NCT00705939

Plant Cell Expressed Recombinant Human Glucocerebrosidase Extension Trial

A Multicenter, Double-Blind, Extension Trial of Two Parallel Dose Groups of Plant Cell Expressed Recombinant Human Glucocerebrosidase (prGCD) in Patients With Gaucher Disease

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 45 (actual)
• Sponsor: Pfizer · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

Gaucher disease, the most prevalent lysosomal storage disorder, is caused by mutations in the human glucocerebrosidase gene (GCD) leading to reduced activity of the lysosomal enzyme glucocerebrosidase and thereby to the accumulation of substrate glucocerebroside (GlcCer) in the cells of the monocyte-macrophage system. This is an extension trial to Study NCT00376168 and NCT00712348.

Detailed description

This will be a multi-center, double-blind, parallel group, extension trial to assess the safety and efficacy of prGCD in patients completing NCT00376168. Patients will receive IV infusion of prGCD every two weeks at the selected medical center. The duration of the extension study will be fifteen months. There will be two treatment groups: 30 units/kg every 2 weeks or 60 units/kg every 2 weeks.

Conditions

• Gaucher Disease

Interventions

Type	Name	Description
DRUG	Taliglucerase alfa	Intravenous infusion every 2 weeks

Timeline

• Start date: 2008-06-01
• Primary completion: 2012-05-01
• Completion: 2013-08-01
• First posted: 2008-06-27
• Last updated: 2018-10-04
• Results posted: 2014-07-15

Locations

11 sites across 8 countries: United States, Australia, Canada, Chile, Israel, South Africa, Spain, United Kingdom