A Global Prospective Observational Registry of Patients With Pompe Disease
Recruiting now
Conditions studied: Pompe Disease
In brief
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 facts
- Study ID
- NCT06121011
- Run by
- Amicus Therapeutics
- People needed
- 500
- Starts
- 2024-02-16
- Expected to finish
- 2034-12-20
- Last updated by the study team
- 2026-03-10
Who can join
Age: any. Sex: any. Healthy volunteers: not accepted.
You may qualify if…
- Diagnosis of LOPD or IOPD based on documented deficiency of GAA enzyme activity and/or GAA genotyping
You may not qualify if…
- Patients who are currently receiving investigational therapy for Pompe disease in a clinical trial, a compassionate use program, or an expanded access program (EAP)
Where it is running
- University of Pennsylvania Perelman Center for Advanced Medicine — Philadelphia, Pennsylvania, United States (enrolling)
- University of California Irvine — Irvine, California, United States (enrolling)
- Wolfson Children's Hospital — Jacksonville, Florida, United States (enrolling)
- Emory University — Atlanta, Georgia, United States (enrolling)
- Indiana University, IU Health Physicians Neurology — Indianapolis, Indiana, United States (enrolling)
- Washington University School of Medicine — St Louis, Missouri, United States (enrolling)
- NYU Neurogenetics, NYU Langone Medical Center — New York, New York, United States (enrolling)
- Duke University Medical Center — Durham, North Carolina, United States (enrolling)
- University of Cincinnati Medical Center — Cincinnati, Ohio, United States (enrolling)
- Cincinnati Children's Hospital — Cincinnati, Ohio, United States (enrolling)
- University of Arkansas Medical Science — Little Rock, Arkansas, United States (enrolling)
- University of Pittsburgh — Pittsburgh, Pennsylvania, United States (enrolling)
- UPMC Children's Hospital of Pittsburgh — Pittsburgh, Pennsylvania, United States (enrolling)
- University of Utah — Salt Lake City, Utah, United States (enrolling)
- Lysosomal and Rare Disorders Research and Treatment Center, Inc. — Fairfax, Virginia, United States (enrolling)
- Ruhr-Universität Bochum im St. Josef-Hospital — Bochum, Germany (enrolling)
- SphinCS, Institute of Clinical Science in LSD — Höchheim, Germany (enrolling)
- Universitätsklinikum Gießen und Marburg GmhH — Marburg, Germany (enrolling)
- Universitat Munchen - Friedrich Baur Institut — München, Germany (enrolling)
- Queen Elizabeth Hospital Birmingham — Birmingham, United Kingdom (enrolling)
- Cambridge University - Addenbrooke's Hospital — Cambridge, United Kingdom (enrolling)
- University Hospital of Wales, Cardiff — Cardiff, United Kingdom (enrolling)
- University Hospital of Wales — Cardiff, United Kingdom (enrolling)
- National Hospital for Neurology and Neurosurgery — London, United Kingdom (enrolling)
- Great Ormond Street Hospital NHS Foundation Trust — London, United Kingdom (enrolling)
Full record on ClinicalTrials.gov
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