Status:
RECRUITING
Pompe Disease Registry Protocol
Lead Sponsor:
Genzyme, a Sanofi Company
Conditions:
Glycogen Storage Disease Type II
Pompe Disease
Eligibility:
All Genders
Brief Summary
The Pompe Registry is a global, multicenter, international, longitudinal, observational, and voluntary program for patients with Pompe disease, designed to track the disease's natural history and outc...
Detailed Description
Study Design Time Perspective: Retrospective and Prospective
Eligibility Criteria
Inclusion
- All patients with a confirmed diagnosis of Pompe disease who have signed the informed consent and authorization form(s) are eligible for inclusion. Confirmed diagnosis is defined as documented GAA enzyme deficiency from blood, skin, or muscle tissue and/or documentation of 2 GAA gene mutations.
Exclusion
- There are no exclusion criteria in this Registry
Key Trial Info
Start Date :
September 15 2004
Trial Type :
OBSERVATIONAL
Allocation :
ESTIMATED
End Date :
January 31 2034
Estimated Enrollment :
2000 Patients enrolled
Trial Details
Trial ID
NCT00231400
Start Date
September 15 2004
End Date
January 31 2034
Last Update
December 1 2025
Active Locations (272)
Enter a location and click search to find clinical trials sorted by distance.
1
University Alabama at Birmingham- Site Number : 840106
Birmingham, Alabama, United States, 35233
2
Barrow Neurological Institute- Site Number : 840087
Phoenix, Arizona, United States, 85013
3
Phoenix Children's Hospital- Site Number : 840003
Phoenix, Arizona, United States, 85013
4
University of Arizona- Site Number : 840015
Tucson, Arizona, United States, 85724