Massive global study launches to track rare muscle disease

NCT ID NCT06121011

Recruiting now Knowledge-focused Sponsor: Amicus Therapeutics Source: ClinicalTrials.gov ↗

Summary

This study is creating a worldwide registry to collect long-term information from 500 people with Pompe disease. It aims to understand how well current treatments work in real life, monitor their safety over time, and learn about the natural course of the disease in untreated patients. The registry will include both people receiving treatment and those who are not.

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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Study contacts

Contact

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Locations

Aarhus Universitets hospital
RECRUITING

Aarhus C, 8000, Denmark
Azienda Ospedaliero Universitaria Consorziale Policlinico di Bari
NOT_YET_RECRUITING

Bari, 70124, Italy
Cambridge University - Addenbrooke's Hospital
RECRUITING

Cambridge, CB20QQ, United Kingdom
Centre of Expertise for muscular diseases and peripheral neuropathies European Reference Network for Rare Neuromuscular Diseases
NOT_YET_RECRUITING

Naples, 80131, Italy
Cincinnati Children's Hospital
RECRUITING

Cincinnati, Ohio, 45229, United States
Department of Neurosciences Rita Levi Montalcini, University of Torino
NOT_YET_RECRUITING

Torino, 10126, Italy
Dept of Pediatrics Erasmus MC - Sophia Children's Hospital
NOT_YET_RECRUITING

Rotterdam, 3015 GD, Netherlands
Duke University Medical Center
RECRUITING

Durham, North Carolina, 27710, United States
Eginition Hospital
NOT_YET_RECRUITING

Athens, 115228, Greece
Emory University
RECRUITING

Atlanta, Georgia, 30322, United States
Great Ormond Street Hospital NHS Foundation Trust
RECRUITING

London, WC1N 3JH, United Kingdom
Indiana University, IU Health Physicians Neurology
RECRUITING

Indianapolis, Indiana, 46202, United States
Laboratory for Muscle Diseases and Neuropathies
RECRUITING

Leuven, 3000, Belgium
Lysosomal and Rare Disorders Research and Treatment Center, Inc.
RECRUITING

Fairfax, Virginia, 22030, United States
Medizinische Universitaet Wien
NOT_YET_RECRUITING

Vienna, 1090, Austria
NYU Neurogenetics, NYU Langone Medical Center
RECRUITING

New York, New York, 10017, United States
National Hospital for Neurology and Neurosurgery
RECRUITING

London, WC1N 3BG, United Kingdom
Queen Elizabeth Hospital Birmingham
RECRUITING

Birmingham, B15 2TH, United Kingdom
Regional Coordinating Centre for Rare Diseases, university Hospital of Udine, Udine, Italy
NOT_YET_RECRUITING

Udine, 33100, Italy
Royal Free Hospital NHS Foundation Trust
RECRUITING

Manchester, NW3 2QG, United Kingdom
Ruhr-Universität Bochum im St. Josef-Hospital
RECRUITING

Bochum, 44791, Germany
Salford Royal NHS Foundation Trust
RECRUITING

Salford, M6 8HD, United Kingdom
SphinCS, Institute of Clinical Science in LSD
RECRUITING

Höchheim, 265239, Germany
UPMC Children's Hospital of Pittsburgh
RECRUITING

Pittsburgh, Pennsylvania, 15224, United States
Universitaetsklinikum Ulm
NOT_YET_RECRUITING

Ulm, 89081, Germany
Universitat Munchen - Friedrich Baur Institut
RECRUITING

München, 80336, Germany
University Hospital of Wales
RECRUITING

Cardiff, CF14 4XW, United Kingdom
University Hospital of Wales, Cardiff
RECRUITING

Cardiff, CF14 4XW, United Kingdom
University Medical Centre Ljubljana, Institute of Clinical Neurophysiology
NOT_YET_RECRUITING

Ljubljana, 1000, Slovenia
University of Arkansas Medical Science
RECRUITING

Little Rock, Arkansas, 72205, United States
University of California Irvine
RECRUITING

Irvine, California, 92697, United States
University of Cincinnati Medical Center
RECRUITING

Cincinnati, Ohio, 45219, United States
University of Pennsylvania Perelman Center for Advanced Medicine
RECRUITING

Philadelphia, Pennsylvania, 19104, United States
University of Pittsburgh
RECRUITING

Pittsburgh, Pennsylvania, 15219, United States
University of Pécs
NOT_YET_RECRUITING

Pécs, 7622, Hungary
University of Szeged, Szent-Györgyi Albert Clinical Center
NOT_YET_RECRUITING

Szeged, 6722, Hungary
University of Utah
RECRUITING

Salt Lake City, Utah, 84108, United States
Universitätsklinikum Gießen und Marburg GmhH
RECRUITING

Marburg, 35043, Germany
Washington University School of Medicine
RECRUITING

St Louis, Missouri, 63110, United States
Wolfson Children's Hospital
RECRUITING

Jacksonville, Florida, 32207, United States
entrum Medyczne Medyk
NOT_YET_RECRUITING

Rzeszów, 35-055, Poland

Conditions

Explore the condition pages connected to this study.

POMPE DISEASE