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
试验速览
- 阶段
- 4 期
- 状态
- 已完成
- 发起方
- 入组人数
- 41
- 试验地点
- 1
- 主要终点
- Change from baseline in Six-minute walk test (6MWT) for the patients ≥5-year old
研究概览
简要总结
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.
详细描述
Study duration per participants is approximatively 56 weeks including a 52-week treatment period.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 3 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- 未提供
排除标准
- 未提供
研究组 & 干预措施
Alglucosidase Alfa
A dose of 20 mg/kg body weight once every 2 weeks for a minimum of 52 weeks
干预措施: ALGLUCOSIDASE ALFA (Drug)
结局指标
主要结局
Change from baseline in Six-minute walk test (6MWT) for the patients ≥5-year old
时间窗: 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
时间窗: 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
时间窗: 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.
次要结局
- Change from baseline in maximal inspiratory pressure (MIP) in upright position for the patients ≥5-year old(Week 52)
- Change from baseline in maximal expiratory pressure (MEP) in upright position for the patients ≥5-year old(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(Week 52)
- Change from baseline in Quick Motor Function Test scores for the patients ≥5-year old(Week 52)