NCT00364858

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

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
• Phase: Phase 4
• Study type: Interventional
• Enrollment: 95 (actual)
• Sponsor: Genzyme, a Sanofi Company · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

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.

Conditions

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

Interventions

Type	Name	Description
DRUG	Cerezyme	Cerezyme doses of 20-60U/kg every 2 weeks (Q2 Arm) or 40-120 U/kg every 4 weeks (Q4 Arm).

Timeline

• Start date: 2001-12-01
• Completion: 2007-02-01
• First posted: 2006-08-16
• Last updated: 2015-04-03
• Results posted: 2009-09-02

Locations

26 sites across 7 countries: United States, Brazil, Canada, Italy, Poland, Spain, United Kingdom