A Single Arm, Prospective, Open-label, Multi-center Study to Evaluate Efficacy and Safety in Chinese Patients With Infantile-Onset Pompe Disease With One Year Alglucosidase Alfa Treatment
试验速览
- 阶段
- 4 期
- 状态
- 已完成
- 发起方
- 入组人数
- 10
- 试验地点
- 1
- 主要终点
- Survival
研究概览
简要总结
Primary Objective:
To evaluate effect of 52-week treatment with Alglucosidase Alfa in the extension of survival and improvement of cardiomyopathy measured by Left Ventricular Mass Index in Chinese patients with infantile-onset Pompe Disease.
Secondary Objectives:
- To observe the improvement of physical growth, motor and cognitive development of 52-week treatment with Alglucosidase Alfa in infantile-onset Pompe Disease from the baseline.
- To observe the efficacy on survival free of invasive ventilation, use of any ventilation support of 52- week treatment with Alglucosidase Alfa in Chinese patients with infantile-onset Pompe Disease.
- To evaluate the safety and tolerability of Alglucosidase Alfa in Chinese patients with infantile-onset Pompe Disease.
详细描述
Total of 56 weeks in the study period, including an up to 28-day screening period and 52-week treatment period, followed by 30-day post-treatment observation period.
After the end of 52-week treatment, patients' guardians could choose to participate in a patient assistance program (PAP) sponsored by Sanofi and launched before first patient out (FPO) or reimbursement from social insurance for continued treatment.
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
- 0 Months 至 12 Months（Child）
- 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 therapy
Alglucosidase Alfa dose is calculated per kg body weight and administered once every 2 weeks for up to 52 weeks.
干预措施: ALGLUCOSIDASE ALFA (MYOZYME) (Drug)
结局指标
主要结局
Survival
时间窗: at week 52
The proportion of patients alive at the end of study
Left Ventricular Mass Index (LVMI)
时间窗: at week 52
Change from baseline in LVMI
次要结局
- Growth in body weight and length(at week 52)
- Motor development milestones(at week 52)
- Any ventilation-free survival(at week 52)
- GESELL Development Scale(at week 52)
- Cardiac failure(at week 52)
- Invasive ventilation-free survival(at week 52)