A Phase 3, Multi-center, Open-label Extension Study to Assess the Safety and Efficacy of Viltolarsen in Ambulant Boys With Duchenne Muscular Dystrophy (DMD)
试验速览
- 阶段
- 3 期
- 状态
- 进行中（未招募）
- 入组人数
- 74
- 试验地点
- 31
- 主要终点
- Number of participants with treatment related Adverse Events as assessed by CTCAE v4.03
研究概览
简要总结
This is a Phase 3, multi-center, open-label extension study in ambulant boys with DMD who have completed the 48-week treatment period of either viltolarsen or placebo in Study NS-065/NCNP-01-301.
详细描述
This Phase 3 study is a multi-center, open-label extension study in ambulant boys with DMD who have completed the 48-week treatment period of either viltolarsen or placebo in Study NS-065/NCNP-01-301. Patients will receive viltolarsen administered IV at weekly doses of 80 mg/kg.
Study NS-065/NCNP-01-302 will be comprised of a 96-week treatment period.
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
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Patient has completed the NS-065/NCNP-01-301 study;
- •Patient's parent(s) or legal guardian(s) has (have) provided written informed consent and Health Insurance Portability and Accountability Act authorization, where applicable, prior to any study-related procedures; patients will be asked to give written or verbal assent according to local requirements;
- •Patient and parent(s)/guardian(s) are willing and able to comply with scheduled visits, investigational product (IP) administration plan, and study procedures.
排除标准
- •Patient had an adverse event in Study NS-065/NCNP-01-301 that, in the opinion of the investigator and/or the sponsor, precludes safe use of viltolarsen 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 NS-065/NCNP-01-301;
- •Patient took any other investigational drug(s) during or after completion of Study NS-065/NCNP-01-301;
- •Patient is judged by the investigator and/or the sponsor not to be appropriate to participate in the extension study for any reason.
研究组 & 干预措施
Viltolarsen
Patients amenable to exon 53 skipping will receive viltolarsen intravenous (IV) infusions, weekly, at 80 mg/kg for up to 96 weeks.
干预措施: Viltolarsen (Drug)
结局指标
主要结局
Number of participants with treatment related Adverse Events as assessed by CTCAE v4.03
时间窗: baseline to up to 96 weeks of treatment
次要结局
- Six-minute Walk Test (6MWT)(baseline to 96 weeks of treatment)
- Time to Run/Walk 10 Meters Test (TTRW)(baseline to 96 weeks of treatment)
- Time to Climb 4 Stairs Test (TTCLIMB)(baseline to 96 weeks of treatment)
- Time to Stand Test (TTSTAND)(baseline to 96 weeks of treatment)
- North Star Ambulatory Assessment (NSAA)(baseline to 96 weeks of treatment)
- Muscle Strength Measured by Hand-Held Dynamometer(baseline to 96 weeks of treatment)