Miglustat in Niemann-Pick Type C Disease
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|ClinicalTrials.gov Identifier: NCT00517153|
Recruitment Status : Completed
First Posted : August 16, 2007
Last Update Posted : March 26, 2010
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|Condition or disease||Intervention/treatment||Phase|
|Niemann-Pick Type C Disease||Drug: miglustat||Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||29 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)|
|Official Title:||A Phase II Randomized Controlled Study of Miglustat in Adult and Juvenile Patients With Niemann-Pick Type C Disease|
|Study Start Date :||January 2002|
|Actual Primary Completion Date :||September 2006|
|Actual Study Completion Date :||January 2008|
OGT-918 - Zavesca (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
Other Name: Zavesca
No Intervention: 2
- The primary endpoints will be two parameters - Horizontal Saccadic α and Horizontal Saccadic β - which are estimated for each patient from their saccadic eye movement data [ Time Frame: Baseline to Month 12 ]
- Highest amplitude for which a velocity measurement is obtained [ Time Frame: Baseline to Month 12 ]
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|Ages Eligible for Study:||4 Years and older (Child, Adult, Older Adult)|
|Sexes Eligible for Study:||All|
|Accepts Healthy Volunteers:||No|
- Patients with Niemann-Pick Type C disease confirmed by abnormal cholesterol esterification and abnormal filipin staining.
- Patients who can ingest a capsule.
- Patients who are above the age of four (4) years of age.
- Patients younger than 18 years who are unable to give informed assent and/or whose legal guardian is unable to provide informed consent.
- Patients aged 18 and over who cannot provide informed consent and/or whose legal guardian is unable to provide witnessed informed consent.
- Sexually active and fertile patients who do not agree to use adequate contraception throughout the study and for three months after cessation of OGT 918 treatment.
- Patients who cannot tolerate the study procedures or who are unable to travel to the study center as required by this protocol.
- Patients currently undergoing therapy with other investigational agents or patients taking drugs or food supplements which may interfere with gastrointestinal absorption or motility.
- Patients suffering from clinically significant diarrhea (>3 liquid stools per day for >7 days) without definable cause within 3 months of Screening Visit, or who have a history of significant gastrointestinal disorders.
- Patients with an intercurrent medical condition that would render them unsuitable for the study e.g. HIV, hepatitis infection.
- Patients who in the opinion of the Investigator (for whatever reason) are thought to be unsuitable for the study.
- Patients with an adjusted Creatinine Clearance of less than 70 ml/min/1.73m2 (CrCl <70).
- Patients younger than four (4) years of age.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00517153
|United States, New York|
|Columbia University Medical Center|
|New York, New York, United States, 10032|
|Principal Investigator:||Claudia Chiriboga, Assoc. Prof. MD||Columbia University|
|Responsible Party:||Dr. Ruben Giorgino, Actelion|
|Other Study ID Numbers:||
|First Posted:||August 16, 2007 Key Record Dates|
|Last Update Posted:||March 26, 2010|
|Last Verified:||March 2010|
Niemann-Pick Type C
Niemann-Pick Disease, Type C
Niemann-Pick Disease, Type A
Lysosomal Storage Diseases, Nervous System
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Central Nervous System Diseases
Nervous System Diseases
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Lipid Metabolism, Inborn Errors
Lysosomal Storage Diseases
Lipid Metabolism Disorders
Molecular Mechanisms of Pharmacological Action
Glycoside Hydrolase Inhibitors
Physiological Effects of Drugs