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

NCT00672022 · Status: COMPLETED · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 10

Last updated 2008-05-06

No results posted yet for this study

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.

Conditions

  • GM2 Gangliosidoses
  • Tay-Sachs
  • Sandhoff Disease

Interventions

DRUG

Zavesca (Miglustat)

Sponsors & Collaborators

  • Actelion

    collaborator INDUSTRY

  • Children's National Research Institute

    lead OTHER

Principal Investigators

  • Cynthia J TIfft, MD, PhD · Children's National Research Institute

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
6 Months
Max Age
5 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2004-07-31
Primary Completion
2007-08-31
Completion
2007-08-31

Countries

  • United States

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 NCT00672022 on ClinicalTrials.gov