Miglustat in Niemann-Pick Type C Disease

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.