NCT04676373

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

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: Completed
• Phase: Phase 4
• Study type: Interventional
• Enrollment: 41 (actual)
• Sponsor: Genzyme, a Sanofi Company · Industry
• Sex: All
• Age: 3 Years
• Healthy volunteers: Not accepted

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.

Detailed description

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

Conditions

• Pompe's Disease

Interventions

Type	Name	Description
DRUG	ALGLUCOSIDASE ALFA	Pharmaceutical form:solution for infusion Route of administration: intravenous

Timeline

• Start date: 2021-03-10
• Primary completion: 2024-07-25
• Completion: 2024-07-25
• First posted: 2020-12-21
• Last updated: 2025-02-10

Locations

1 site across 1 country: China