Summary

Location
at Irvine, California and other locations
Dates
study started
completion around

Description

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

Keywords

Pompe Disease, Glycogen Storage Disease Type II, Miglustat, Cipaglucosidase alfa, Alglucosidase alfa or Avalglucosidase alfa, Untreated

Eligibility

You can join if…

  • Diagnosis of LOPD or IOPD based on documented deficiency of GAA enzyme activity and/or GAA genotyping

You CAN'T join 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)

Locations

  • University of California Irvine not yet accepting patients
    Irvine California 92697 United States
  • University of Arkansas Medical Science accepting new patients
    Little Rock Arkansas 72205 United States
  • Indiana University, IU Health Physicians Neurology accepting new patients
    Indianapolis Indiana 46202 United States

Details

Status
accepting new patients at some sites,
but this study is not currently recruiting here
Start Date
Completion Date
(estimated)
Sponsor
Amicus Therapeutics
ID
NCT06121011
Study Type
Observational [Patient Registry]
Participants
Expecting 500 study participants
Last Updated