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alglucosidase alfa

Expanded Access Use of Myozyme (Alglucosidase Alfa) in Patients With Infantile-onset Pompe Disease

Ensayo de Sanofi en Glycogen Storage Disease Type II · Glycogenosis 2 (del registro, en inglés).

Fase

no aplica

Estado

Aprobado para comercializar

Fin del objetivo primario

feb 2007

fecha real

Qué significa cada fase y cada estado.

Estudio de acceso ampliado. Comenzó en dic 2003.

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 protocol is to provide enzyme replacement therapy with rhGAA on an expanded access basis, to severely affected patients with infantile-onset Pompe disease for whom there is no alternative treatment and who do not meet the clinical characteristics described in the inclusion criteria for participation in other Genzyme Corporation-sponsored study currently enrolling patients with infantile-onset Pompe disease.

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Ficha completa en ClinicalTrials.gov (NCT00074919), actualizada en feb 2014.