Miglustat in Niemann-Pick Type C Disease

• ClinicalTrials.gov Identifier: NCT00517153
• Recruitment Status: Completed
• First Posted: August 16, 2007
• Last Update Posted: March 26, 2010
• Sponsor: Actelion
• Information provided by: Actelion

Tracking Information

• First Submitted Date: August 15, 2007
• First Posted Date: August 16, 2007
• Last Update Posted Date: March 26, 2010
• Study Start Date: January 2002
• Actual Primary Completion Date: September 2006 (Final data collection date for primary outcome measure)
• Current Primary Outcome Measures (submitted: August 26, 2008): 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 ]
• Original Primary Outcome Measures: Not Provided
• Current Secondary Outcome Measures (submitted: August 26, 2008): Highest amplitude for which a velocity measurement is obtained [ Time Frame: Baseline to Month 12 ]
• Original Secondary Outcome Measures: Not Provided
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: Miglustat in Niemann-Pick Type C Disease
• Official Title: A Phase II Randomized Controlled Study of Miglustat in Adult and Juvenile Patients With Niemann-Pick Type C Disease
• Brief Summary: 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.
• Detailed Description: Not Provided
• Study Type: Interventional
• Study Phase: Phase 2
• Study Design: Allocation: Randomized; Intervention Model: Parallel Assignment; Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor); Primary Purpose: Treatment
• Condition: Niemann-Pick Type C Disease

Intervention

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

Study Arms

• Experimental: 1
  OGT-918 - Zavesca (miglustat)
  Intervention: Drug: miglustat
• No Intervention: 2
  Standard treatment

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Completed
• Actual Enrollment (submitted: March 24, 2010): 29
• Original Estimated Enrollment (submitted: August 15, 2007): 42
• Actual Study Completion Date: January 2008
• Actual Primary Completion Date: September 2006 (Final data collection date for primary outcome measure)

Eligibility Criteria

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.

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 4 Years and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: United States

Administrative Information

• NCT Number: NCT00517153
• Other Study ID Numbers: OGT-918-007
• Has Data Monitoring Committee: No
• U.S. FDA-regulated Product: Not Provided
• IPD Sharing Statement: Not Provided
• Current Responsible Party: Dr. Ruben Giorgino, Actelion
• Original Responsible Party: Not Provided
• Current Study Sponsor: Actelion
• Original Study Sponsor: Same as current
• Collaborators: Not Provided

Investigators

• Principal Investigator: Claudia Chiriboga, Assoc. Prof. MD; Columbia University

• PRS Account: Actelion
• Verification Date: March 2010