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Sponsors and Collaborators: |
The Hospital for Sick Children Actelion |
---|---|
Information provided by: | The Hospital for Sick Children |
ClinicalTrials.gov Identifier: | NCT00418847 |
The purpose of the study is to investigate the pharmacokinetics of Zavesca (miglustat, OGT918) when given as single and multiple doses in juvenile patients with GM2 gangliosidosis.
Condition | Intervention | Phase |
---|---|---|
Gangliosidoses GM2 |
Drug: miglustat |
Phase II |
Study Type: | Interventional |
Study Design: | Treatment, Non-Randomized, Open Label, Uncontrolled, Single Group Assignment, Pharmacokinetics Study |
Official Title: | Pharmacokinetics and Tolerability of Zavesca® (Miglustat) In Patients With Juvenile GM2 Gangliosidosis: Single and Multiple Oral Doses |
Estimated Enrollment: | 5 |
Study Start Date: | July 2004 |
Estimated Study Completion Date: | October 2006 |
The GM2 gangliosidoses are a group of neuro-degenerative lysosomal storage diseases resulting from accumulation of GM2 and related glycolipids in the central nervous system (CNS). Tay-Sachs and Sandhoff disease are two variants which are indistinguishable in clinical grounds. According to the onset and rate of disease progression, the condition can be categorized in infantile, juvenile and adult forms. This open-label, single-arm study is designed to assess the pharmacokinetics, safety and tolerability of miglustat in juvenile patients. Miglustat will be administered at a maximum dose of 600 mg/day, divided into three doses per day. The dose used for patients in this pediatric age range will be related to the patient’s body surface area. The pharmacokinetics assessments for the study will be performed in-hospital during a 24 hour period, and will take place at the day one and at the month 3 visits. The clinical (which includes safety and tolerability) assessments will be performed throughout the 24-month study period.
Ages Eligible for Study: | 6 Years to 20 Years |
Genders Eligible for Study: | Both |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
Exclusion Criteria:
Canada, Ontario | |
The Hospital for Sick Children | |
Toronto, Ontario, Canada, M5G 1X8 |
Principal Investigator: | Joe TR Clarke, MD | The Hospital for Sick Children, Toronto Canada |
Study ID Numbers: | 1000004763 |
Study First Received: | January 4, 2007 |
Last Updated: | January 4, 2007 |
ClinicalTrials.gov Identifier: | NCT00418847 |
Health Authority: | Canada: Health Canada |
pediatrics lysosomal storage diseases Gangliosidoses GM2 Tay-Sachs disease |
Sandhoff disease Infantile GM2-activator deficiency Miglustat |
Lipid Metabolism, Inborn Errors Gangliosidosis (GM2) Type1 Sphingolipidoses Metabolic Diseases Lysosomal Storage Diseases Sphingolipidosis Central Nervous System Diseases Tay-Sachs disease Brain Diseases Metabolism, Inborn Errors Miglustat |
Genetic Diseases, Inborn Gangliosidoses, GM2 Brain Diseases, Metabolic, Inborn Lipidoses Metabolic disorder Tay-Sachs Disease Sandhoff disease Gangliosidoses Sandhoff Disease Lipid Metabolism Disorders Brain Diseases, Metabolic |
Anti-Infective Agents Anti-HIV Agents Anti-Retroviral Agents Molecular Mechanisms of Pharmacological Action Lysosomal Storage Diseases, Nervous System |
Therapeutic Uses Nervous System Diseases Enzyme Inhibitors Antiviral Agents Pharmacologic Actions |