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Alglucosidase alfa

A Study of the Safety and Pharmacokinetics of rhGAA in Siblings With Glycogen Storage Disease Type II

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

Fase

Fase 2

Estado

Completado

Participantes

2

previstos

Centros

1

Fin del objetivo primario

abr 2003

fecha real

Qué significa cada fase y cada estado.

Estudio de intervención, no aleatorizado, abierto. Comenzó en ene 2003.

Qué mide

Evaluate safety, pharmacokinetics and pharmacodynamics (del registro, en inglés) (52 semanas)

Cómo lo describe el promotor

En el documentoEn inglés, del registroGSD-II (also known as Pompe disease) 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 GSD-II, 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, pharmacokinetics, pharmacodynamics and efficacy of recombinant human acid alpha-glucosidase (rhGAA) as a potential enzyme replacement therapy for a pair of siblings with GSD-II. To be eligible for this study, a patient must have a confirmed diagnosis of GSD-II and have a sister or brother who also has a confirmed diagnosis of GSD-II.

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Ficha completa en ClinicalTrials.gov (NCT00051935), actualizada en feb 2014.