A Phase II, Long-term, Extension Study of DS-5141b in Patients With Duchenne Muscular Dystrophy
试验速览
- 阶段
- 2 期
- 状态
- 进行中（未招募）
- 入组人数
- 8
- 试验地点
- 2
- 主要终点
- Incidence of Adverse Events (AEs)
研究概览
简要总结
This is a multicenter, open-label, long-term, extension, phase 2 study to evaluate the safety and efficacy of long-term treatment with DS-5141b in patients with DMD who have completed DS5141-A-J101.
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
- 年龄范围
- 5 Years 至 —（Child, Adult, Older Adult）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Has competed a study of DS5141-A-J101
排除标准
- •Significant safety issues in a study of DS5141-A-J101
- •Patient who does not consent to use appropriate contraception
- •Patient not appropriate to participant in the study as determined by the Investigator
研究组 & 干预措施
DS-5141b 2.0 mg/kg
Participants who will receive DS-5141b 2.0 mg/kg once weekly.
干预措施: DS-5141b (Drug)
DS-5141b 6.0 mg/kg
Participants who will receive DS-5141b 6.0 mg/kg once weekly.
干预措施: DS-5141b (Drug)
结局指标
主要结局
Incidence of Adverse Events (AEs)
时间窗: From first injection to after the last injection of DS-5141b in this study (within approximately 2 years)
Change in distance walked during 6-minute walk test (6MWT)
时间窗: Every 3 months (within approximately 2 years)
Change in time to stand (TTSTAND)
时间窗: Every 3 months (within approximately 2 years)
Change in time in Timed up and go test
时间窗: Every 3 months (within approximately 2 years)
Change in time in 10-meter Run/Walk test
时间窗: Every 3 months (within approximately 2 years)
Change in score in the North Star Ambulatory Assessment (NSAA)
时间窗: Every 3 months (within approximately 2 years)
Change in score in the Performance of Upper Limb (PUL)
时间窗: Every 3 months (within approximately 2 years)
Change in Left Ventricular Ejection Fraction percentage (LVEF %)
时间窗: Every 6 months (within approximately 2 years)
Change in in Forced Vital Capacity (FVC) (percent predicted)
时间窗: Every 6 months (within approximately 2 years)
Change in Muscle Strength Measured by Quantitative Muscle Strength Assessment
时间窗: Every 3 months (within approximately 2 years)
次要结局
- Concentration of DS-5141a in plasma(Every 3 months (within approximately 2 years))