A Global Prospective Observational Registry of Patients With Pompe Disease

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

NCT Number	NCT06121011
Lead Sponsor	Amicus Therapeutics
Conditions	Pompe Disease
Enrollment	500 participants
Start Date	2024-02-16
Primary Completion	2034-12-20 (estimated)
Study Completion	2034-12-20 (estimated)
Updated on ClinicalTrials.gov	2026-03-10