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Sponsored by: |
Genzyme |
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Information provided by: | Genzyme |
ClinicalTrials.gov Identifier: | NCT00365131 |
This is a multicenter, open-label, prospective study of the efficacy of Cerezyme in treating patients with skeletal manifestations secondary to Type I Gaucher disease. The study objective is to evaluate and quantify skeletal responses as compared to baseline in Type I gaucher disease patients receiving Cerezyme therapy for 48 months. Additional objectives were to assess the usefulness of various skeletal parameters, such as bone pain, bone crises, bone mineral density, and serum and urine bone markers, as indicative of treatment response and may be useful in dose management.
Condition | Intervention | Phase |
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Gaucher Disease Type I Cerebroside Lipidosis Syndrome Clucocerebrosidase Deficiency Disease Glucosylceramide Beta-Glucosidase Deficiency Disease Gaucher Disease, Non-Neuronopathic Form |
Drug: Cerezyme (imiglucerase for injection) |
Phase IV |
Study Type: | Interventional |
Study Design: | Treatment, Non-Randomized, Open Label, Uncontrolled, Single Group Assignment, Efficacy Study |
Ages Eligible for Study: | 10 Years to 65 Years |
Genders Eligible for Study: | Both |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
Exclusion Criteria:
Study ID Numbers: | RC96-1101 |
Study First Received: | August 15, 2006 |
Last Updated: | July 16, 2007 |
ClinicalTrials.gov Identifier: | NCT00365131 History of Changes |
Health Authority: | United States: Food and Drug Administration |
Type I Gaucher disease Glucocerebrosidase Deficiency Disease |
Lipid Metabolism, Inborn Errors Sphingolipidoses Metabolic Diseases Gaucher Disease Type 1 Lysosomal Storage Diseases Sphingolipidosis Central Nervous System Diseases Brain Diseases Lymphatic Diseases Metabolism, Inborn Errors |
Malnutrition Genetic Diseases, Inborn Nutrition Disorders Lipidoses Brain Diseases, Metabolic, Inborn Gaucher Disease Metabolic Disorder Lipid Metabolism Disorders Deficiency Diseases Brain Diseases, Metabolic |
Lipid Metabolism, Inborn Errors Sphingolipidoses Disease Metabolic Diseases Reticuloendotheliosis Lysosomal Storage Diseases, Nervous System Lysosomal Storage Diseases Nervous System Diseases Central Nervous System Diseases Brain Diseases Metabolism, Inborn Errors Lymphatic Diseases |
Pathologic Processes Malnutrition Genetic Diseases, Inborn Syndrome Nutrition Disorders Lipidoses Brain Diseases, Metabolic, Inborn Gaucher Disease Lipid Metabolism Disorders Deficiency Diseases Brain Diseases, Metabolic |