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

Sponsored by:	Actelion
Information provided by:	Actelion
ClinicalTrials.gov Identifier:	NCT00517153

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 II

Drug Information available for: SC 48334

U.S. FDA Resources

Study Type:	Interventional
Study Design:	Treatment, Randomized, Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Historical Control, Parallel Assignment, Safety/Efficacy Study
Official Title:	A Phase II Randomized Controlled Study of Miglustat in Adult and Juvenile Patients With Niemann-Pick Type C Disease

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 ]

Estimated Enrollment:	42
Study Start Date:	January 2002
Estimated Study Completion Date:	January 2009
Estimated Primary Completion Date:	January 2009 (Final data collection date for primary outcome measure)

Arms	Assigned Interventions
1: Experimental 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
2: No Intervention 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 Medical Center

More Information

No publications provided

Responsible Party:	Actelion ( Dr. Ruben Giorgino )
Study ID Numbers:	OGT-918-007
Study First Received:	August 15, 2007
Last Updated:	August 26, 2008
ClinicalTrials.gov Identifier:	NCT00517153 History of Changes
Health Authority:	United States: Food and Drug Administration; United States: Institutional Review Board

Keywords provided by Actelion:

Zavesca
miglustat
Niemann-Pick Type C
Actelion

Study placed in the following topic categories:

Miglustat
Anti-HIV Agents
Anti-Retroviral Agents
Antiviral Agents

Additional relevant MeSH terms:

Anti-Infective Agents
Miglustat
Anti-HIV Agents
Anti-Retroviral Agents
Molecular Mechanisms of Pharmacological Action

Therapeutic Uses
Enzyme Inhibitors
Antiviral Agents
Pharmacologic Actions

ClinicalTrials.gov processed this record on May 07, 2009