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alglucosidase alfa

Growth and Development Study of Alglucosidase Alfa

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

Fase

Fase 4

Estado

Completado

Participantes

12

previstos

Centros

3

Fin del objetivo primario

nov 2021

fecha real

Qué significa cada fase y cada estado.

Estudio de intervención, de un solo grupo, abierto. Comenzó en ago 2008.

Qué mide

Recumbent Height/Length of Participants in Centimeters (cm) (Participants 1-12:Baseline, Participant1: Wk 52, Participant2: Wk82, Participants 3-4: Wk208, Participant5: Wk12, Participant6: Wk365, Participant7: Wk64, Participant8:Wk156, Participant9:Wk364, Participant10:Wk52, Participant11:Wk156, Participant12:Wk520) (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 a rare autosomal recessive metabolic muscle disease caused by the deficiency of acid α glucosidase (GAA), an enzyme that degrades lysosomal glycogen. As opposed to the exclusively cytoplasmic accumulation of glycogen that occurs in other glycogen storage disorders, Pompe disease is characterized by organelle bound (lysosomal) and extra-lysosomal accumulation of glycogen in many body tissues, ultimately leading to multisystemic pathology. The overall objective of this study was to evaluate the long-term growth and development of participants with infantile-onset Pompe disease with alglucosidase alfa before 1 year of age. Participants were to be followed for a 10-year period.

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Todos los de SNY, en su ficha.

Ficha completa en ClinicalTrials.gov (NCT00486889), actualizada en jul 2022.