NCT00081497

A Study of the Safety and Efficacy of Fabrazyme in Patients With Fabry Disease

Multi-Center, Open-Label Study of the Safety and Efficacy of Fabrazyme in Patients With Fabry Disease That Previously Participated in the AGAL-008-00 Study

• Status: Completed
• Phase: Phase 4
• Study type: Interventional
• Enrollment: 67 (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 alpha-galactosidase A enzyme. Fabrazyme (agalsidase beta) is a drug that helps to break down and removes 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 (globatriaosylceramide or GL-3) levels in these tissues in particular is thought to cause the clinical symptoms that are common to Fabry disease. This study analyzed the safety and efficacy of Fabrazyme in the treatment of patients with Fabry disease that previously participated in the AGAL-008-00 (NCT0074984) study.

Conditions

• Fabry Disease

Interventions

Type	Name	Description
BIOLOGICAL	agalsidase beta	1.0 mg/kg every 2 weeks

Timeline

• Start date: 2004-01-01
• Primary completion: 2005-09-01
• Completion: 2005-09-01
• First posted: 2004-04-16
• Last updated: 2015-04-02
• Results posted: 2010-08-18

Locations

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