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

NCT00418847 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 5

Last updated 2016-05-19

No results posted yet for this study

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.

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.

Sponsors & Collaborators

  • Actelion

    collaborator INDUSTRY

  • The Hospital for Sick Children

    lead OTHER

Principal Investigators

  • Joe TR Clarke, MD · The Hospital for Sick Children, Toronto Canada

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
6 Years
Max Age
20 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2004-07-31
Primary Completion
2009-04-30
Completion
2009-04-30

Countries

  • Canada

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT00418847 on ClinicalTrials.gov