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Myozyme
Safety and Effectiveness Study of rhGAA in Patients With Advanced Late-Onset Pompe Disease Receiving Respiratory Support
Ensayo de Sanofi en Pompe Disease (Late-onset) · Glycogen Storage Disease Type II (GSD-II) · Acid Maltase Deficiency Disease · Glycogenosis 2 (del registro, en inglés).
- Fase
- Fase 3
- Estado
- Completado
- Participantes
- 5
- Centros
- 1
- Fin del objetivo primario
- mar 2007
previstos
fecha real
Qué significa cada fase y cada estado.
Estudio de intervención, no aleatorizado, abierto. Comenzó en dic 2005.
Qué mide
Treatment effect on muscle strength and functional status. (six months and one year) (del registro, en inglés)
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 overall objective is to evaluate the safety and efficacy of rhGAA in patients with advanced Late-onset Pompe disease.
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Ficha completa en ClinicalTrials.gov (NCT00268944), actualizada en feb 2014.