Miglustat in Niemann-Pick Type C Disease

Launched by ACTELION · Aug 15, 2007

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Eligibility criteria

• Inclusion Criteria:
• • 1. Patients with Niemann-Pick Type C disease confirmed by abnormal cholesterol esterification and abnormal filipin staining.
• • 2. Patients who can ingest a capsule.
• • 3. Patients who are above the age of four (4) years of age.
• Exclusion Criteria:
• • 1. Patients younger than 18 years who are unable to give informed assent and/or whose legal guardian is unable to provide informed consent.
• • 2. Patients aged 18 and over who cannot provide informed consent and/or whose legal guardian is unable to provide witnessed informed consent.
• • 3. 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.
• • 4. Patients who cannot tolerate the study procedures or who are unable to travel to the study center as required by this protocol.
• • 5. Patients currently undergoing therapy with other investigational agents or patients taking drugs or food supplements which may interfere with gastrointestinal absorption or motility.
• • 6. 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.
• • 7. Patients with an intercurrent medical condition that would render them unsuitable for the study e.g. HIV, hepatitis infection.
• • 8. Patients who in the opinion of the Investigator (for whatever reason) are thought to be unsuitable for the study.
• • 9. Patients with an adjusted Creatinine Clearance of less than 70 ml/min/1.73m2 (CrCl \<70).
• • 10. Patients younger than four (4) years of age.