NCT02629393已完成2 期
A Phase 2/3, Multicenter, Multinational, Open Label Study to Evaluate the Efficacy and Safety of ORGN001 (Formerly ALXN1101) in Neonates, Infants and Children With Molybdenum Cofactor Deficiency (MOCD) Type A
Origin Biosciences11 个研究点 分布在 6 个国家目标入组 5 人开始时间: 2016年5月最近更新:
适应症
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- 入组人数
- 5
- 试验地点
- 11
- 主要终点
- Overall Survival
研究概览
简要总结
To evaluate the safety and efficacy of ORGN001(formerly ALXN1101) in neonate patients with MoCD Type A
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 Day 至 5 Years（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
ORGN001 (formerly ALXN1101)
Experimental
干预措施: ORGN001 (formerly ALXN1101) (Drug)
结局指标
主要结局
Overall Survival
时间窗: Through last observation (average of 24 months)
Patients with a confirmed diagnosis of MOCD Type A, treated with ORGN001 and still alive at last observation.
次要结局
- Feeding Pattern(At Month 12 visit)
研究者
研究点 (11)
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