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Pompe disease drug combo shows Long-Term safety in phase 3 trial

NCT ID NCT04138277

Disease control Sponsor: Amicus Therapeutics Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed This study
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This study tested the long-term safety of a combination therapy (ATB200 and AT2221) in 119 adults with late-onset Pompe disease who had completed a prior study. Participants received the drugs for an extended period, and researchers tracked side effects, walking distance, and lung function. The goal was to see if the treatment remains safe and helps maintain motor and breathing abilities over time.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
cipaglucosidase alfa (enzyme replacement therapy) plus miglustat
What this could lead to
If successful, this could confirm a long-term treatment option that helps maintain walking ability and breathing function in adults with late-onset Pompe disease.
What could go wrong
This is an extension study, not a new test of effectiveness. Side effects like infusion reactions or serious adverse events may still occur, and not all patients may benefit equally.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

119 people

The number who actually took part.

Started

Dec 2019

Finished

Dec 2024

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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.

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Conditions

The condition(s) this trial relates to.

glycogen storage disease due to acid maltase deficiency, late-onset glycogen storage disease II Glycogen Storage Disease Type II

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Pompe disease (late-onset)

Contacts and locations

Locations

  • Aarhus Universitetshospital

    Aarhus, Denmark

  • Alberta Children's Hospital, Heritage Medical Research Clinic

    Calgary, Alberta, T3B 6A8, Canada

  • Billings Clinic

    Billings, Montana, 59101, United States

  • Cambridge University Hospitals NHS Foundation Trust

    Cambridge, CB2 0QQ, United Kingdom

  • Centrum Medyczne Medyk

    Rzeszów, Podkarpackie Voivodeship, 35-326, Poland

  • Cincinnati Children's Hospital

    Cincinnati, Ohio, 45229, United States

  • Duke University Medical Center

    Durham, North Carolina, 27710, United States

  • Eginition Hospital

    Athens, 11528, Greece

  • Emory Clinic

    Atlanta, Georgia, 30322, United States

  • Erasmus MC

    Rotterdam, 3015 GD, Netherlands

  • Friedrich-Baur Institut

    München, 80336, Germany

  • Fukuoka University Hospital

    Fukuoka, 814-0180, Japan

  • Hackensack University Medical Center

    Hackensack, New Jersey, 07061, United States

  • Hokkaido University Hospital

    Sapporo, 060 8648, Japan

  • Hospital Universitario Austral

    Buenos Aires, B1629ODT, Argentina

  • Hospital de la Santa Creu I Sant Pau

    Barcelona, Spain

  • Hôpital Pasteur 2

    Nice, 06001, France

  • Hôpital Pierre Wertheimer

    Bron, 69577, France

  • Hôpital Raymond Poincaré

    Garches, 92380, France

  • Hôpital Salengro

    Lille, 59037, France

  • Hôpital de la Timone

    Marseille, 13385, France

  • IU Health Neuroscience Center

    Indianapolis, Indiana, 46202, United States

  • Izumi City General Hospital

    Osaka, 594 0073, Japan

  • Kagoshima University Hospital

    Kagashima, 890-8520, Japan

  • Lysosomal and Rare Disorders Research and Treatment Center, Inc.

    Fairfax, Virginia, 22030, United States

  • McMaster University Medical Centre

    Hamilton, Ontario, L8N 3Z5, Canada

  • Medizinische Universität Innsbruck

    Innsbruck, A-6020, Austria

  • Monash Medical Centre

    Clayton, Victoria, 3168, Australia

  • NYU School of Medicine

    New York, New York, 10017, United States

  • National Taiwan University Hospital

    Taipei, 100, Taiwan

  • Northwell Health

    Great Neck, New York, 11021, United States

  • Pusan National University

    Yangsan, Gyeongsangnam-do, 50612, South Korea

  • Queen Elizabeth Hospital Birmingham

    Birmingham, B15 2TH, United Kingdom

  • Royal Adelaide Hospital

    Adelaide, South Australia, 5000, Australia

  • Royal Brisbane & Women's Hospital

    Brisbane, Queensland, 4029, Australia

  • Royal Free Hospital NHS Foundation Trust

    London, NW3 2QG, United Kingdom

  • Sahlgrenska University Hospital

    Gothenburg, 41345, Sweden

  • Salford Royal NHS Foundation Trust

    Salford, M6 8HD, United Kingdom

  • Semmelweis University

    Budapest, 1085, Hungary

  • The Jikei University Hospital

    Tokyo, 105-8471, Japan

  • The Ohio State University Wexner Medical Center

    Columbus, Ohio, 43210, United States

  • UOC di Neurologia e Malattie Neuromuscolari

    Messina, 98124, Italy

  • UPMC Montefiore Clinical and Translational Research Center

    Pittsburgh, Pennsylvania, 15213, United States

  • UZ Leuven

    Leuven, 3000, Belgium

  • Universitaria Federico II

    Naples, 80131, Italy

  • University Clinical Centre of the Republic of Srpska

    Banja Luka, The Republic of Srpska, 78000, Bosnia and Herzegovina

  • University Medical Centre Ljubljana

    Ljubljana, 1000, Slovenia

  • University of Arkansas for Medical Sciences

    Little Rock, Arkansas, 72205, United States

  • University of Auckland

    Auckland, 1142, New Zealand

  • University of California, Irvine

    Irvine, California, 92868, United States

  • University of Cincinnati Gardner Neuroscience Institute

    Cincinnati, Ohio, 45219, United States

  • University of Florida Clinical Research Center

    Gainesville, Florida, 32610, United States

  • University of Pennsylvania

    Philadelphia, Pennsylvania, 19104, United States

  • University of Pécs

    Pécs, 7623, Hungary

  • University of Szeged

    Szeged, 6725, Hungary

  • University of Utah

    Salt Lake City, Utah, 84108, United States

  • Universitätsklinikum Bonn

    Bonn, 53127, Germany

  • Universitätsklinikum Münster

    Münster, 48149, Germany

  • Washington University School of Medicine

    St Louis, Missouri, 63110, United States

  • Westmead Hospital

    Westmead, New South Wales, Australia

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Other studies related to the condition(s) this trial covers.