NCT00520143

Alglucosidase Alfa Temporary Access Program

• Status: Approved For Marketing
• Phase: —
• Study type: Expanded Access
• Enrollment: —
• Sponsor: Genzyme, a Sanofi Company · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: —

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 expanded access study is to provide patients with Pompe disease in the United States (US), access to alglucosidase alfa produced from a scaled up manufacturing process for a limited time until production at this scale is approved for commercial use by the Food and Drug Administration.

Conditions

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

Interventions

Type	Name	Description
BIOLOGICAL	alglucosidase alfa (recombinant human acid alpha-glucosidase [rhGAA])	IV infusion: 20mg/kg qow

Timeline

• First posted: 2007-08-23
• Last updated: 2014-02-06

Locations

74 sites across 1 country: United States