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

NCT04676373 · Status: COMPLETED · Phase: PHASE4 · Type: INTERVENTIONAL · Enrollment: 41

Last updated 2025-02-10

No results posted yet for this study

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.

Conditions

  • Pompe's Disease

Interventions

DRUG

ALGLUCOSIDASE ALFA

Pharmaceutical form:solution for infusion Route of administration: intravenous

Sponsors & Collaborators

  • Genzyme, a Sanofi Company

    lead INDUSTRY

Principal Investigators

  • Clinical Sciences & Operations · Sanofi

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
3 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2021-03-10
Primary Completion
2024-07-25
Completion
2024-07-25

Countries

  • China

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04676373 on ClinicalTrials.gov