NCT00763932

Extension Study of Long-term Safety and Efficacy of Myozyme in Patients With Pompe Disease Who Were Previously Enrolled in Genzyme Sponsored Enzyme Replacement Therapy (ERT) Studies

A Multicenter, Open-Label Extension Study of the Long-Term Safety and Efficacy of Recombinant Human Acid α-Glucosidase (rhGAA) in Patients With Pompe Disease (Glycogen Storage Disease Type II) Who Were Previously Enrolled in Genzyme-Sponsored Enzyme Replacement Therapy Studies

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 7 (actual)
• Sponsor: Genzyme, a Sanofi Company · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

This extension study was to monitor the long-term safety and efficacy of rhGAA treatment in patients with infantile-onset Pompe disease who were previously treated with rhGAA derived from the Synpac cell line

Conditions

• Pompe Disease Infantile-Onset
• Glycogen Storage Disease Type II

Interventions

Type	Name	Description
BIOLOGICAL	Myozyme	10 mg/kg or 20 mg/kg qw OR 20 mg/kg or 40 mg/kg qow

Timeline

• Start date: 2003-04-01
• Primary completion: 2006-06-01
• Completion: 2006-07-01
• First posted: 2008-10-01
• Last updated: 2014-02-05

Locations

8 sites across 3 countries: United States, France, South Africa