Pharmacokinetics and Tolerability of Zavesca® (Miglustat) In Patients With Juvenile GM2 Gangliosidosis
Recruitment status was Active, not recruiting
The purpose of the study is to investigate the pharmacokinetics of Zavesca (miglustat, OGT918) when given as single and multiple doses in juvenile patients with GM2 gangliosidosis.
|Study Design:||Allocation: Non-Randomized
Endpoint Classification: Pharmacokinetics Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||Pharmacokinetics and Tolerability of Zavesca® (Miglustat) In Patients With Juvenile GM2 Gangliosidosis: Single and Multiple Oral Doses|
- Concentration of miglustat in plasma [ Time Frame: Periodic intervals up to 24 hours ] [ Designated as safety issue: No ]
- Changes in volume loss and signal intensity from baseline MRI [ Time Frame: 12 months ] [ Designated as safety issue: No ]
- Change in single-voxel N acetylaspartate (NAA) from baseline MRS [ Time Frame: 1 month, 3 months, 6 months, 9 months, and 12 months ] [ Designated as safety issue: No ]
- Change in neuropsychological testing from baseline [ Time Frame: 6 months and 12 months ] [ Designated as safety issue: No ]
- Change in nerve conduction [ Time Frame: 6 months and 12 months ] [ Designated as safety issue: No ]
- Change in neurological examination from baseline [ Time Frame: 1 month, 3 months, 6 months, 9 months, and 12 months ] [ Designated as safety issue: No ]
|Study Start Date:||July 2004|
|Estimated Study Completion Date:||October 2006|
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.
Other Name: Zavesca
The GM2 gangliosidoses are a group of neuro-degenerative lysosomal storage diseases resulting from accumulation of GM2 and related glycolipids in the central nervous system (CNS). Tay-Sachs and Sandhoff disease are two variants which are indistinguishable in clinical grounds. According to the onset and rate of disease progression, the condition can be categorized in infantile, juvenile and adult forms. This open-label, single-arm study is designed to assess the pharmacokinetics, safety and tolerability of miglustat in juvenile patients. Miglustat will be administered at a maximum dose of 600 mg/day, divided into three doses per day. The dose used for patients in this pediatric age range will be related to the patient's body surface area. The pharmacokinetics assessments for the study will be performed in-hospital during a 24 hour period, and will take place at the day one and at the month 3 visits. The clinical (which includes safety and tolerability) assessments will be performed throughout the 24-month study period.
|The Hospital for Sick Children|
|Toronto, Ontario, Canada, M5G 1X8|
|Principal Investigator:||Joe TR Clarke, MD||The Hospital for Sick Children, Toronto Canada|