NCT01526785终止4 期
A Phase 4 Open Label, Prospective Study in Patients With Pompe Disease to Evaluate The Efficacy and Safety of Alglucosidase Alfa Produced at the 4000L Scale
Genzyme, a Sanofi Company0 个研究点目标入组 113 人开始时间: 2012年3月最近更新:
适应症
干预措施
相关药物
试验速览
- 阶段
- 4 期
- 状态
- 终止
- 发起方
- 入组人数
- 113
- 主要终点
- Percentage of Participants Who Were Clinically Stable or Improved at Week 52
研究概览
简要总结
The objective of this study was to evaluate the efficacy and safety of treatment with 4000 litre (L) alglucosidase alfa (Lumizyme®) in Pompe participants.
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
- 1 Year 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •A participant might meet all of the following criteria to be eligible for this study.
- •The participant and/or their parent/legal guardian was willing and able to provide signed informed consent.
- •The participant might be at least 1 year of age at the time of informed consent.
- •The participant had a diagnosis of Pompe disease and might have received treatment with 160 L alglucosidase alfa prior to screening.
- •The participant, if female and of childbearing potential, might have a negative pregnancy test (urine beta-human chorionic gonadotropin) at baseline. Note: all female participants of childbearing potential and sexually mature males might agree to use a medically accepted method of contraception throughout the study.
排除标准
- •A participant who met any of the following criteria were to be excluded from this study.
- •The participant had within the past 3 months received or was currently receiving any investigational product other than 160 L alglucosidase alfa or was currently participating in another clinical treatment study.
- •The participant, in the opinion of the Investigator, was clinically unstable and would not be expected to survive to completion of the 52-week treatment period.
- •The participant and/or their parent/legal guardian, in the opinion of the Investigator, was unable to adhere to the requirements of the study.
研究组 & 干预措施
Alglucosidase alfa
Experimental
Alglucosidase alfa (4000 L scale) intravenous (IV) infusion administered for 52 weeks as per physician's routine practice.
干预措施: Alglucosidase alfa (Drug)
结局指标
主要结局
Percentage of Participants Who Were Clinically Stable or Improved at Week 52
时间窗: Week 52
Clinical stability was defined as absence of death due to disease progression or new dependency on invasive ventilation and; decline in cardiac status, motor function, and pulmonary function from baseline.
次要结局
- Survival Rate at Week 52(Week 52)
- Change From Baseline on Gross Motor Function Measure-88 (GMFM-88) at Week 52(Baseline, Week 52)
- Change From Baseline in Forced Vital Capacity (FVC) at Week 52- At Supine Position(Baseline, Week 52)
- Change From Baseline in Forced Vital Capacity (FVC) at Week 52- At Sitting Position(Baseline, Week 52)
- Invasive Ventilator-Free Survival Rate at Week 52(Week 52)
- Change From Baseline on Left Ventricular Mass Z-Score (LVM-Z) at Week 52(Baseline, Week 52)
研究者
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