Pharmacokinetics and Tolerability of Zavesca® (Miglustat) In Patients With Juvenile GM2 Gangliosidosis

PHASE2CompletedINTERVENTIONAL
Enrollment

5

Participants

Timeline

Start Date

July 31, 2004

Primary Completion Date

April 30, 2009

Study Completion Date

April 30, 2009

Conditions
Gangliosidoses GM2
Interventions
DRUG

miglustat

Target dose of 320 mg/m\^2/day (divided in 3 doses) will be based on the Body Surface Area (BSA). For children with a BSA \> 1.3, 200 mg TID will be administered. For children with a BSA of 0.8-1.3, 100 mg TID will be administered.

Trial Locations (1)

M5G 1X8

The Hospital for Sick Children, Toronto

Sponsors

Collaborators (1)

All Listed Sponsors
collaborator

Actelion

INDUSTRY

lead

The Hospital for Sick Children

OTHER