NCT00765414

Extension Study of Long-term Safety and Efficacy of Myozyme for a Single Patient With Pompe Disease Who Were Previously Enrolled in Genzyme Sponsored ERT Studies.

An Open-Label Extension Study of the Long-Term Safety and Efficacy of Recombinant Human Acid α-Glucosidase (rhGAA) Given as Enyzme Replacement Therapy to a Single Patient 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: 1 (actual)
• Sponsor: Genzyme, a Sanofi Company · Industry
• Sex: Female
• Age: 16 Years
• Healthy volunteers: Not accepted

Summary

This extension study was to monitor the long-term safety and efficacy of rhGAA treatment in a single patient with infantile-onset Pompe disease who were previously treated with rhGAA in a Genzyme study.

Conditions

• Pompe Disease Late-Onset
• Glycogen Storage Disease Type II GSD II

Interventions

Type	Name	Description
BIOLOGICAL	Myozyme	30 mg/kg qow f and 40 mg/kg qow

Timeline

• Start date: 2003-04-01
• Primary completion: 2006-04-01
• Completion: 2006-08-01
• First posted: 2008-10-03
• Last updated: 2014-02-05

Locations

1 site across 1 country: United States