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Alglucosidase Alfa

Exploratory Muscle Biopsy Assessment Study in Patients With Late-Onset Pompe Disease Treated With Alglucosidase Alfa

Ensayo de Sanofi en Pompe Disease (Late-Onset) · Glycogen Storage Disease Type II (GSD II) · Glycogenesis 2 Acid Maltase Deficiency (del registro, en inglés).

Fase

Fase 4

Estado

Completado

Participantes

16

previstos

Centros

15

Fin del objetivo primario

dic 2013

fecha real

Qué significa cada fase y cada estado.

Estudio de intervención, no aleatorizado, abierto. Comenzó en jun 2011.

Qué mide

Change From Baseline in Tissue Glycogen Content in Quadriceps Muscle Biopsy Samples at Week 26 (del registro, en inglés) (inicio y semana 26)

Cómo lo describe el promotor

En el documentoEn inglés, del registroThis is an open-label, multicenter study of participants with late-onset Pompe disease naive to treatment with enzyme replacement therapy (ERT). The primary objective of this study is to evaluate glycogen clearance in muscle tissue samples collected pre and post alglucosidase alfa treatment in participants with Late-Onset Pompe disease. The secondary objectives are to characterize the disease burden in participants with late-onset Pompe disease and explore imaging, histologic, and functional assessments in these participants and to explore potential plasma or urine biomarkers relative to late-onset Pompe disease and participant's response to treatment with alglucosidase alfa (Myozyme®/Lumizyme®/GZ419829).

Más ensayos de SNY

Todos los de SNY, en su ficha.

Ficha completa en ClinicalTrials.gov (NCT01288027), actualizada en dic 2014.