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

NCT ID NCT04138277

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

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.

Disclaimer Read more

This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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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

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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