A Phase 3/4, Prospective, Multinational, Open-label, Noninferiority Study of Alglucosidase Alfa Manufactured at the 160 L and 4000 L Scales in Treatment Naïve Patients With Infantile-Onset Pompe Disease
试验速览
- 阶段
- 4 期
- 状态
- 终止
- 发起方
- 入组人数
- 4
- 试验地点
- 17
- 主要终点
- Change From Baseline in Cardiac Function at Week 52
研究概览
简要总结
A study to demonstrate comparable safety, efficacy, and pharmacokinetics (PK) of alglucosidase alfa manufactured at the 160 litre (L) and 4000 L scales in participants who had been diagnosed with infantile-onset Pompe disease. Participants were treated with alglucosidase alfa 160 L scale product in the United States (US) and 4000 L scale product in the regions outside the US.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- — 至 12 Months（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •The participant's parent/legal guardian was willing and able to provide signed informed consent.
- •The participant might be less than or equal to 12 months of age.
- •The participant might have documented GAA enzyme deficiency from blood, skin, or muscle tissue.
- •The participant might be naïve to treatment with alglucosidase alfa.
排除标准
- •The participant was cross-reactive immunologic material negative.
- •The participant required invasive ventilator support at the time of enrollment.
- •The participant had decompensated clinical heart failure.
- •The participant had a major congenital abnormality, excluding cardiac hypertrophy.
- •The participant had a clinically significant organ disease (excluding the signs and symptoms of Pompe disease).
- •The participant was currently receiving any investigational product.
- •The participant was participating in another clinical study.
- •The participant and/or the patient's parent/legal guardian was unable to adhere to the requirements of the study.
结局指标
主要结局
Change From Baseline in Cardiac Function at Week 52
时间窗: Baseline, Week 52
Cardiac function was measured by the left ventricular mass Z-score (LVM-Z). Z-Scores indicate the number of standard deviations (SD) from the mean in a normal distribution. A negative change from baseline indicates a decrease and positive change from baseline indicates an increase in LVM Z-score. The normal range is -2 to 2 and greater than 2 may indicate left ventricular hypertrophy.
次要结局
- Percentage of Participants With Estimated Probability of Survival(Up to Week 52)
- Number of Participants With Invasive Ventilator-Free Survival(Up to Week 52)
- Change From Baseline in Motor Development Status at Week 52(Baseline, Week 52)