NCT01230801已完成1 期
A Phase 1/2 Open-label Study of the Safety, Tolerability, Pharmacokinetics, Pharmacodynamic and Preliminary Efficacy of BMN 701 (GILT-tagged Recombinant Human GAA) in Patients With Late-onset Pompe Disease
适应症
干预措施
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 入组人数
- 22
- 试验地点
- 20
- 主要终点
- Number of Participants With Adverse Events
研究概览
简要总结
A Phase 1/2, open-label, multicenter, multiple dose escalation study of BMN 701 administered by intravenous infusion every 2 weeks over a 24-week treatment period to patients with late-onset Pompe disease.
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
- 13 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
BMN 701
Experimental
IV infusion
干预措施: BMN 701 (Biological)
结局指标
主要结局
Number of Participants With Adverse Events
时间窗: 24 weeks
Number of Participants with Adverse Events as a Measure of Safety and Tolerability
次要结局
- Change From Baseline in Six Minutes Walk Test(Baseline up to 24 weeks)
研究者
研究点 (20)
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