Clinical Trial: A Study of the Safety and Efficacy of rhGAA in Patients With Infantile-onset Pompe Disease
Study Status: Completed
Recruit Status: Completed
Study Type: Interventional
Official Title: An Open-label, Multicenter, Multinational Study of the Safety, Efficacy, Pharmacokinetics, and Pharmacodynamics of Recombinant Human Acid Alpha-glucosidase Treatment in Pa
Brief Summary: Pompe disease (also known as glycogen storage disease type II, "GSD-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. This study is being conducted to evaluate the safety and effectiveness of recombinant human acid alpha-glucosidase (rhGAA) as a potential enzyme replacement therapy for Pompe disease. Patients diagnosed with infantile-onset Pompe disease who are less than or equal to 6 months old will be studied.
Detailed Summary:
Sponsor: Genzyme, a Sanofi Company
Current Primary Outcome:
- Evaluate the safety profile of MZ [ Time Frame: 52 weeks ]
- To estimate the proportion of patients treated w/ MZ who were alive and free of ventilator support at 12 months of age; compared to historical cohort [ Time Frame: 52 weeks ]
- Determine PK/PD profile of MZ [ Time Frame: 52 weeks ]
- Determine effect of different doses of MZ on safety and efficacy [ Time Frame: 52 weeks ]
Original Primary Outcome:
Current Secondary Outcome:
Original Secondary Outcome:
Information By: Sanofi
Dates:
Date Received: April 22, 2003
Date Started: April 2003
Date Completion:
Last Updated: February 4, 2014
Last Verified: July 2006
