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Sponsored by: |
Genzyme |
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Information provided by: | Genzyme |
ClinicalTrials.gov Identifier: | NCT00486889 |
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 study is to evaluate the long-term growth and development of patients with infantile-onset Pompe disease who are treated with Myozyme before 1 year of age over a 10-year period.
Condition | Intervention | Phase |
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Pompe Disease Glycogen Storage Disease Type II (GSD-II) Acid Maltase Deficiency Disease |
Biological: Myozyme® (alglucosidase alfa) |
Phase IV |
Study Type: | Interventional |
Study Design: | Treatment, Non-Randomized, Open Label, Single Group Assignment, Safety/Efficacy Study |
Official Title: | A Long-Term Study to Evaluate Growth and Development Outcomes in Patients With Infantile-Onset Pompe Disease Who Are Receiving Myozyme® (Alglucosidase Alfa) |
Estimated Enrollment: | 30 |
Study Start Date: | December 2007 |
Estimated Study Completion Date: | September 2020 |
Estimated Primary Completion Date: | September 2018 (Final data collection date for primary outcome measure) |
Arms | Assigned Interventions |
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1: Experimental |
Biological: Myozyme® (alglucosidase alfa)
IV infusion: 20mg/kg qow
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Ages Eligible for Study: | up to 12 Months |
Genders Eligible for Study: | Both |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
Exclusion Criteria:
Contact: Medical Information | 800-745-4447 | medinfo@genzyme.com |
Contact: Medical Information | 617-252-7832 | medinfo@genzyme.com |
United States, Georgia | |
Recruiting | |
Decatur, Georgia, United States | |
United States, Massachusetts | |
Recruitment is not limited to the facility listed; facilities not yet active may be added upon identification of a patient. | Recruiting |
Cambridge, Massachusetts, United States |
Study Director: | Medical Monitor | Genzyme |
Responsible Party: | Genzyme Corporation ( Medical Monitor ) |
Study ID Numbers: | AGLU03606 |
Study First Received: | June 13, 2007 |
Last Updated: | June 9, 2009 |
ClinicalTrials.gov Identifier: | NCT00486889 History of Changes |
Health Authority: | United States: Food and Drug Administration |
Glycogenesis 2 |
Metabolic Diseases Glycogen Storage Disease Lysosomal Storage Diseases Central Nervous System Diseases Glycogen Storage Disease Type II Brain Diseases Metabolism, Inborn Errors Glycogen Storage Disease Type 2 |
Malnutrition Genetic Diseases, Inborn Nutrition Disorders Brain Diseases, Metabolic, Inborn Metabolic Disorder Deficiency Diseases Brain Diseases, Metabolic |
Metabolic Diseases Lysosomal Storage Diseases, Nervous System Lysosomal Storage Diseases Glycogen Storage Disease Nervous System Diseases Central Nervous System Diseases Glycogen Storage Disease Type II Brain Diseases |
Metabolism, Inborn Errors Malnutrition Genetic Diseases, Inborn Nutrition Disorders Brain Diseases, Metabolic, Inborn Carbohydrate Metabolism, Inborn Errors Deficiency Diseases Brain Diseases, Metabolic |