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New drug cocktail aims to boost walking and breathing in pompe patients

NCT ID NCT03729362

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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 26, 2026 · Last updated Jun 26, 2026

Summary

This phase 3 trial tested a new treatment for adults with late-onset Pompe disease, a rare genetic disorder that weakens muscles and breathing. Participants received either the experimental combo (cipaglucosidase alfa plus miglustat) or the current standard therapy (alglucosidase alfa plus placebo). The study measured how far patients could walk in 6 minutes and their lung function after 52 weeks. The goal is to see if the new combo works better at preserving mobility and breathing.

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 (stabilizer)
What this could lead to
If successful, this combination could improve walking distance and breathing function for adults with late-onset Pompe disease, offering a more effective treatment option.
What could go wrong
This is a phase 3 trial, but results may not show significant benefit over existing therapy. Side effects from the infusion or oral drug are possible, and long-term outcomes are not yet known.

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

125 people

The number who actually took part.

Started

Dec 2018

Finished

Jan 2021

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 provide signed informed consent prior to any study-related procedures being performed. 2. Male and female subjects are ≥ 18 years old and weigh ≥ 40 kg at screening. 3. Female subjects of childbearing potential and male subjects must agree to use medically accepted methods of contraception during the study and for 90 days after the last dose of study drug. 4. Subject must have a diagnosis of LOPD based on documentation of one of the following: 1. deficiency of GAA enzyme 2. GAA genotyping 5. Subject is classified as one of the following with respect to ERT status: 1. ERT-experienced, defined as currently receiving standard of care ERT (alglucosidase alfa) at the recommended dose and regimen (ie, 20 mg/kg dose every 2 weeks) for ≥ 24 months 2. ERT-naïve, defined as never having received investigational or commercially available ERT 6. Subject has a sitting FVC ≥ 30% of the predicted value for healthy adults (National Health and Nutrition Examination Survey III) at screening. 7. Subject performs two 6MWTs at screening that are valid, as determined by the clinical evaluator, and that meet all of the following criteria: 1. both screening values of 6MWD are ≥ 75 meters 2. both screening values of 6MWD are ≤ 90% of the predicted value for healthy adults 3. the lower value of 6MWD is within 20% of the higher value of 6MWD Exclusion Criteria 1. Subject has received any investigational therapy or pharmacological treatment for Pompe disease, other than alglucosidase alfa, within 30 days or 5 half-lives of the therapy or treatment, whichever is longer, before Day 1 or is anticipated to do so during the study. 2. Subject has received gene therapy for Pompe disease 3. Subject is taking any of the following prohibited medications within 30 days before Day 1: * miglitol (eg, Glyset) * miglustat (eg, Zavesca) * acarbose (eg, Precose or Glucobay) * voglibose (eg, Volix, Vocarb, or Volibo) Note: None of these medications have a half-life that, when multiplied by 5, is longer than 30 days. 4. Subject requires the use of invasive or noninvasive ventilation support for \> 6 hours per day while awake. 5. Subject has a hypersensitivity to any of the excipients in ATB200, alglucosidase alfa, or AT2221. 6. Subject has a medical condition or any other extenuating circumstance that may, in the opinion of the investigator or medical monitor, pose an undue safety risk to the subject or may compromise his/her ability to comply with or adversely impact protocol requirements. This includes clinical depression (as diagnosed by a psychiatrist or other mental health professional) with uncontrolled or poorly controlled symptoms. 7. Subject, if female, is pregnant or breastfeeding at screening. 8. Subject, whether male or female, is planning to conceive a child during the study. 9. Subject does not have documentation of diagnosis of Pompe disease and refuses to undergo genetic testing.

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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 Universitets Hospital

