- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT00517153
Miglustat in Niemann-Pick Type C Disease
March 24, 2010 updated by: Actelion
A Phase II Randomized Controlled Study of Miglustat in Adult and Juvenile Patients With Niemann-Pick Type C Disease
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.
Study Overview
Study Type
Interventional
Enrollment (Actual)
29
Phase
- Phase 2
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Locations
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New York
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New York, New York, United States, 10032
- Columbia University Medical Center
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Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
4 years and older (ADULT, OLDER_ADULT, CHILD)
Accepts Healthy Volunteers
No
Genders Eligible for Study
All
Description
Inclusion Criteria:
- 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.
Exclusion Criteria:
- 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.
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: TREATMENT
- Allocation: RANDOMIZED
- Interventional Model: PARALLEL
- Masking: QUADRUPLE
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
EXPERIMENTAL: 1
OGT-918 - Zavesca (miglustat)
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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 Names:
|
NO_INTERVENTION: 2
Standard treatment
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Time Frame |
---|---|
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
|
Baseline to Month 12
|
Secondary Outcome Measures
Outcome Measure |
Time Frame |
---|---|
Highest amplitude for which a velocity measurement is obtained
Time Frame: Baseline to Month 12
|
Baseline to Month 12
|
Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Investigators
- Principal Investigator: Claudia Chiriboga, Assoc. Prof. MD, Columbia University
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start
January 1, 2002
Primary Completion (ACTUAL)
September 1, 2006
Study Completion (ACTUAL)
January 1, 2008
Study Registration Dates
First Submitted
August 15, 2007
First Submitted That Met QC Criteria
August 15, 2007
First Posted (ESTIMATE)
August 16, 2007
Study Record Updates
Last Update Posted (ESTIMATE)
March 26, 2010
Last Update Submitted That Met QC Criteria
March 24, 2010
Last Verified
March 1, 2010
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
- Metabolic Diseases
- Brain Diseases
- Central Nervous System Diseases
- Nervous System Diseases
- Lymphatic Diseases
- Genetic Diseases, Inborn
- Metabolism, Inborn Errors
- Lysosomal Storage Diseases
- Lipid Metabolism Disorders
- Brain Diseases, Metabolic
- Brain Diseases, Metabolic, Inborn
- Sphingolipidoses
- Lysosomal Storage Diseases, Nervous System
- Lipidoses
- Lipid Metabolism, Inborn Errors
- Histiocytosis, Non-Langerhans-Cell
- Histiocytosis
- Niemann-Pick Diseases
- Niemann-Pick Disease, Type A
- Niemann-Pick Disease, Type C
- Hypoglycemic Agents
- Physiological Effects of Drugs
- Molecular Mechanisms of Pharmacological Action
- Anti-Infective Agents
- Antiviral Agents
- Enzyme Inhibitors
- Anti-HIV Agents
- Anti-Retroviral Agents
- Glycoside Hydrolase Inhibitors
- Miglustat
Other Study ID Numbers
- OGT-918-007
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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