Trial | NCT00517153  Report issue

Title

Miglustat in Niemann-Pick Type C Disease
A Phase II Randomized Controlled Study of Miglustat in Adult and Juvenile Patients With Niemann-Pick Type C Disease

  • Phase

    Phase 2

  • Study Type

    Interventional

  • Status

    Completed No Results Posted

  • Intervention/Treatment

    miglustat ...

  • Study Participants

    29
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 Started
Jan 31
2002
Primary Completion
Sep 30
2006
Study Completion
Jan 31
2008
Last Update
Mar 26
2010
Estimate

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: Zavesca

1 Experimental

OGT-918 - Zavesca (miglustat)

2 No Intervention

Standard treatment

Criteria 

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.
No Results Posted