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alglucosidase alfa

High Dose or High Dose Frequency Study of Alglucosidase Alfa

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

Fase

Fase 4

Estado

Completado

Participantes

13

previstos

Centros

11

Fin del objetivo primario

dic 2009

fecha real

Qué significa cada fase y cada estado.

Estudio de intervención, aleatorizado, abierto. Comenzó en may 2007.

Qué mide

Participants' Efficacy Response During the Treatment Period as Compared to Baseline for Participants With Respiratory Decline on Standard Treatment (del registro, en inglés) (inicio y semana 52)

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 exploratory study is to evaluate the safety and efficacy of alternative dosing regimens of alglucosidase alfa in patients with Pompe disease who have not demonstrated an optimal response to the standard dosing regimen of 20 mg/kg every other week after a minimum of 6 months treatment immediately prior to study entry.

Más ensayos de SNY

Todos los de SNY, en su ficha.

Ficha completa en ClinicalTrials.gov (NCT00483379), actualizada en feb 2014.