Ignite Creation Date:
2024-05-05 @ 5:14 PM
Last Modification Date:
2024-10-26 @ 9:30 AM
Study NCT ID:
NCT00418847
Status:
COMPLETED
Last Update Posted:
2016-05-19
First Post:
2007-01-04
Brief Title:
Pharmacokinetics and Tolerability of Zavesca Miglustat In Patients With Juvenile GM2 Gangliosidosis
Sponsor:
The Hospital for Sick Children
Organization:
The Hospital for Sick Children
Study Overview
Official Title:
Pharmacokinetics and Tolerability of Zavesca Miglustat In Patients With Juvenile GM2 Gangliosidosis Single and Multiple Oral Doses
Status:
COMPLETED
Status Verified Date:
2016-05
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
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
Detailed Description:
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 mgday divided into three doses per day The dose used for patients in this pediatric age range will be related to the patients 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
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None