Clinical Trial: Expanded Access Use of Myozyme (Alglucosidase Alfa) in Patients With Infantile-onset Pompe Disease

Study Status: Approved for marketing
Recruit Status: Approved for marketing
Study Type: Expanded Access

Official Title: Expanded Access Use of Myozyme (Alglucosidase Alfa) in Patients With Infantile-onset Pompe Disease

Brief 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 protocol is to provide enzyme replacement therapy with rhGAA on an expanded access basis, to severely affected patients with infantile-onset Pompe disease for whom there is no alternative treatment and who do not meet the clinical characteristics described in the inclusion criteria for participation in other Genzyme Corporation-sponsored study currently enrolling patients with infantile-onset Pompe disease.

Detailed Summary:
Sponsor: Genzyme, a Sanofi Company

Current Primary Outcome:

Original Primary Outcome:

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Original Secondary Outcome:

Information By: Sanofi

Dates:
Date Received: December 23, 2003
Date Started: December 2003
Date Completion:
Last Updated: February 4, 2014
Last Verified: February 2014