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Sponsored by: |
Actelion |
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Information provided by: | Actelion |
ClinicalTrials.gov Identifier: | NCT00517153 |
This is a phase II randomized controlled study of miglustat in adult and juvenile patients with Niemann-Pick Type C disease. Up to 42 patients will be randomised in a 2:1 ratio to either treatment with miglustat or to a non-treatment group. Both groups will follow an identical visit schedule.
Condition | Intervention | Phase |
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Niemann-Pick Type C Disease |
Drug: miglustat |
Phase II |
Study Type: | Interventional |
Study Design: | Treatment, Randomized, Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Historical Control, Parallel Assignment, Safety/Efficacy Study |
Official Title: | A Phase II Randomized Controlled Study of Miglustat in Adult and Juvenile Patients With Niemann-Pick Type C Disease |
Estimated Enrollment: | 42 |
Study Start Date: | January 2002 |
Estimated Study Completion Date: | January 2009 |
Estimated Primary Completion Date: | January 2009 (Final data collection date for primary outcome measure) |
Arms | Assigned Interventions |
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1: Experimental
OGT-918 - Zavesca (miglustat)
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Drug: miglustat
Patients aged 12 years or more randomised to the treatment group will receive 200 mg OGT 918 three times daily initially for twelve months. An additional sub-study of up to 12 patients aged under 12 years will receive a lower dose of OGT 918 according to their BSA
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2: No Intervention
Standard treatment
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Ages Eligible for Study: | 4 Years and older |
Genders Eligible for Study: | Both |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
Exclusion Criteria:
United States, New York | |
Columbia University Medical Center | |
New York, New York, United States, 10032 |
Principal Investigator: | Claudia Chiriboga, Assoc. Prof. MD | Columbia University Medical Center |
Responsible Party: | Actelion ( Dr. Ruben Giorgino ) |
Study ID Numbers: | OGT-918-007 |
Study First Received: | August 15, 2007 |
Last Updated: | August 26, 2008 |
ClinicalTrials.gov Identifier: | NCT00517153 History of Changes |
Health Authority: | United States: Food and Drug Administration; United States: Institutional Review Board |
Zavesca miglustat Niemann-Pick Type C Actelion |
Miglustat Anti-HIV Agents Anti-Retroviral Agents Antiviral Agents |
Anti-Infective Agents Miglustat Anti-HIV Agents Anti-Retroviral Agents Molecular Mechanisms of Pharmacological Action |
Therapeutic Uses Enzyme Inhibitors Antiviral Agents Pharmacologic Actions |