NCT00125879

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

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

• Status: Completed
• Phase: Phase 2 / Phase 3
• Study type: Interventional
• Enrollment: 16 (actual)
• Sponsor: Genzyme, a Sanofi Company · Industry
• Sex: All
• Age: —
• Healthy volunteers: Not accepted

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.

Conditions

• Glycogen Storage Disease Type II

Interventions

Type	Name	Description
BIOLOGICAL	Myozyme	20 mg/kg qow or 40 mg/kg qow

Timeline

• Start date: 2005-06-01
• Primary completion: 2006-06-01
• Completion: 2006-12-01
• First posted: 2005-08-02
• Last updated: 2014-02-06

Locations

14 sites across 7 countries: United States, France, Germany, Israel, Italy, Netherlands, Taiwan