CompletedPhase 2

Miglustat in Niemann-Pick Type C Disease

29 participantsStarted 2002-01

Plain-language 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.

Who can participate

Age range4 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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.

What they're measuring

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

Timeframe: Baseline to Month 12

Trial details

NCT IDNCT00517153

SponsorActelion

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2006-09

View on ClinicalTrials.gov More Niemann-Pick Type C Disease trials