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A Global Prospective Observational Registry of Patients With Pompe Disease
Who can join
All ages · All sexes
Full eligibility criteria
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)
About the study
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
What is being tested
- Cipaglucosidase alfa (biological)
- Miglustat (drug)
- Alglucosidase alfa or Avalglucosidase alfa (biological)
- Untreated (other)
Sponsor: Amicus Therapeutics · Participants: 500 · Started: Feb 16, 2024
Contact the study team
- For Site · Phone: 609-662-2000
- For Patient · Phone: 609-662-2000
Official record on ClinicalTrials.gov — NCT06121011
Locations in the U.S.
Conditions
From ClinicalTrials.gov, data retrieved Sep 30, 2026. Each study sets its own eligibility; the study team decides who can join.