Miglustat in Niemann-Pick Type C Disease

Phase 2

Completed

• Conditions: Niemann-Pick Type C Disease
• Interventions: Drug: miglustat

• Registration Number: NCT00517153
• Lead Sponsor: Actelion

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 available

Study Timeline

• Study Start: 2002-01
• Primary Completion: 2006-09
• Study First Submitted: 2007-08-15
• Study First Submitted QC: 2007-08-15
• Study First Posted: 2007-08-16
• Study Completion: 2008-01
• Disposition First Submitted: 2010-02-11
• Disposition First Submitted QC: 2010-02-11
• Disposition First Posted: 2010-02-15
• Status Verified: 2025-01
• Last Update Submitted: 2025-01-31
• Last Update Posted: 2025-02-04

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 29

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
1	miglustat	OGT-918 - Zavesca (miglustat)

Primary Outcome Measures

Name	Time	Method
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	Baseline to Month 12

Secondary Outcome Measures

Name	Time	Method
Highest amplitude for which a velocity measurement is obtained	Baseline to Month 12

Trial Locations

Locations (1)

Columbia University Medical Center; 🇺🇸 New York, New York, United States