Miglustat in Niemann-Pick Type C Disease
Study Details
Study Description
Brief Summary
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 or Disease | Intervention/Treatment | Phase |
---|---|---|
Phase 2 |
Study Design
Arms and Interventions
Arm | Intervention/Treatment |
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Experimental: 1 OGT-918 - Zavesca (miglustat) |
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
Other Names:
|
No Intervention: 2 Standard treatment |
Outcome Measures
Primary Outcome Measures
- 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 [Baseline to Month 12]
Secondary Outcome Measures
- Highest amplitude for which a velocity measurement is obtained [Baseline to Month 12]
Eligibility Criteria
Criteria
Inclusion Criteria:
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Patients with Niemann-Pick Type C disease confirmed by abnormal cholesterol esterification and abnormal filipin staining.
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Patients who can ingest a capsule.
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Patients who are above the age of four (4) years of age.
Exclusion Criteria:
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Patients younger than 18 years who are unable to give informed assent and/or whose legal guardian is unable to provide informed consent.
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Patients aged 18 and over who cannot provide informed consent and/or whose legal guardian is unable to provide witnessed informed consent.
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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.
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Patients who cannot tolerate the study procedures or who are unable to travel to the study center as required by this protocol.
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Patients currently undergoing therapy with other investigational agents or patients taking drugs or food supplements which may interfere with gastrointestinal absorption or motility.
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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.
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Patients with an intercurrent medical condition that would render them unsuitable for the study e.g. HIV, hepatitis infection.
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Patients who in the opinion of the Investigator (for whatever reason) are thought to be unsuitable for the study.
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Patients with an adjusted Creatinine Clearance of less than 70 ml/min/1.73m2 (CrCl <70).
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Patients younger than four (4) years of age.
Contacts and Locations
Locations
Site | City | State | Country | Postal Code | |
---|---|---|---|---|---|
1 | Columbia University Medical Center | New York | New York | United States | 10032 |
Sponsors and Collaborators
- Actelion
Investigators
- Principal Investigator: Claudia Chiriboga, Assoc. Prof. MD, Columbia University
Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- OGT-918-007