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Sponsored by: |
National Institute of Neurological Disorders and Stroke (NINDS) |
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Information provided by: | National Institutes of Health Clinical Center (CC) |
ClinicalTrials.gov Identifier: | NCT00357786 |
This study will continue to evaluate the safety of using intravenous doses of Replagal for two patients with Fabry disease. Fabry disease is a genetic disorder inherited as an X-linked recessive trait. It causes a deficiency in the enzyme alpha galactosidase, which normally breaks down a lipid, or fatty substance called ceramidetrihexoside, a building block in all cells of the body. The deficiency in breaking down the lipid eventually causes that lipid to accumulate and injure cells. Vascular, renal, and neurological problems are the results. It is not known exactly how lipid accumulation brings about such problems, studies of another lipid storage disorder.
Two patients 7 to 17 years of age who have Fabry disease and have been receiving intravenous infusions of Replagal at a dose of 0.2 mg/kg of body weight every 2 weeks may be eligible for this study.
Participants will undergo the following tests and procedures:
Participants will go through a baseline evaluation, over a period of about 1 day. They will receive an intravenous infusion of Replagal every other week, at the dose of 0.2 mg/kg of body weight. Vital signs will be measured before the infusion and immediately and after and 1 hour afterward. There will be careful monitoring for allergic reactions and side effects. The infusion time takes approximately 40 minutes.
This study will last at least 1 year, or until the sponsor doing the investigating or the drug manufacturer decides to withdraw support of the study.
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Condition | Intervention | Phase |
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Fabry Disease |
Drug: Replagal agalsidase alfa Drug: Replagal |
Phase III |
Study Type: | Interventional |
Study Design: | Treatment, Open Label, Single Group Assignment, Safety Study |
Official Title: | An Open-Label Maintenance Study of the Enzyme Replacement Therapy Replagal® (Registered Trademark) in Patients With Fabry Disease |
Estimated Enrollment: | 3 |
Study Start Date: | October 2003 |
Estimated Study Completion Date: | December 2008 |
Arms | Assigned Interventions |
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A: Experimental
Enzyme replacement for Fabry's Disease
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Drug: Replagal agalsidase alfa
N/A
Drug: Replagal
N/A
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Objectives: This goal of this study is to continue treating two patients with Fabry disease using enzyme replacement therapy (ERT) using Replagal (agalsidase alfa) at a dose of 0.2 mg/kg of body weight administered every 2 weeks. Study Population: Two patients with Fabry disease who are currently on clinical research protocols 00-N-0185/TKT011 or 02-N-0220/TKT015 and who are stable on ERT. Design: This is an open label study. Outcome Measures: Mainly safety parameters will be obtained. Study duration is estimated to be 2 years.
Ages Eligible for Study: | 39 Years to 45 Years |
Genders Eligible for Study: | Male |
Accepts Healthy Volunteers: | No |
Responsible Party: | National Institutes of Health ( Raphael Schiffmann, M.D./National Institute of Neurological Disorders and Stroke ) |
Study ID Numbers: | 040027, 04-N-0027 |
Study First Received: | July 26, 2006 |
Last Updated: | December 16, 2008 |
ClinicalTrials.gov Identifier: | NCT00357786 |
Health Authority: | United States: Federal Government |
Lysosomal Disease Glycolipid Stroke Peripheral Neuropathy Storage Disorder |
Lipid Metabolism, Inborn Errors Sphingolipidoses Metabolic Diseases Cerebral Infarction Lysosomal Storage Diseases Stroke Fabry disease Sphingolipidosis Central Nervous System Diseases Brain Diseases Metabolism, Inborn Errors |
Ceramide trihexosidosis Genetic Diseases, Inborn Peripheral Nervous System Diseases Fabry Disease Genetic Diseases, X-Linked Lipidoses Brain Diseases, Metabolic, Inborn Metabolic disorder Lipid Metabolism Disorders Brain Diseases, Metabolic |
Lysosomal Storage Diseases, Nervous System Nervous System Diseases |