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Sponsored by: |
Genzyme |
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Information provided by: | Genzyme |
ClinicalTrials.gov Identifier: | NCT00074984 |
People with Fabry disease have an alteration in their genetic material (DNA) which causes a deficiency of the
a-galactosidase A enzyme. Fabrazyme is a drug that helps to breakdown and remove certain types of fatty substances called "glycolipids." These glycolipids are normally present within the body in most cells.
In Fabry disease, glycolipids build up in various tissues such as the liver, kidney, skin, and blood vessels because a-galactosidase A is not present, or is present in small quantities. The build up of glycolipid ("globatriaosylceramide" or "GL-3") levels in these tissues in particular is thought to cause the clinical symptoms that are common to Fabry disease. This study will test the safety and efficacy of Fabrazyme in the treatment of patients with Fabry disease.
Condition | Intervention | Phase |
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Fabry Disease |
Drug: Fabrazyme (agalsidase beta) |
Phase IV |
Study Type: | Interventional |
Study Design: | Treatment, Randomized, Double-Blind, Placebo Control, Parallel Assignment, Safety/Efficacy Study |
Official Title: | Multi-Center, Randomized, Double-Blind, Placebo-Controlled Study of the Safety and Efficacy of Fabrazyme on Progression of Renal Disease and Significant Clinical Events in Patients With Fabry Disease |
Ages Eligible for Study: | 16 Years and older |
Genders Eligible for Study: | Both |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
Exclusion Criteria:
Study Director: | Medical Monitor | Genzyme Coorporation |
Responsible Party: | Genzyme Coporation ( Medical Monitor ) |
Study ID Numbers: | AGAL-008-00 |
Study First Received: | December 24, 2003 |
Last Updated: | August 11, 2009 |
ClinicalTrials.gov Identifier: | NCT00074984 History of Changes |
Health Authority: | United States: Food and Drug Administration |
a-Galactosidase A aGAL r-haGAL |
Fabry GL-3 Fabrazyme |
Lipid Metabolism, Inborn Errors Sphingolipidoses Metabolic Diseases Lysosomal Storage Diseases Disease Progression Sphingolipidosis Central Nervous System Diseases Brain Diseases Metabolism, Inborn Errors Genetic Diseases, Inborn |
Fabry Disease Genetic Diseases, X-Linked Ceramide Trihexosidosis Kidney Diseases Brain Diseases, Metabolic, Inborn Lipidoses Metabolic Disorder Lipid Metabolism Disorders Brain Diseases, Metabolic |
Lipid Metabolism, Inborn Errors Sphingolipidoses Metabolic Diseases Lysosomal Storage Diseases, Nervous System Lysosomal Storage Diseases Nervous System Diseases Central Nervous System Diseases Brain Diseases |
Metabolism, Inborn Errors Genetic Diseases, Inborn Fabry Disease Genetic Diseases, X-Linked Brain Diseases, Metabolic, Inborn Lipidoses Lipid Metabolism Disorders Brain Diseases, Metabolic |