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.
Study duration per participants is approximatively 56 weeks including a 52-week treatment period.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
41
Pharmaceutical form:solution for infusion Route of administration: intravenous
Investigational Site
China, China
Change from baseline in Six-minute walk test (6MWT) for the patients ≥5-year old
This test measures the distance that a patient can quickly walk on a flat, hard surface in a period of 6 minutes.
Time frame: Baseline to 12 months
Change from baseline in percent predicted forced vital capacity (%FVC) in upright position for the patients ≥5-year old
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.
Time frame: Baseline to 12 months
Number of participants with adverse events Number of AEs
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.
Time frame: From signing the ICF to the 30th day after the last dosage of the study medications
Change from baseline in maximal inspiratory pressure (MIP) in upright position for the patients ≥5-year old
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.
Time frame: Week 52
Change from baseline in maximal expiratory pressure (MEP) in upright position for the patients ≥5-year old
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.
Time frame: Week 52
Change from baseline in manual muscle test (MMT) for deltoid muscle, quadriceps femoris, iliopsoas, neck stretch flexor for the patients ≥5-year old
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.
Time frame: Week 52
Change from baseline in Quick Motor Function Test scores for the patients ≥5-year old
The Quick Motor Function Test is a reliable and valid test for assessing motor function in patients with Pompe's disease.
Time frame: Week 52
Change from baseline in Quick Motor Function Test scores for the patients ≥5-year old
The 12-Item Short Form Health Survey (SF-12) was developed for the Medical Outcomes evaluation of patients with chronic conditions.
Time frame: Week 52
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