NCT00455195

Late-Onset Treatment Study Extension Protocol

An Open-Label Extension Study of Patients With Late-Onset Pompe Disease Who Were Previously Enrolled in Protocol AGLU02704

• Status: Completed
• Phase: Phase 4
• Study type: Interventional
• Enrollment: 81 (actual)
• Sponsor: Genzyme, a Sanofi Company · Industry
• Sex: All
• Age: 8 Years
• 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 objective of this extension study is to assess the long-term safety and efficacy of alglucosidase alfa treatment in patients with Late-Onset Pompe Disease who were previously treated under the placebo-controlled, double-blind study AGLU02704 (NCT00158600).

Conditions

• Pompe Disease (Late-Onset)
• Glycogen Storage Disease Type II (GSD-II)
• Glycogenesis Type II
• Acid Maltase Deficiency (AMD)

Interventions

Type	Name	Description
BIOLOGICAL	alglucosidase alfa	IV infusion of 20 mg/kg; every other week (qow)

Timeline

• Start date: 2007-03-01
• Primary completion: 2008-10-01
• Completion: 2008-11-01
• First posted: 2007-04-03
• Last updated: 2014-03-07
• Results posted: 2012-01-05

Locations

31 sites across 5 countries: United States, Australia, Canada, France, Netherlands