Application of Miglustat in Patients With Niemann-Pick Type C

Phase 1: Researchers test a new drug or treatment in a small group of people for the first time to evaluate its safety, determine a safe dosage range, and identify side effects.
Phase 2: The drug or treatment is given to a larger group of people to see if it is effective and to further evaluate its safety.
Phase 3: The drug or treatment is given to large groups of people to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the drug or treatment to be used safely.
Phase 4: Studies are done after the drug or treatment has been marketed to gather information on the drug's effect in various populations and any side effects associated with long-term use.

Niemann-Pick Disease Type C Clinical Trial

Official title:
Application of Miglustat in Patients With Niemann-Pick Type C

Status Completed

Clinical Trial Summary

To evaluate the changes in cognitive function after miglustat treatment in Niemann-Pick type C patients.

Patients with Niemann-Pick C disease will be treated with Miglutat 200mg tid. Neurologic evaluation and safety will be monitored periodically. ;

Endpoint Classification: Safety Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

NCT number	NCT01760564
Study type	Interventional
Source	National Taiwan University Hospital
Contact
Status	Completed
Phase	Phase 3
Start date	January 2008
Completion date	December 2010