NCT00268944

Safety and Effectiveness Study of rhGAA in Patients With Advanced Late-Onset Pompe Disease Receiving Respiratory Support

Prospective, Open-label, Single-arm, Exploratory Study of the Effect and Safety of rhGAA in Patients With Advanced Late-onset Pompe Disease Who Are Receiving Respiratory Support

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 5 (actual)
• Sponsor: Genzyme, a Sanofi Company · Industry
• Sex: All
• Age: 18 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 overall objective is to evaluate the safety and efficacy of rhGAA in patients with advanced Late-onset Pompe disease.

Conditions

• Pompe Disease (Late-onset)
• Glycogen Storage Disease Type II (GSD-II)
• Acid Maltase Deficiency Disease
• Glycogenosis 2

Interventions

Type	Name	Description
BIOLOGICAL	Myozyme	20 mg/kg qow

Timeline

• Start date: 2005-12-01
• Primary completion: 2007-03-01
• Completion: 2007-06-01
• First posted: 2005-12-23
• Last updated: 2014-02-05

Locations

1 site across 1 country: France