Clinical Trial: A Prospective, Observational Study in Patients With Late-Onset Pompe Disease

Study Status: Completed
Recruit Status: Completed
Study Type: Observational

Official Title: A Prospective, Observational Study in Patients With Late-Onset Pompe Disease

Brief Summary:

Pompe disease (also known as glycogen storage disease type II, "GSD-II") is caused by a deficiency of a critical enzyme in the body called acid alpha-glucosidase (GAA). Normally, GAA is used by the body's cells to break down glycogen (a stored form of sugar) within specialized structures called lysosomes. In patients with Pompe disease, an excessive amount of glycogen accumulates and is stored in various tissues, especially heart and skeletal muscle, which prevents their normal function.

This study is being conducted to collect prospective, observational data on patients with late-onset Pompe disease. Approximately 60 subjects with late-onset Pompe disease will be enrolled.

Detailed Summary:
Sponsor: Genzyme, a Sanofi Company

Current Primary Outcome:

Original Primary Outcome:

Current Secondary Outcome:

Original Secondary Outcome:

Information By: Sanofi

Dates:
Date Received: February 10, 2004
Date Started: March 2004
Date Completion:
Last Updated: May 4, 2015
Last Verified: May 2015