New hope for pompe disease: advanced Two-Drug therapy tested in major trial

NCT ID NCT03729362

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

Summary

This study tested a new treatment for adults with late-onset Pompe disease, a rare genetic disorder that weakens muscles. The treatment combines an intravenous drug (ATB200) with an oral pill (AT2221). Researchers compared this new combination to the standard enzyme replacement therapy over one year to see if it was better at improving walking ability, breathing, and muscle strength.

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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Contacts and locations

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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

Conditions

Explore the condition pages connected to this study.

POMPE DISEASE (LATE-ONSET)