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Sponsored by: |
Genzyme |
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Information provided by: | Genzyme |
ClinicalTrials.gov Identifier: | NCT00025896 |
Pompe disease 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 infants with severe cases of Pompe disease (called Classical Infantile Pompe disease), an excessive amount of glycogen accumulates and is stored in various tissues, especially heart, skeletal muscle, and liver, which prevents their normal function. This study 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 Classical Infantile Pompe disease who have a small, but inactive, amount of natural GAA enzyme present in their bodies (called Cross-Reacting Immunologic Material-Positive or "CRIM (+)" patients), will be studied.
Condition | Intervention | Phase |
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Pompe Disease Glycogen Storage Disease Type II Acid Maltase Deficiency Disease Glycogenosis 2 |
Drug: recombinant human acid alpha-glucosidase (rhGAA) |
Phase II |
Study Type: | Interventional |
Study Design: | Treatment, Non-Randomized, Open Label, Historical Control, Single Group Assignment, Safety/Efficacy Study |
Official Title: | A Prospective Multinational, Multicenter, Clinical Trial of the Safety and Efficacy of Recombinant Human Acid Alpha-Glucosidase (rhGAA) in Cross-Reacting Immunologic Material-Positive Patients With Classical Infantile Pompe Disease |
Genders Eligible for Study: | Both |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
Exclusion Criteria:
Responsible Party: | Genzyme Corporation ( Medical Monitor ) |
Study ID Numbers: | AGLU-001-00 |
Study First Received: | October 31, 2001 |
Last Updated: | August 4, 2009 |
ClinicalTrials.gov Identifier: | NCT00025896 History of Changes |
Health Authority: | United States: Food and Drug Administration |
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 |