Trials / Completed
CompletedNCT00517153
Miglustat in Niemann-Pick Type C Disease
A Phase II Randomized Controlled Study of Miglustat in Adult and Juvenile Patients With Niemann-Pick Type C Disease
- Status
- Completed
- Phase
- Phase 2
- Study type
- Interventional
- Enrollment
- 29 (actual)
- Sponsor
- Actelion · Industry
- Sex
- All
- Age
- 4 Years
- Healthy volunteers
- Not accepted
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.
Conditions
Interventions
| Type | Name | Description |
|---|---|---|
| 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 |
Timeline
- Start date
- 2002-01-01
- Primary completion
- 2006-09-01
- Completion
- 2008-01-01
- First posted
- 2007-08-16
- Last updated
- 2025-02-04
Source: ClinicalTrials.gov record NCT00517153. Inclusion in this directory is not an endorsement.