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Sponsored by: |
Genzyme |
Information provided by: | Genzyme |
ClinicalTrials.gov Identifier: | NCT00074919 |
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.
Condition | Intervention |
Glycogen Storage Disease Type II Glycogenosis 2 |
Biological: Myozyme |
Genetics Home Reference related topics: | Pompe disease |
ChemIDplus related topics: | Alglucosidase Alfa Glucan 1,4-alpha-Glucosidase |
Study Type: | Interventional |
Study Design: | Treatment, Non-Randomized, Open Label, Uncontrolled, Single Group Assignment, Safety/Efficacy Study |
Official Title: | Expanded Access Use of Myozyme (Alglucosidase Alfa) in Patients With Infantile-Onset Pompe Disease |
Enrollment: | 33 |
Study Start Date: | December 2003 |
Study Completion Date: | February 2007 |
Primary Completion Date: | July 2006 (Final data collection date for primary outcome measure) |
Arms | Assigned Interventions |
1 |
Biological: Myozyme
20 mg/kg qow
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Genders Eligible for Study: | Both |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
Exclusion Criteria:
US FDA Approved Full Prescribing Information for Myozyme® 
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Responsible Party: | Genzyme Corporation ( Medical Monitor ) |
Study ID Numbers: | AGLU02203 |
First Received: | December 23, 2003 |
Last Updated: | September 30, 2008 |
ClinicalTrials.gov Identifier: | NCT00074919 |
Health Authority: | United States: Food and Drug Administration |
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