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Sponsored by: |
Genzyme |
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Information provided by: | Genzyme |
ClinicalTrials.gov Identifier: | NCT00268944 |
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 and efficacy of rhGAA in patients with advanced Late-onset Pompe disease.
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 III |
Study Type: | Interventional |
Study Design: | Treatment, Non-Randomized, Open Label, Uncontrolled, Single Group Assignment, Safety/Efficacy Study |
Official Title: | Prospective, Open-Label, Single-Arm, Exploratory Study of the Effect and Safety of rhGAA in Patients With Advanced Late-Onset Pompe Disease Who Are Receiving Respiratory Support |
Enrollment: | 5 |
Study Start Date: | December 2005 |
Study Completion Date: | June 2007 |
Primary Completion Date: | March 2007 (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: | 18 Years and older |
Genders Eligible for Study: | Both |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
Exclusion Criteria:
Responsible Party: | Genzyme Corporation ( Medical Monitor ) |
Study ID Numbers: | AGLU03105 |
Study First Received: | December 22, 2005 |
Last Updated: | September 23, 2008 |
ClinicalTrials.gov Identifier: | NCT00268944 |
Health Authority: | France: Afssaps - French Health Products Safety 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 |