Condition: Pompe Disease · Sponsor: Amicus Therapeutics
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
This description comes directly from the study's public registry record.
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Always discuss trial participation with your own doctor first.
| University of Arkansas Medical Science | Little Rock, Arkansas, United States | Recruiting |
| University of California Irvine | Irvine, California, United States | Recruiting |
| Wolfson Children's Hospital | Jacksonville, Florida, United States | Recruiting |
| Emory University | Atlanta, Georgia, United States | Recruiting |
| Indiana University, IU Health Physicians Neurology | Indianapolis, Indiana, United States | Recruiting |
| Washington University School of Medicine | St Louis, Missouri, United States | Recruiting |
| NYU Neurogenetics, NYU Langone Medical Center | New York, New York, United States | Recruiting |
| Duke University Medical Center | Durham, North Carolina, United States | Recruiting |
| University of Cincinnati Medical Center | Cincinnati, Ohio, United States | Recruiting |
| Cincinnati Children's Hospital | Cincinnati, Ohio, United States | Recruiting |
| University of Pennsylvania Perelman Center for Advanced Medicine | Philadelphia, Pennsylvania, United States | Recruiting |
| University of Pittsburgh | Pittsburgh, Pennsylvania, United States | Recruiting |
+ 29 more locations — full list on the registry record.
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Source record: clinicaltrials.gov/study/NCT06121011