Calendario›SNY›Screening Protocol to Evaluate Acid Alpha-Glucosidase (GAA) Activity and GAA Gene Mutations in Patients With Late Onset Pompe Disease
Screening Protocol to Evaluate Acid Alpha-Glucosidase (GAA) Activity and GAA Gene Mutations in Patients With Late Onset Pompe Disease
Ensayo de Sanofi en Pompe Disease (del registro, en inglés).
- Fase
- no aplica
- Estado
- Completado
- Participantes
- 60
- Centros
- 5
previstos
Qué significa cada fase y cada estado.
Estudio observacional. Comenzó en may 2005.
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 primary objective of this study is to identify potential candidates for future clinical studies in Pompe disease.
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Ficha completa en ClinicalTrials.gov (NCT00113035), actualizada en may 2015.