Study to Evaluate Efficacy and Safety in Chinese Patients With Late Onset Pompe Disease With Alglucosidase Alfa Treatmen
Status:
Recruiting
Trial end date:
2024-12-01
Target enrollment:
Participant gender:
Summary
Primary Objective:
To evaluate the effect of one-year Alglucosidase alfa treatment on motor function [Six-minute
walk test (6MWT) and lung function predicted Forced vital capacity (FVC)] among Chinese Late
Onset Pompe Disease patients above 5 years old.
To evaluate the safety of Myozyme 20mg/kg, IV biweekly in Chinese LOPD patients above 3 years
old.
Secondary Objective:
To evaluate the effect of one-year treatment with Alglucosidase alfa on improvement of manual
muscle test (MMT), Maximal inspiratory and expiratory pressure (MIP and MEP)], Quick Motor
Function Test scores, and health-related quality of life (SF-12) among LOPD patients over 5
years old.