NCT00672022

Pharmacokinetics, Safety and Tolerability of Zavesca (Miglustat) in Patients With Infantile Onset Gangliosidosis: Single and Steady State Oral Doses

Pharmacokinetics, Safety and Tolerability of Zavesca (Miglustat) in Patients With Infantile Onset GM2 Gangliosidosis: Single and Steady State Oral Doses

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 10 (estimated)
• Sponsor: Children's National Research Institute · Academic / Other
• Sex: All
• Age: 6 Months – 5 Years
• Healthy volunteers: Not accepted

Summary

We want to see if Zavesca (or miglustat) is safe and can be tolerated by patients with acute infantile onset GM2 gangliosidosis - classical Tay-Sachs and infantile onset Sandhoff disease. We know that miglustat inhibits the formation of GM2 ganglioside, the compound that is stored in the brains of children with Tay-Sachs and Sandhoff disease. Since it inhibits the synthesis of ganglioside, miglustat may be able to reduce or delay the onset of clinical symptoms.

Detailed description

Specific Aims The primary objective of the study is to investigate the pharmacokinetics of ZAVESCA® (miglustat, OGT918), when given as a single dose and at steady state, in infantile patients with GM2 gangliosidosis. The secondary objectives are to evaluate the tolerability and safety of single and multiple doses of miglustat and to monitor disease progression using physical and developmental assessments and disease-specific biomarkers.

Conditions

• GM2 Gangliosidoses
• Tay-Sachs
• Sandhoff Disease

Interventions

Type	Name	Description
DRUG	Zavesca (Miglustat)

Timeline

• Start date: 2004-07-01
• Primary completion: 2007-08-01
• Completion: 2007-08-01
• First posted: 2008-05-06
• Last updated: 2008-05-06

Locations

1 site across 1 country: United States