Miglustat in Niemann-Pick Type C Disease

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00517153
Recruitment Status : Completed
First Posted : August 16, 2007
Last Update Posted : March 26, 2010
Information provided by:

August 15, 2007
August 16, 2007
March 26, 2010
January 2002
September 2006   (Final data collection date for primary outcome measure)
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 [ Time Frame: Baseline to Month 12 ]
Not Provided
Complete list of historical versions of study NCT00517153 on Archive Site
Highest amplitude for which a velocity measurement is obtained [ Time Frame: Baseline to Month 12 ]
Not Provided
Not Provided
Not Provided
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
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.
Not Provided
Phase 2
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Niemann-Pick Type C Disease
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 Name: Zavesca
  • Experimental: 1
    OGT-918 - Zavesca (miglustat)
    Intervention: Drug: miglustat
  • No Intervention: 2
    Standard treatment
Not Provided

*   Includes publications given by the data provider as well as publications identified by Identifier (NCT Number) in Medline.
January 2008
September 2006   (Final data collection date for primary outcome measure)

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.
Sexes Eligible for Study: All
4 Years and older   (Child, Adult, Older Adult)
Contact information is only displayed when the study is recruiting subjects
United States
Not Provided
Not Provided
Dr. Ruben Giorgino, Actelion
Not Provided
Principal Investigator: Claudia Chiriboga, Assoc. Prof. MD Columbia University
March 2010

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP