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Myozyme

A Study of the Safety and Efficacy of rhGAA in Patients With Infantile-onset Pompe Disease

Ensayo de Sanofi en Glycogen Storage Disease Type II (del registro, en inglés).

Fase

Fase 2/3

Estado

Completado

Participantes

16

previstos

Centros

8

Fin del objetivo primario

jun 2005

fecha real

Qué significa cada fase y cada estado.

Estudio de intervención, aleatorizado, abierto. Comenzó en abr 2003.

Qué mide

Evaluate the safety profile of MZ (del registro, en inglés) (52 semanas)

Cómo lo describe el promotor

En el documentoEn inglés, del registroPompe disease (also known as glycogen storage disease type II, "GSD-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. This study is being conducted to evaluate the safety and effectiveness of recombinant human acid alpha-glucosidase (rhGAA) as a potential enzyme replacement therapy for Pompe disease. Patients diagnosed with infantile-onset Pompe disease who are less than or equal to 6 months old will be studied.

Más ensayos de SNY

Todos los de SNY, en su ficha.

Ficha completa en ClinicalTrials.gov (NCT00059280), actualizada en feb 2014.