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

Status: Completed
Location: See location...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 2

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.

Participation Requirements
Sex: All
Minimum Age: 4
Healthy Volunteers: No

• 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.

United States
New York
Columbia University Medical Center
New York
Time Frame
Start Date: January 2002
Completion Date: January 2008
Target number of participants: 29
Experimental: 1
OGT-918 - Zavesca (miglustat)
No Intervention: 2
Standard treatment
Leads: Actelion

This content was sourced from clinicaltrials.gov

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