Clinical Trial: Extension Study of Patients With Infantile-Onset Pompe Disease Who Were Previously Enrolled in Protocol AGLU01602

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

Official Title: A Long-Term Continuation Study of Patients With Infantile-Onset Pompe Disease Who Were Previously Enrolled in Protocol AGLU01602

Brief Summary: Pompe disease (also known as glycogen storage disease type 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. The overall objective of this study is to evaluate the long-term safety and efficacy of Myozyme treatment in patients with infantile-onset Pompe disease.

Detailed Summary:
Sponsor: Genzyme, a Sanofi Company

Current Primary Outcome: Long-term Safety and Efficacy [ Time Frame: 52 weeks ]

Original Primary Outcome:

Current Secondary Outcome:

Original Secondary Outcome:

Information By: Sanofi

Dates:
Date Received: August 1, 2005
Date Started: June 2005
Date Completion:
Last Updated: February 4, 2014
Last Verified: February 2014