Safety and Efficacy of Cerezyme® Infusions Every 4 Weeks Versus Every 2 Weeks in Type 1 Gaucher Disease

Gaucher Disease, Type 1 Clinical Trial

Official title: A Phase IV, Multicenter, Randomized, Dose Frequency Study of the Safety and Efficacy of Cerezyme® Infusions Every Four Weeks Versus Every Two Weeks in the Maintenance Therapy of Patients With Type 1 Gaucher Disease

Status Completed

Clinical Trial Summary

This is a multicenter, randomized trial to compare the safety and efficacy of two dosing frequencies of Cerezyme® in patients with Gaucher disease who are currently being treated with Cerezyme®.

Approximately 90 patients will be randomized in a 2:1 (q4 : q2) ratio to one of two treatment arms at up to 26 study centers worldwide. Patients will continue to receive the same total 4-week dose that they were receiving prior to study enrollment, however, they will be randomized to receive either their total 4-week dose in two infusions, one infusion every 2 weeks or their total 4-week dose in one infusion every 4 weeks. The randomization scheme will ensure a 2:1 balance between the every 4-week versus every 2-week infusion groups, respectively.

Clinical Trial Description

n/a

Study Design

Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Cerebroside Lipidosis Syndrome
• Deficiency Diseases
• Gaucher Disease
• Gaucher Disease, Non-Neuronopathic Form
• Gaucher Disease, Type 1
• Glucocerebrosidase Deficiency Disease
• Glucosylceramide Beta-Glucosidase Deficiency Disease

• NCT number: NCT00364858
• Study type: Interventional
• Source: Sanofi
• Status: Completed
• Phase: Phase 4
• Start date: December 2001
• Completion date: February 2007