may have compromised his/her ability to comply with or adversely impacted protocol requirements.

Locations (60)

University of Arkansas for Medical Sciences

Little Rock, Arkansas, United States

University of California, Irvine

Irvine, California, United States

University of Florida Clinical Research Center

Gainesville, Florida, United States

Emory Clinic

Atlanta, Georgia, United States

IU Health Neuroscience Center

Indianapolis, Indiana, United States

Washington University School of Medicine

St Louis, Missouri, United States

Billings Clinic

Billings, Montana, United States

Hackensack University Medical Center

Hackensack, New Jersey, United States

Northwell Health

Great Neck, New York, United States

NYU School of Medicine

New York, New York, United States

Key Dates

Start Date

December 18, 2019

Primary Completion Date

December 31, 2024

Completion Date

December 31, 2024

Last Updated

March 5, 2026

Enrollment

119

ACTUAL participants

Conditions

Pompe Disease (Late-onset)

Interventions

AT2221

DRUG

ATB200

BIOLOGICAL

ZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y.o. w/LOPD

NCT03911505

Pompe Disease (Late-onset)

View study

ACTIVE_NOT_RECRUITING

Developing a Management Approach for Patients With "Late-Onset" Pompe Disease

NCT03694561

Pompe DiseasePompe Disease (Late-onset)+1 more

View study

Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: March 5, 2026Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

View ClinicalTrials.gov Terms and Conditions

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

Inclusion Criteria

Exclusion Criteria

ZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y.o. w/LOPD

Developing a Management Approach for Patients With "Late-Onset" Pompe Disease

A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE)