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Sponsored by: |
Genzyme |
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Information provided by: | Genzyme |
ClinicalTrials.gov Identifier: | NCT00250939 |
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 overall objective is to evaluate the safety, pharmacokinetics (PK) and efficacy of Myozyme treatment.
Condition | Intervention | Phase |
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Pompe Disease (Late-Onset) Glycogen Storage Disease Type II (GSD-II) Acid Maltase Deficiency Disease Glycogenosis 2 |
Biological: Myozyme |
Phase II |
Study Type: | Interventional |
Study Design: | Treatment, Non-Randomized, Open Label, Uncontrolled, Single Group Assignment, Safety/Efficacy Study |
Official Title: | Single-Center, Open-Label Study of Safety, Pharmacokinetics and Efficacy of rhGAA in Patients With Late-Onset Pompe Disease |
Enrollment: | 5 |
Study Start Date: | February 2005 |
Study Completion Date: | November 2006 |
Primary Completion Date: | July 2006 (Final data collection date for primary outcome measure) |
Arms | Assigned Interventions |
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1: Experimental |
Biological: Myozyme
20 mg/kg qow
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Ages Eligible for Study: | 5 Years to 18 Years |
Genders Eligible for Study: | Both |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
Exclusion Criteria:
Responsible Party: | Genzyme Corporation ( Medical Monitor ) |
Study ID Numbers: | AGLU02804 |
Study First Received: | November 8, 2005 |
Last Updated: | October 14, 2008 |
ClinicalTrials.gov Identifier: | NCT00250939 |
Health Authority: | European Union: European Medicines Agency |
Glycogen Storage Disease Type II GSD-II Pompe 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 |
Malnutrition Genetic Diseases, Inborn Nutrition Disorders Brain Diseases, Metabolic, Inborn Metabolic disorder Deficiency Diseases Brain Diseases, Metabolic |
Lysosomal Storage Diseases, Nervous System Nervous System Diseases Carbohydrate Metabolism, Inborn Errors |