Sandhoff Disease Clinical Trial
Official title:
Pharmacokinetics, Safety and Tolerability of Zavesca (Miglustat) in Patients With Infantile Onset GM2 Gangliosidosis: Single and Steady State Oral Doses
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.
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.
;
Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
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