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Fabrazyme
A Study of Fabrazyme in Pediatric Patients With Fabry Disease
Ensayo de Sanofi en Fabry Disease (del registro, en inglés).
- Fase
- Fase 2
- Estado
- Completado
- Participantes
- 16
- Centros
- 7
- Fin del objetivo primario
- may 2005
previstos
fecha real
Qué significa cada fase y cada estado.
Estudio de intervención, de un solo grupo, abierto. Comenzó en oct 2002.
Qué mide
Globotriaosylceramide (GL-3) Clearance in Capillary Endothelium in the Skin (Baseline, Week 24 and Week 48) (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 a-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 a-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 study explored the safety, efficacy and pharmacokinetics of Fabrazyme in pediatric patients aged between 7 and 15 years.
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Ficha completa en ClinicalTrials.gov (NCT00074958), actualizada en mar 2015.