NCT00074984

A Study of the Safety and Efficacy of Fabrazyme (Agalsidase Beta) as Compared to Placebo in Patients With Advanced Fabry Disease

Multi-Center, Randomized, Double-Blind, Placebo-Controlled Study of the Safety and Efficacy of Fabrazyme on Progression of Renal Disease and Significant Clinical Events in Patients With Fabry Disease

• Status: Completed
• Phase: Phase 4
• Study type: Interventional
• Enrollment: 82 (actual)
• Sponsor: Genzyme, a Sanofi Company · Industry
• Sex: All
• Age: 16 Years
• Healthy volunteers: Not accepted

Summary

People with Fabry disease have an alteration in their genetic material (DNA) which causes a deficiency of the a-galactosidase A enzyme. Fabrazyme (agalsidase beta) is a drug that helps to breakdown and remove certain types of fatty substances called "glycolipids." These glycolipids are normally present within the body in most cells. In Fabry disease, glycolipids build up in various tissues such as the liver, kidney, skin, and blood vessels because a-galactosidase A is not present, or is present in small quantities. The build up of glycolipid ("globotriaosylceramide" or "GL-3") levels in these tissues in particular is thought to cause the clinical symptoms that are common to Fabry disease. This study will test the safety and efficacy of Fabrazyme in the treatment of patients with Fabry disease.

Conditions

• Fabry Disease

Interventions

Type	Name	Description
BIOLOGICAL	Fabrazyme (agalsidase beta)	1mg/kg Fabrazyme (agalsidase beta) every 2 weeks
BIOLOGICAL	Placebo	1 mg/kg placebo intravenously every 2 weeks

Timeline

• Start date: 2001-02-01
• Primary completion: 2004-01-01
• Completion: 2004-01-01
• First posted: 2003-12-25
• Last updated: 2013-12-27
• Results posted: 2010-12-15

Locations

26 sites across 6 countries: United States, Canada, Czechia, Hungary, Poland, United Kingdom