A Study of the Safety and Efficacy of Fabrazyme in Patients With Fabry Disease
Status:
Completed
Trial end date:
2004-12-01
Target enrollment:
Participant gender:
Summary
People with Fabry disease have an alteration in their genetic material (DNA) which causes a
deficiency of the a-galactosidase A enzyme. Fabrazyme 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
("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 will test the safety and
efficacy of Fabrazyme in the treatment of patients with Fabry disease.