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Miglustat in Niemann-Pick Type C Disease

This study has been completed.
Information provided by:
Actelion Identifier:
First received: August 15, 2007
Last updated: March 24, 2010
Last verified: March 2010
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.

Condition Intervention Phase
Niemann-Pick Type C Disease Drug: miglustat Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Phase II Randomized Controlled Study of Miglustat in Adult and Juvenile Patients With Niemann-Pick Type C Disease

Resource links provided by NLM:

Further study details as provided by Actelion:

Primary Outcome Measures:
  • 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 ]

Secondary Outcome Measures:
  • Highest amplitude for which a velocity measurement is obtained [ Time Frame: Baseline to Month 12 ]

Enrollment: 29
Study Start Date: January 2002
Study Completion Date: January 2008
Primary Completion Date: September 2006 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: 1
OGT-918 - Zavesca (miglustat)
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
No Intervention: 2
Standard treatment


Ages Eligible for Study:   4 Years and older   (Child, Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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.
  Contacts and Locations
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Please refer to this study by its identifier: NCT00517153

United States, New York
Columbia University Medical Center
New York, New York, United States, 10032
Sponsors and Collaborators
Principal Investigator: Claudia Chiriboga, Assoc. Prof. MD Columbia University
  More Information

Responsible Party: Dr. Ruben Giorgino, Actelion Identifier: NCT00517153     History of Changes
Other Study ID Numbers: OGT-918-007
Study First Received: August 15, 2007
Last Updated: March 24, 2010

Keywords provided by Actelion:
Niemann-Pick Type C

Additional relevant MeSH terms:
Niemann-Pick Diseases
Niemann-Pick Disease, Type A
Niemann-Pick Disease, Type C
Lysosomal Storage Diseases, Nervous System
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Lymphatic Diseases
Genetic Diseases, Inborn
Lysosomal Storage Diseases
Metabolic Diseases
Lipid Metabolism Disorders
Histiocytosis, Non-Langerhans-Cell
Metabolism, Inborn Errors
Lipid Metabolism, Inborn Errors
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action
Anti-HIV Agents
Anti-Retroviral Agents
Antiviral Agents
Anti-Infective Agents
Glycoside Hydrolase Inhibitors
Hypoglycemic Agents processed this record on September 21, 2017