Calendario›SNY›alglucosidase alfa

alglucosidase alfa

Late-Onset Treatment Study Extension Protocol

Ensayo de Sanofi en Pompe Disease (Late-Onset) · Glycogen Storage Disease Type II (GSD-II) · Glycogenesis Type II · Acid Maltase Deficiency (AMD) (del registro, en inglés).

Fase

Fase 4

Estado

Completado

Participantes

81

previstos

Centros

31

Fin del objetivo primario

oct 2008

fecha real

Qué significa cada fase y cada estado.

Estudio de intervención, no aleatorizado, abierto. Comenzó en mar 2007.

Qué mide

Summary of Participants Reporting Treatment-Emergent Adverse Events For Participants Treated With Alglucosidase Alfa During Study AGLU02704 (NCT00158600) (del registro, en inglés) (de la semana 0 a los 2,5 años)

Cómo lo describe el promotor

En el documentoEn inglés, del registroPompe disease (also known as glycogen storage disease Type II) is caused by a deficiency of a critical enzyme in the body called acid alpha-glucosidase (GAA). Normally, GAA is used by the body's cells to break down glycogen (a stored form of sugar) within specialized structures called lysosomes. In patients with Pompe disease, an excessive amount of glycogen accumulates and is stored in various tissues, especially heart and skeletal muscle, which prevents their normal function. The objective of this extension study is to assess the long-term safety and efficacy of alglucosidase alfa treatment in patients with Late-Onset Pompe Disease who were previously treated under the placebo-controlled, double-blind study AGLU02704 (NCT00158600).

Más ensayos de SNY

Todos los de SNY, en su ficha.

Ficha completa en ClinicalTrials.gov (NCT00455195), actualizada en feb 2014.