HomeTrialNCT06121011
🔬NCT06121011RECRUITING

A Global Prospective Observational Registry of Patients With Pompe Disease

Study Summary

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

Conditions
View on ClinicalTrials.gov ↗
Study Details
Enrollment500 participants
Primary Completion
Lead SponsorAmicus Therapeutics
Data Retrieved2026-04-11 04:14:12.640555+00:00
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