Miglustat in Niemann-Pick Type C Disease

Niemann-Pick Type C Disease Clinical Trial

Official title:

A Phase II Randomized Controlled Study of Miglustat in Adult and Juvenile Patients With Niemann-Pick Type C Disease

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00517153
• Other study ID #: OGT-918-007
• Status: Completed
• Phase: Phase 2
• First received: August 15, 2007
• Last updated: March 24, 2010
• Start date: January 2002
• Est. completion date: January 2008

Study information

• Verified date: March 2010
• Source: Actelion
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Food and Drug AdministrationUnited States: Institutional Review Board
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 29
• Est. completion date: January 2008
• Est. primary completion date: September 2006
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 4 Years and older

Eligibility

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

Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Primary Purpose: Treatment

Related Conditions & MeSH terms

• Niemann-Pick Disease, Type C
• Niemann-Pick Diseases
• 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

Locations

Country	Name	City	State
United States	Columbia University Medical Center	New York	New York

Sponsors (1)

Lead Sponsor	Collaborator
Actelion

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	The primary endpoints will be two parameters - Horizontal Saccadic a and Horizontal Saccadic ß - which are estimated for each patient from their saccadic eye movement data		Baseline to Month 12	No
Secondary	Highest amplitude for which a velocity measurement is obtained		Baseline to Month 12	No