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A Global Prospective Observational Registry of Patients With Pompe Disease
Sponsor: Amicus Therapeutics
Summary
This is a global, multicenter, prospective, observational registry of patients with Pompe disease, including those with late-onset pompe disease (LOPD) and infantile-onset pompe disease (IOPD). Both untreated patients and those being treated with an approved therapy for Pompe disease are eligible to participate. The objectives of the registry are: * To evaluate the long-term safety of Pompe disease treatments through collection of data that describe the frequency of adverse events (AEs)/serious adverse events (SAEs) occurring in Pompe disease patients * To evaluate the long-term real-world effectiveness of Pompe disease treatments * To evaluate the long-term real-world impact of Pompe disease treatments on quality of life (QOL) and patient-reported outcomes (PROs) * To describe the natural history of untreated Pompe disease
Key Details
Gender
All
Age Range
Any - Any
Study Type
OBSERVATIONAL
Enrollment
500
Start Date
2024-02-16
Completion Date
2034-12-20
Last Updated
2026-03-10
Healthy Volunteers
No
Conditions
Interventions
Cipaglucosidase alfa
Enzyme Replacement Therapy (ERT) via intravenous infusion
Miglustat
Participants received ATB200 co-administered with AT2221 (Miglustat)
Alglucosidase alfa or Avalglucosidase alfa
Patients prescribed other commercially available ERT after local regulatory approval
Untreated
Patients who are not currently receiving any medical therapy for Pompe disease.
Locations (41)
University of Arkansas Medical Science
Little Rock, Arkansas, United States
University of California Irvine
Irvine, California, United States
Wolfson Children's Hospital
Jacksonville, Florida, United States
Emory University
Atlanta, Georgia, United States
Indiana University, IU Health Physicians Neurology
Indianapolis, Indiana, United States
Washington University School of Medicine
St Louis, Missouri, United States
NYU Neurogenetics, NYU Langone Medical Center
New York, New York, United States
Duke University Medical Center
Durham, North Carolina, United States
University of Cincinnati Medical Center
Cincinnati, Ohio, United States
Cincinnati Children's Hospital
Cincinnati, Ohio, United States
University of Pennsylvania Perelman Center for Advanced Medicine
Philadelphia, Pennsylvania, United States
University of Pittsburgh
Pittsburgh, Pennsylvania, United States
UPMC Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, United States
University of Utah
Salt Lake City, Utah, United States
Lysosomal and Rare Disorders Research and Treatment Center, Inc.
Fairfax, Virginia, United States
Medizinische Universitaet Wien
Vienna, Austria
Laboratory for Muscle Diseases and Neuropathies
Leuven, Belgium
Aarhus Universitets hospital
Aarhus C, Denmark
Ruhr-Universität Bochum im St. Josef-Hospital
Bochum, Germany
SphinCS, Institute of Clinical Science in LSD
Höchheim, Germany
Universitätsklinikum Gießen und Marburg GmhH
Marburg, Germany
Universitat Munchen - Friedrich Baur Institut
München, Germany
Universitaetsklinikum Ulm
Ulm, Germany
Eginition Hospital
Athens, Greece
University of Pécs
Pécs, Hungary
University of Szeged, Szent-Györgyi Albert Clinical Center
Szeged, Hungary
Azienda Ospedaliero Universitaria Consorziale Policlinico di Bari
Bari, Italy
Centre of Expertise for muscular diseases and peripheral neuropathies European Reference Network for Rare Neuromuscular Diseases
Naples, Italy
Department of Neurosciences Rita Levi Montalcini, University of Torino
Torino, Italy
Regional Coordinating Centre for Rare Diseases, university Hospital of Udine, Udine, Italy
Udine, Italy
Dept of Pediatrics Erasmus MC - Sophia Children's Hospital
Rotterdam, Netherlands
entrum Medyczne Medyk
Rzeszów, Poland
University Medical Centre Ljubljana, Institute of Clinical Neurophysiology
Ljubljana, Slovenia
Queen Elizabeth Hospital Birmingham
Birmingham, United Kingdom
Cambridge University - Addenbrooke's Hospital
Cambridge, United Kingdom
University Hospital of Wales, Cardiff
Cardiff, United Kingdom
University Hospital of Wales
Cardiff, United Kingdom
National Hospital for Neurology and Neurosurgery
London, United Kingdom
Great Ormond Street Hospital NHS Foundation Trust
London, United Kingdom
Royal Free Hospital NHS Foundation Trust
Manchester, United Kingdom
Salford Royal NHS Foundation Trust
Salford, United Kingdom