NCT05135663进行中（未招募）2 期
A Phase II, Open-Label, Extension Study of NS-089/NCNP-02 in Patients With Duchenne Muscular Dystrophy
适应症
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 进行中（未招募）
- 入组人数
- 6
- 试验地点
- 2
- 主要终点
- Incidence of adverse events
研究概览
简要总结
This is the extension study of NS-089/NCNP-02 (Study NCNP/DMT02), which is designed to assess the safety, tolerability and efficacy of NS-089/NCNP-02 in patients with Duchenne muscular dystrophy (DMD).
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
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Patient completed Study NCNP/DMT02
排除标准
- •Patient had any serious adverse events in Study NCNP/DMT02 that, in the opinion of the Investigator and/or Sponsor, was probably or definitely related to NS-089/NCNP-02 use and precludes safe use of NS-089/NCNP-02 for the patient in this study.
- •Patient had a treatment which was made for the purpose of dystrophin or dystrophin-related protein induction after completion of Study NCNP/DMT
- •Patient took any other investigational drugs after completion of Study NCNP/DMT
- •Patient was judged by the investigator and/or the Sponsor that it was not appropriate to participate in the extension study for other reasons.
研究组 & 干预措施
NS-089/NCNP-02 40 mg/kg
Experimental
干预措施: NS-089/NCNP-02 (Drug)
NS-089/NCNP-02 80 mg/kg
Experimental
干预措施: NS-089/NCNP-02 (Drug)
结局指标
主要结局
Incidence of adverse events
时间窗: Up to Week 319
次要结局
- Expression of dystrophin protein (Western blot)(Week 99)
- Percentage of exon 44-skipped mRNA of dystrophin(Week 99)
- North Star Ambulatory Assessment (NSAA)(Up to Week 315)
- Time to stand test(Up to Week 315)
- Time to run/walk 10 meters test(Up to Week 315)
- Six-minute walk test/Two-minute walk test(Up to Week 315)
- Timed Up & Go test(Up to Week 315)
- Quantitative muscle strength assessment(Up to Week 315)
- Performance of Upper Limb test(Up to Week 315)
- Change in serum creatine kinase concentration from baseline(Up to Week 315)
研究者
研究点 (2)
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