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Fabrazyme

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

Ensayo de Sanofi en Fabry Disease (del registro, en inglés).

Fase

Fase 2

Estado

Completado

Participantes

21

previstos

Centros

4

Fin del objetivo primario

abr 2006

fecha real

Qué significa cada fase y cada estado.

Estudio de intervención, de un solo grupo, abierto. Comenzó en jun 2003.

Qué mide

Globotriaosylceramide (GL-3) Clearance in Kidney Interstitial Capillary Endothelium (Throughout study; 96 weeks) (del registro, en inglés)

Cómo lo describe el promotor

En el documentoEn inglés, del registroPeople with Fabry disease have an alteration in their genetic material (DNA) which causes a deficiency of the alpha-galactosidase A enzyme. This enzyme helps to break down and remove certain types of fatty substances called "glycolipids." These glycolipids are normally present within the body in most cells. In people with Fabry disease, glycolipids build up in various tissues such as the liver, kidney, skin, and blood vessels because alpha-galactosidase A is not present, or is present in small quantities. The build up of glycolipid levels (also referred to as "globotriaosylceramide" or "GL-3") in these tissues is thought to cause the clinical symptoms that are common to Fabry disease. Symptoms commonly appear during childhood with pain in the hands and feet. This trial is designed to evaluate the efficacy of a lower dose of Fabrazyme in patients who initially received 1.0 mg/kg every 2 weeks of Fabrazyme by investigating if the achieved clearance of glycosphingolipid deposits in the vascular endothelium of the kidney can be maintained at a lower dose.

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Ficha completa en ClinicalTrials.gov (NCT00196716), actualizada en mar 2015.