Study to Evaluate Efficacy and Safety in Chinese Patients With Late Onset Pompe Disease With Alglucosidase Alfa Treatmen — APOLLO-LOPD  

Pompe's Disease Clinical Trial

Official title: A Single Arm, Prospective, Open-label, Multi-center Study to Evaluate Efficacy and Safety in Chinese Patients With Late Onset Pompe Disease With Alglucosidase Alfa Treatment

Status Active, not recruiting

Clinical Trial 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.

Clinical Trial Description

Study duration per participants is approximatively 56 weeks including a 52-week treatment period. ;

Related Conditions & MeSH terms

• Glycogen Storage Disease Type II
• Pompe's Disease

• NCT number: NCT04676373
• Study type: Interventional
• Source: Sanofi
• Status: Active, not recruiting
• Phase: Phase 4
• Start date: March 10, 2021
• Completion date: July 25, 2024