Miglustat in Niemann-Pick Type C Disease - Full Text View

Purpose

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 Endpoint Classification: Safety/Efficacy Study Intervention Model: Parallel Assignment Masking: Double Blind (Subject, Caregiver, 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:

Genetics Home Reference related topics: Chanarin-Dorfman syndrome CHMP2B-related frontotemporal dementia cholesteryl ester storage disease Farber lipogranulomatosis frontotemporal dementia with parkinsonism-17 GRN-related frontotemporal dementia inclusion body myopathy with early-onset Paget disease and frontotemporal dementia Niemann-Pick disease Schindler disease succinic semialdehyde dehydrogenase deficiency

Drug Information available for: Miglustat

U.S. FDA Resources

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 ] [ Designated as safety issue: No ]

Secondary Outcome Measures:

Highest amplitude for which a velocity measurement is obtained [ Time Frame: Baseline to Month 12 ] [ Designated as safety issue: No ]

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

Eligibility

Ages Eligible for Study:	4 Years and older
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

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.

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00517153

Locations

United States, New York
Columbia University Medical Center
New York, New York, United States, 10032

Sponsors and Collaborators

Actelion

Investigators

Principal Investigator:

Claudia Chiriboga, Assoc. Prof. MD

Columbia University

More Information

No publications provided

Responsible Party:	Dr. Ruben Giorgino, Actelion
ClinicalTrials.gov Identifier:	NCT00517153 History of Changes
Other Study ID Numbers:	OGT-918-007
Study First Received:	August 15, 2007
Last Updated:	March 24, 2010
Health Authority:	United States: Food and Drug Administration United States: Institutional Review Board

Keywords provided by Actelion:

Zavesca
miglustat
Niemann-Pick Type C
Actelion

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
Sphingolipidoses

Histiocytosis, Non-Langerhans-Cell
Histiocytosis
Metabolism, Inborn Errors
Lipidoses
Lipid Metabolism, Inborn Errors
Miglustat
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action
Pharmacologic Actions
Anti-HIV Agents
Anti-Retroviral Agents
Antiviral Agents
Anti-Infective Agents
Therapeutic Uses

ClinicalTrials.gov processed this record on May 16, 2013