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Sponsored by: |
Genzyme |
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Information provided by: | Genzyme |
ClinicalTrials.gov Identifier: | NCT00059280 |
Pompe disease (also known as glycogen storage disease type II, "GSD-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. This study is being conducted to evaluate the safety and effectiveness of recombinant human acid alpha-glucosidase (rhGAA) as a potential enzyme replacement therapy for Pompe disease. Patients diagnosed with infantile-onset Pompe disease who are less than or equal to 6 months old will be studied.
Condition | Intervention | Phase |
---|---|---|
Glycogen Storage Disease Type II |
Biological: Myozyme |
Phase II Phase III |
Study Type: | Interventional |
Study Design: | Treatment, Randomized, Open Label, Historical Control, Factorial Assignment, Safety/Efficacy Study |
Official Title: | An Open-Label, Multicenter, Multinational Study of the Safety, Efficacy, Pharmacokinetics, and Pharmacodynamics of Recombinant Human Acid Alpha-Glucosidase Treatment in Patients Less Than 6 Months Old With Infantile-Onset Pompe Disease |
Enrollment: | 16 |
Study Start Date: | April 2003 |
Study Completion Date: | September 2005 |
Primary Completion Date: | June 2005 (Final data collection date for primary outcome measure) |
Arms | Assigned Interventions |
---|---|
1: Experimental |
Biological: Myozyme
20 mg/kg qow or 40mg/kg qow
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Ages Eligible for Study: | up to 26 Weeks |
Genders Eligible for Study: | Both |
Accepts Healthy Volunteers: | No |
Inclusion criteria:
Exclusion criteria:
United States, Florida | |
University of Florida College of Medicine | |
Gainesville, Florida, United States, 32610-00266 | |
United States, North Carolina | |
Duke University Medical Center | |
Durham, North Carolina, United States, 27710 | |
United States, Ohio | |
Children's Hospital Medical Center | |
Cincinnati, Ohio, United States, 45229 | |
United States, Utah | |
University of Utah Medical Center | |
Salt Lake City, Utah, United States, 84132 | |
France | |
Pediatrique Hopital deBrousse | |
Lyon, France | |
Israel | |
Rambam Medical Center | |
Haifa, Israel, 31096 | |
Taiwan | |
National Taiwan University Hospital | |
Taipei, Taiwan, 100 | |
United Kingdom | |
Royal Manchester Children's Hospital | |
Manchester, United Kingdom |
Study Director: | Medical Monitor | Genzyme |
Responsible Party: | Genzyme Corporation ( Medical Monitor ) |
Study ID Numbers: | AGLU01602 |
Study First Received: | April 22, 2003 |
Last Updated: | September 23, 2008 |
ClinicalTrials.gov Identifier: | NCT00059280 |
Health Authority: | United States: Food and Drug Administration |
Pompe disease Glycogen storage disease type II GSD-II Acid maltase deficiency disease Glycogenosis 2 |
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 |