jRCT2080223236未知2 期
Phase 1/2 study of NS-065/NCNP-01 in patients with Duchenne muscular dystrophy
适应症
试验速览
- 阶段
- 2 期
- 入组人数
- 16
- 主要终点
- -
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
- 干预模型
- Multicenter, Uncontrolled, Open-Label
入排标准
- 年龄范围
- 5age old over 至 17age old under（—）
- 性别
- Male
入选标准
- •Ambulant or non-ambulant patient
- •Able to treat with skipping of exon 53
排除标准
- •Has participated in other clinical trial that might recover dystrophin protein by the readthrough or the exon-skipping therapy, and/or upregulate the dystrophin-associated proteins such as utrophin.
结局指标
主要结局
-
Efficacy
次要结局
未报告次要终点
研究者
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不适用
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