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Sponsored by: |
Actelion |
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Information provided by: | Actelion |
ClinicalTrials.gov Identifier: | NCT00319046 |
Although miglustat has been approved as a treatment for mild to moderate type 1 Gaucher disease in patients who are unsuitable for ERT, more data are required to establish the long term efficacy, safety and tolerability of miglustat in maintaining diseases stability after a switch from ERT.
Condition | Intervention | Phase |
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Type 1 Gaucher Disease |
Drug: miglustat |
Phase III |
Study Type: | Interventional |
Study Design: | Treatment, Non-Randomized, Open Label, Uncontrolled, Single Group Assignment, Safety/Efficacy Study |
Official Title: | Open-Label, Non Comparative, Multi-Center Study to Evaluate the Long Term Efficacy, Safety and Tolerability of Oral Miglustat as a Maintenance Therapy After a Switch From Enzyme Replacement Therapy in Adult Patients With Stable Type 1 Gaucher Disease |
Enrollment: | 42 |
Study Start Date: | February 2006 |
Estimated Study Completion Date: | July 2010 |
Estimated Primary Completion Date: | June 2010 (Final data collection date for primary outcome measure) |
Arms | Assigned Interventions |
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1: Experimental |
Drug: miglustat
miglustat oral capsules 100mg TID
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Ages Eligible for Study: | 18 Years and older |
Genders Eligible for Study: | Both |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
Clinically and biologically stable disease for the previous 2 years, with at least 2 time points assessments (including Baseline as one potential time point), defined as:
Stable organomegaly (assessed by MRI or CT):
Chitotriosidase activity within 20% of the mean.
Exclusion Criteria:
Principal Investigator: | Timothy Cox, Prof | University of Cambridge |
Responsible Party: | Actelion ( Ruben Giorgino, MD ) |
Study ID Numbers: | OGT 918-011 |
Study First Received: | April 26, 2006 |
Last Updated: | July 1, 2008 |
ClinicalTrials.gov Identifier: | NCT00319046 History of Changes |
Health Authority: | United States: Food and Drug Administration |
Type 1 Gaucher Disease miglustat enzyme replacement therapy |
Lipid Metabolism, Inborn Errors Sphingolipidoses Anti-HIV Agents Metabolic Diseases Lysosomal Storage Diseases Sphingolipidosis Central Nervous System Diseases Brain Diseases Antiviral Agents Lymphatic Diseases |
Metabolism, Inborn Errors Miglustat Anti-Retroviral Agents Genetic Diseases, Inborn Brain Diseases, Metabolic, Inborn Lipidoses Gaucher Disease Metabolic Disorder Lipid Metabolism Disorders Brain Diseases, Metabolic |
Lipid Metabolism, Inborn Errors Anti-Infective Agents Sphingolipidoses Molecular Mechanisms of Pharmacological Action Brain Diseases Metabolism, Inborn Errors Anti-Retroviral Agents Therapeutic Uses Brain Diseases, Metabolic, Inborn Reticuloendotheliosis Metabolic Diseases Anti-HIV Agents Lysosomal Storage Diseases, Nervous System |
Lysosomal Storage Diseases Nervous System Diseases Central Nervous System Diseases Enzyme Inhibitors Antiviral Agents Pharmacologic Actions Lymphatic Diseases Miglustat Genetic Diseases, Inborn Lipidoses Gaucher Disease Brain Diseases, Metabolic Lipid Metabolism Disorders |