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

Phase 4

Completed

Brief 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.

Recruitment & Eligibility

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Arm && Interventions

Group	Intervention	Description
Alglucosidase Alfa	ALGLUCOSIDASE ALFA	A dose of 20 mg/kg body weight once every 2 weeks for a minimum of 52 weeks

Primary Outcome Measures

Name	Time	Method
Change from baseline in Six-minute walk test (6MWT) for the patients ≥5-year old	Baseline to 12 months	This test measures the distance that a patient can quickly walk on a flat, hard surface in a period of 6 minutes.
Number of participants with adverse events Number of AEs	From signing the ICF to the 30th day after the last dosage of the study medications	An adverse event (AE) is any untoward medical occurrence in a patient or clinical investigation patient administered a pharmaceutical product and which does not necessarily have to have a causal relationship with this treatment.
Change from baseline in percent predicted forced vital capacity (%FVC) in upright position for the patients ≥5-year old	Baseline to 12 months	The measurements of pulmonary function and respiratory strength including FVC, MEP and MIP use a pneumograph or the spirometry system with the patient in upright seated and supine positions, according to American Thoracic Society (ATS) and European Respiratory Society (ERS) guidelines.

Secondary Outcome Measures

Name	Time	Method
Change from baseline in maximal inspiratory pressure (MIP) in upright position for the patients ≥5-year old	Week 52	The measurements of pulmonary function and respiratory strength including FVC, MEP and MIP use a pneumograph or the spirometry system with the patient in upright seated and supine positions, according to American Thoracic Society (ATS) and European Respiratory Society (ERS) guidelines.
Change from baseline in maximal expiratory pressure (MEP) in upright position for the patients ≥5-year old	Week 52	The measurements of pulmonary function and respiratory strength including FVC, MEP and MIP use a pneumograph or the spirometry system with the patient in upright seated and supine positions, according to American Thoracic Society (ATS) and European Respiratory Society (ERS) guidelines.
Change from baseline in manual muscle test (MMT) for deltoid muscle, quadriceps femoris, iliopsoas, neck stretch flexor for the patients ≥5-year old	Week 52	MMT has been reported most often as a summary score of a total number of proximal, distal, and axial muscle groups tested bilaterally or as a proximal score that sums a number of proximal muscle groups from the upper and lower extremities.
Change from baseline in Quick Motor Function Test scores for the patients ≥5-year old	Week 52	The 12-Item Short Form Health Survey (SF-12) was developed for the Medical Outcomes evaluation of patients with chronic conditions.