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โ† Trials
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A Global Prospective Observational Registry of Patients With Pompe Disease

Recruitingn/aAges any - anyAllAmicus Therapeutics
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In plain words

What the trial is, and whether it could suit you

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

Sign in to get the eligibility rules as simple questions to take to your doctor.

๐Ÿ“‹ Eligibility rules (as published)
Inclusion Criteria: * Diagnosis of LOPD or IOPD based on documented deficiency of GAA enzyme activity and/or GAA genotyping Exclusion Criteria: * Patients who are currently receiving investigational therapy for Pompe disease in a clinical trial, a compassionate use program, or an expanded access program (EAP)
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What it tests

  • Cipaglucosidase alfa
  • Miglustat
  • Alglucosidase alfa or Avalglucosidase alfa
  • Untreated
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Where

41 sites
University of Arkansas Medical Science
Little Rock, United States
University of California Irvine
Irvine, United States
Wolfson Children's Hospital
Jacksonville, United States
Emory University
Atlanta, United States
Indiana University, IU Health Physicians Neurology
Indianapolis, United States
Washington University School of Medicine
St Louis, United States
NYU Neurogenetics, NYU Langone Medical Center
New York, United States
Duke University Medical Center
Durham, United States

โ€ฆand 33 more on ClinicalTrials.gov.

๐Ÿค Taking part is your choice, and you can leave at any time. Only the trial team can confirm you are eligible โ€” always decide together with your own doctor.
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