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Miglustat in Niemann-Pick Type C Disease

Primary Purpose

Niemann-Pick Type C Disease

Status

Completed

Phase

Phase 2

Locations

United States

Study Type

Interventional

Intervention

miglustat

About this trial

This is an interventional treatment trial for Niemann-Pick Type C Disease focused on measuring Zavesca, miglustat, Niemann-Pick Type C, Actelion

Eligibility Criteria

4 Years - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

Columbia University Medical Center

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

No Intervention

Arm Label

Arm Description

OGT-918 - Zavesca (miglustat)

Standard treatment

Outcomes

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

Secondary Outcome Measures

Highest amplitude for which a velocity measurement is obtained

Full Information

NCT ID

NCT00517153

First Posted

August 15, 2007

Last Updated

March 24, 2010

Sponsor

Actelion

1. Study Identification

Unique Protocol Identification Number

NCT00517153

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

Study Type

Interventional

2. Study Status

Record Verification Date

March 2010

Overall Recruitment Status

Completed

Study Start Date

January 2002 (undefined)

Primary Completion Date

September 2006 (Actual)

Study Completion Date

January 2008 (Actual)

3. Sponsor/Collaborators

Name of the Sponsor

Actelion

4. Oversight

Data Monitoring Committee

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Niemann-Pick Type C Disease

Keywords

Zavesca, miglustat, Niemann-Pick Type C, Actelion

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 2

Interventional Study Model

Parallel Assignment

Masking

ParticipantCare ProviderInvestigatorOutcomes Assessor

Allocation

Randomized

Enrollment

29 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Arm Type

Experimental

Arm Description

OGT-918 - Zavesca (miglustat)

Arm Title

Arm Type

No Intervention

Arm Description

Standard treatment

Intervention Type

Drug

Intervention Name(s)

miglustat

Other Intervention Name(s)

Zavesca

Intervention Description

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

Primary Outcome Measure Information:

Title

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 Measure Information:

Title

Highest amplitude for which a velocity measurement is obtained

Time Frame

Baseline to Month 12

10. Eligibility

Sex

All

Minimum Age & Unit of Time

4 Years

Accepts Healthy Volunteers

Eligibility 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.

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Claudia Chiriboga, Assoc. Prof. MD

Organizational Affiliation

Columbia University

Official's Role

Principal Investigator

Facility Information:

Facility Name

Columbia University Medical Center

City

New York

State/Province

New York

ZIP/Postal Code

10032

Country

United States

12. IPD Sharing Statement

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Miglustat in Niemann-Pick Type C Disease

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