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

Completed · Phase 3

Conditions studied: Pompe Disease (Late-onset)

In brief

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.

Key facts

Study ID

NCT04138277

Run by

Amicus Therapeutics

People needed

119

Starts

2019-12-18

Expected to finish

2024-12-31

Last updated by the study team

2026-03-05

Who can join

Age: 18 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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.

You may not qualify if…

• Subject plans to receive gene therapy or participate in another interventional study for Pompe disease.
• Subject, if female, is pregnant or breastfeeding.
• Subject, whether male or female, is planning to conceive a child during the study.
• 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.

Where it is running

• 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
• Duke University Medical Center — Durham, North Carolina, United States
• University of Cincinnati Gardner Neuroscience Institute — Cincinnati, Ohio, United States
• Cincinnati Children's Hospital — Cincinnati, Ohio, United States
• The Ohio State University Wexner Medical Center — Columbus, Ohio, United States
• University of Pennsylvania — Philadelphia, Pennsylvania, United States
• UPMC Montefiore Clinical and Translational Research Center — Pittsburgh, Pennsylvania, United States
• University of Utah — Salt Lake City, Utah, United States
• Lysosomal and Rare Disorders Research and Treatment Center, Inc. — Fairfax, Virginia, United States
• Hospital Universitario Austral — Buenos Aires, Argentina
• Westmead Hospital — Westmead, New South Wales, Australia
• Royal Brisbane & Women's Hospital — Brisbane, Queensland, Australia
• Royal Adelaide Hospital — Adelaide, South Australia, Australia
• Monash Medical Centre — Clayton, Victoria, Australia
• Medizinische Universität Innsbruck — Innsbruck, Austria
• UZ Leuven — Leuven, Belgium
• University of Arkansas for Medical Sciences — Little Rock, Arkansas, United States

Full record on ClinicalTrials.gov

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