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

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

Official Title
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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

Conditions
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Pompe's Disease

Intervention / Treatment
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* Drug: ALGLUCOSIDASE ALFA


Participation Criteria
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Eligibility Criteria
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Inclusion criteria : Patients (or and patient's legal guardian) must provide written informed consent prior to any study-related procedures The patient must be ≥ 3 years of age at the time of enrollment. For patient ≥ 3-year and < 5-year old: must be able to walk 10 meters or climb 4-step stairs independently. For patients ≥5-year old i. Must be able to ambulate 40 meters in 6 minutes without assistance ii. Must be able to successfully perform repeated forced vital capacity (VC) measurements in upright position of ≥ 30% predicted and ≤85% predicted. The patient has confirmed Pompe's Disease with at least 2 of the following condition, GAA enzyme deficiency from any tissue source. 2 confirmed GAA gene mutations. muscle pathology meet the diagnosis of Pompe disease. The patient (and patient's legal guardian if patient is legally minor as defined by local regulation) must have the ability to comply with the clinical protocol. The patient, if female and of childbearing potential, must have a negative pregnancy test (beta-human chorionic gonadotropin) at baseline. Exclusion criteria: Use of invasive ventilatory support (Invasive ventilation is defined as any form of ventilatory support applied with the use of an endotracheal tube.) Use of non-invasive ventilatory support while awake and in an upright position. (Non-invasive ventilation is defined as any form of ventilatory support applied without the use of an endotracheal tube.) Previously treated with Enzyme Replacement Treatment. A Female patient of childbearing potential with a positive pregnancy test. Wheelchair dependent. The patient has a major congenital anomaly. The patient has a medical condition, serious intercurrent illness, or other extenuating circumstance, in the opinion of the Investigator, may significantly interfere with study compliance, including all prescribed evaluations and follow-up activities. The patients with ≥5-year old are unable to ambulate 40 meters without assistance or unable to successfully perform repeated FVC of >30% and <85% predicted (upright). The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.

Ages Eligible for Study
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Minimum Age: 3 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Alglucosidase Alfa<br>A dose of 20 mg/kg body weight once every 2 weeks for a minimum of 52 weeks | Drug: ALGLUCOSIDASE ALFA<br>* Pharmaceutical form:solution for infusion Route of administration: intravenous<br>* Other names: MYOZYME;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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. | 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. | 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. | From signing the ICF to the 30th day after the last dosage of the study medications | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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. | 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. | 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. | 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. | 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. | Week 52 |