    Aarhus N, 8200, Denmark

  • Billings Clinic

    Billings, Montana, 59101, United States

  • Cambridge University Hospitals

    Cambridge, United Kingdom

  • Centrum Medyczne

    Rzeszów, 35-326, Poland

  • Cincinnati Children's Hospital Medical Center

    Cincinnati, Ohio, 45229, United States

  • Duke University Medical Center

    Durham, North Carolina, 27710, United States

  • Eginition Hospital

    Athens, Attica, 11528, Greece

  • Emory Clinic

    Atlanta, Georgia, 30322, United States

  • Erasmus MC

    Rotterdam, 3015GD, Netherlands

  • Friedrich-Baur Institut

    Munich, Bavaria, 80336, Germany

  • Fukuoka University Hospital

    Fukuoka, 814-0180, Japan

  • Hackensack University Medical Center

    Hackensack, New Jersey, 07601, United States

  • Heritage Medical Research Clinic, University of Calgary

    Calgary, Alberta, T2N 4Z6, Canada

  • Hokkaido University Hospital

    Sapporo, Hokkaido, 060 8648, Japan

  • Hospital Universitario Austral

    Buenos Aires, B1629ODT, Argentina

  • Hospital de la Santa Creu I Sant Pau

    Barcelona, 08026, Spain

  • Hôpital Neurologique Pierre Wertheimer

    Bron, 69677, France

  • Hôpital Pasteur

    Nice, 06001, France

  • Hôpital Raymond Poincaré

    Garches, 92380, France

  • Hôpital Salengro

    Lille, 59037, France

  • Hôpital de la Timone

    Marseille, 13385, France

  • Indiana University Health Neuroscience Center

    Indianapolis, Indiana, 46202, United States

  • Izumi City General Hospital

    Osaka, Japan

  • Kagoshima University Hospital

    Kagoshima, Japan

  • Lysosomal and Rare Disorders Research

    Fairfax, Virginia, 22030, United States

  • McMaster University Medical Centre

    Hamilton, Ontario, L8N 3Z5, Canada

  • Medizinische Universität Innsbruck

    Innsbruck, Austria

  • Monash Medical Centre

    Melbourne, Victoria, 3168, Australia

  • NYU School of Medicine

    New York, New York, 10017, United States

  • National Center of Neurology and Psychiatry

    Tokyo, Japan

  • National Taiwan University Hospital

    Taipei, 100, Taiwan

  • Neuromuscular Research Center

    Phoenix, Arizona, 85028, United States

  • Oregon Health & Science University

    Portland, Oregon, 97239, United States

  • Penn State Health Milton S. Hershey Medical Center

    Hershey, Pennsylvania, 17033, United States

  • Pusan National University

    Yangsan, Gyeongsangnam-do, 50612, South Korea

  • Queen Elizabeth Hospital Birmingham

    Birmingham, United Kingdom

  • Rigshospitalet Copenhagen Neuromuscular Center

    Copenhagen, 2100, Denmark

  • Royal Adelaide Hospital

    Adelaide, South Australia, 5000, Australia

  • Royal Brisbane & Women's Hospital

    Herston, Queensland, 4029, Australia

  • Royal Free Hospital NHS

    London, NW3 2QG, United Kingdom

  • Sahlgrenska University Hospital

    Gothenburg, 41345, Sweden

  • Salford Royal NHS Foundation Trust

    Salford, M6 8HD, United Kingdom

  • Semmelweis University, Institute of Genomic Medicine and Rare Disease

    Budapest, 1083, Hungary

  • Seoul National University Hospital

    Seoul, 03080, South Korea

  • Szpital Uniwersytecki w Krakowie

    Małogoskie, 31-066, Poland

  • The Feinstein Institute for Medical Research

    Manhasset, New York, 11030, United States

  • The Jikei University Hospital

    Tokyo, 105-8471, Japan

  • The Ohio State University Wexner Medical Center

    Columbus, Ohio, 43210, United States

  • UF Helath: University of Florida Clinical Research Center

    Gainesville, Florida, 32610, United States

  • UMHAT Alexandrovska

    Sofia, Bulgaria

  • UOC Genetica Medica

    Naples, NAP, 80131, Italy

  • UOC di Neurologia e Malattie Neuromuscolari

    Messina, NAP, 98125, Italy

  • UZ Leuven

    Leuven, 3000, Belgium

  • University Clinical Centre of the Republic of Srpska

    Banja Luka, 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, New Zealand

  • University of California, Irvine

    Irvine, California, 92868, United States

  • University of Cincinnati Neurology

    Cincinnati, Ohio, 45219, United States

  • University of Kansas Medical Center

    Kansas City, Kansas, 66205, United States

  • University of Minnesota Clinical Research Unit

    Minneapolis, Minnesota, 55455, United States

  • University of Pennsylvania

    Philadelphia, Pennsylvania, 19104, United States

  • University of Pittsburgh

    Pittsburgh, Pennsylvania, 15213, United States

  • University of Pécs

    Pécs, 7623, Hungary

  • University of South Florida Research Center

    Tampa, Florida, 33612, United States

  • University of Szeged

    Szeged, 6725, Hungary

  • University of Texas Health Science Center San Antonio

    San Antonio, Texas, 78229, United States

  • University of Utah, Center for Clinical and Translational Sciences

    Salt Lake City, Utah, 84108, United States

  • Universitätsklinikum Bonn

    Bonn, North Rhine-Westphalia, 53105, Germany

  • Universitätsklinikum Halle (Saale)

    Halle, Saxony-Anhalt, 06120, Germany

  • Universitätsklinikum Münster

    Münster, North Rhine-Westphalia, 48149, Germany

  • Washington University School of Medicine

    St Louis, Missouri, 63110, United States

  • Westmead Hospital

    Westmead, 2145, Australia

More trials for these conditions

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