Four-Year study tests promising treatment for rare Muscle-Weakening disease

NCT ID NCT04138277

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

Summary

This study followed 119 adults with late-onset Pompe disease for up to four years to see how safe and effective the treatment ATB200/AT2221 is over the long term. Participants had already completed an earlier study and received regular intravenous infusions plus oral medication. Researchers measured walking ability, breathing function, muscle strength, and quality of life to see if benefits were maintained.

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.

Get updates

Get notified about this study

Our safety recommendation!

By submitting, you agree to our Terms of use

Contacts and locations

Show contact details

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

Napoli, 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

Conditions

Explore the condition pages connected to this study.

POMPE DISEASE (LATE-ONSET)