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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
Age
All ages
Sex
ALL
Healthy Volunteers
No
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
Start Date
February 16, 2024
Primary Completion Date
December 20, 2034
Completion Date
December 20, 2034
Last Updated
March 10, 2026
500
ESTIMATED participants
Cipaglucosidase alfa
BIOLOGICAL
Miglustat
DRUG
Alglucosidase alfa or Avalglucosidase alfa
BIOLOGICAL
Untreated
OTHER
Lead Sponsor
Amicus Therapeutics
NCT01665326
NCT06109948
Data Source & Attribution
This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.
Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.
Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.
View ClinicalTrials.gov Terms and ConditionsNCT03911505