Status:
RECRUITING
A Global Prospective Observational Registry of Patients With Pompe Disease
Lead Sponsor:
Amicus Therapeutics
Conditions:
Pompe Disease
Eligibility:
All Genders
Brief 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 u...
Eligibility Criteria
Inclusion
- Diagnosis of LOPD or IOPD based on documented deficiency of GAA enzyme activity and/or GAA genotyping
Exclusion
- Patients who are currently receiving investigational therapy for Pompe disease in a clinical trial, a compassionate use program, or an expanded access program (EAP)
Key Trial Info
Start Date :
February 16 2024
Trial Type :
OBSERVATIONAL
Allocation :
ESTIMATED
End Date :
December 20 2034
Estimated Enrollment :
500 Patients enrolled
Trial Details
Trial ID
NCT06121011
Start Date
February 16 2024
End Date
December 20 2034
Last Update
December 22 2025
Active Locations (42)
Enter a location and click search to find clinical trials sorted by distance.
1
University of Arkansas Medical Science
Little Rock, Arkansas, United States, 72205
2
University of California Irvine
Irvine, California, United States, 92697
3
Wolfson Children's Hospital
Jacksonville, Florida, United States, 32207
4
Emory University
Atlanta, Georgia, United States, 30322