A Global Prospective Observational Registry of Patients With Pompe Disease
Amicus Therapeutics
500 participants
Feb 16, 2024
OBSERVATIONAL
Conditions
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
Eligibility
Inclusion Criteria1
- Diagnosis of LOPD or IOPD based on documented deficiency of GAA enzyme activity and/or GAA genotyping
Exclusion Criteria1
- 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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Interventions
Enzyme Replacement Therapy (ERT) via intravenous infusion
Participants received ATB200 co-administered with AT2221 (Miglustat)
Patients prescribed other commercially available ERT after local regulatory approval
Patients who are not currently receiving any medical therapy for Pompe disease.
Locations(41)
View Full Details on ClinicalTrials.gov
For the most up-to-date information, visit the official listing.
NCT06121011