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Sponsored by: |
Genzyme |
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Information provided by: | Genzyme |
ClinicalTrials.gov Identifier: | NCT00074932 |
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 alglucosidase alfa on an expanded access basis, to severely affected patients with late-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 studies currently enrolling patients with late-onset Pompe disease.
Condition | Intervention |
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Glycogen Storage Disease Type II Glycogenosis 2 |
Biological: Myozyme |
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 Late-Onset Pompe Disease |
Enrollment: | 9 |
Study Start Date: | November 2004 |
Study Completion Date: | December 2006 |
Primary Completion Date: | August 2006 (Final data collection date for primary outcome measure) |
Arms | Assigned Interventions |
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1 |
Biological: Myozyme
20 mg/kg
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Genders Eligible for Study: | Both |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
Exclusion Criteria:
United States, Colorado | |
Colorado Health Science Center | |
Aurora, Colorado, United States | |
United States, Kansas | |
Galichia Heart Hospital | |
Wichita, Kansas, United States | |
United States, Massachusetts | |
Genzyme Medical Information | |
Cambridge, Massachusetts, United States, 02142 | |
United States, Missouri | |
Freeman Health Systems | |
Joplin, Missouri, United States | |
United States, New York | |
The Women's and Children's Hospital of Buffalo | |
Buffalo, New York, United States | |
Macoumb/Oakland Adult Medicine | |
Rochester Hills, New York, United States | |
North Shore University Hospital | |
Manhasset, New York, United States | |
United States, Virginia | |
Riverside Regional Medical Center | |
Newport News, Virginia, United States |
Study Director: | Medical Monitor | Genzyme |
Responsible Party: | Genzyme Corporation ( Medical Monitor ) |
Study ID Numbers: | AGLU02603 |
Study First Received: | December 23, 2003 |
Last Updated: | October 14, 2008 |
ClinicalTrials.gov Identifier: | NCT00074932 |
Health Authority: | United States: Food and Drug Administration |
Glycogen Storage Disease Type II GSD-II Pompe Disease Acid Maltase Deficiency Disease |
Metabolic Diseases Glycogen Storage Disease Lysosomal Storage Diseases Central Nervous System Diseases Glycogen Storage Disease Type II Brain Diseases Glycogen storage disease type 2 |
Metabolism, Inborn Errors Genetic Diseases, Inborn Brain Diseases, Metabolic, Inborn Metabolic disorder Deficiency Diseases Brain Diseases, Metabolic |
Lysosomal Storage Diseases, Nervous System Nervous System Diseases Carbohydrate Metabolism, Inborn Errors |