A Phase II, Open-Label, Extension Study to Assess the Safety and Efficacy of NS-065/NCNP-01 in Boys With Duchenne Muscular Dystrophy (DMD)
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 16
- 试验地点
- 6
- 主要终点
- Change From Baseline in Time to Stand (TTSTAND) Versus Matched Historical Controls
研究概览
简要总结
This is an open-label, extension study of NS-065/NCNP-01 administered intravenously once weekly for an additional 192 weeks to boys with DMD who complete Study NS-065/NCNP-01-201.
详细描述
This is a Phase II, multicenter, open-label, extension study of NS-065/NCNP-01 administered intravenously once weekly for an additional 192 weeks to boys with DMD who complete Study NS-065/NCNP-01-201. This study will evaluate the safety, tolerability, and clinical efficacy of NS-065/NCNP-01 at dose levels of up to 80 mg/kg/week administered by weekly IV infusion over an additional treatment period of 192 weeks or until enrollment in a separate long-term follow up program of NS-065/NCNP-01, whichever is earlier.
Patients who complete the Phase II Dose-finding Study NS-065/NCNP-01-201 are eligible to enroll.
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
- 4 Years 至 10 Years（Child）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Completed Study NS-065/NCNP-01-201 through Week
- •Willing and able to comply with scheduled visits, investigational product administration plan, and study procedures.
- •Stable dose of glucocorticoid (GC), and is expected to remain on the stable dose for the duration of the study.
排除标准
- •Serious or severe adverse event in Study NS-065/NCNP-01-201 that precludes safe use of NS-065/NCNP-
- •Patient had a treatment which was made for the purpose of dystrophin or its related protein induction after completion of Study NS-065/NCNP-01-
- •Patient took any other investigational drugs after completion of Study NS-065/NCNP-01-
- •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-065/NCNP-01 40mg/kg
Patients receiving 40mg/kg in the NS-065-NCNP-201 study will continue their current dose for an additional 192 weeks or until enrollment in a separate long-term follow up program of NS-065/NCNP-01, whichever is earlier.
干预措施: NS-065/NCNP-01 (Drug)
NS-065/NCNP-01 80mg/kg
Patients receiving 80mg/kg in the NS-065-NCNP-201 study will continue their current dose for an additional 192 weeks or until enrollment in a separate long-term follow up program of NS-065/NCNP-01, whichever is earlier.
干预措施: NS-065/NCNP-01 (Drug)
结局指标
主要结局
Change From Baseline in Time to Stand (TTSTAND) Versus Matched Historical Controls
时间窗: Baseline 201, Weeks 37, 49, 73, 109, 157, 205 in Study 202
A primary efficacy endpoint was compared to Baseline of Study 201 (NCT02740972): Time to Stand (TTSTAND)
Number of Participants With Treatment Related Adverse Events as Assessed by CTCAE v4.0.
时间窗: Up to 192 weeks of treatment
For adverse events (AEs) starting in study 201 (NCT02740972) which are not resolved at the time of enrollment into this study 202, any change in outcome or relatedness were reported in study 201. For AEs starting in study 201 which increase in severity or becomes serious after enrollment in this study 202, a new AE was reported in this study. Treatment-emergent AEs (TEAEs) were summarized by dose level. Coding was done by system organ class and preferred term (using the Medical Dictionary for Regulatory Activities (MedDRA)). Level of severity was assessed using the CTCAE grading system.
Change From Baseline in Time to Stand (TTSTAND) Velocity Versus Matched Historical Controls
时间窗: Baseline 201, Weeks 37, 49, 73, 109, 157, 205 in Study 202
A primary efficacy endpoint was compared to Baseline of Study 201 (NCT02740972): Time to Stand (TTSTAND) Velocity
次要结局
- Change From Baseline in Time to Run/Walk 10 Meters Test (TTRW) Versus Matched Historical Controls(Baseline 201, Weeks 37, 49, 73, 109, 157, 205 in Study 202)
- Change From Baseline in Time to Run/Walk 10 Meters Test (TTRW) Velocity Versus Matched Historical Controls(Baseline 201, Weeks 37, 49, 73, 109, 157, 205 in Study 202)
- Change From Baseline in Time to Climb 4 Stairs (TTCLIMB) Versus Matched Historical Controls(Baseline 201, Weeks 37, 49, 73, 109, 157, 205 in Study 202)
- Change From Baseline in Time to Climb 4 Stairs (TTCLIMB) Velocity Versus Matched Historical Controls(Baseline 201, Weeks 37, 49, 73, 109, 157, 205 in Study 202)
- Change From Baseline in North Star Ambulatory Assessment (NSAA) Score Versus Matched Historical Controls(Baseline 201, Weeks 37, 49, 73, 109, 157 in Study 202)
- Change From Baseline in Six-Minute Walk Test (6MWT) Versus Matched Historical Controls(Baseline 201, Weeks 37, 49, 73, 109, 157 in Study 202)
- Change From Baseline in Quantitative Muscle Testing (QMT) for Handgrip Versus Matched Historical Controls(Baseline 201, Weeks 37, 49, 73, 109, 157, 205 in Study 202)
- Change From Baseline in Quantitative Muscle Testing (QMT) for Elbow Flexors Versus Matched Historical Controls(Baseline 201, Weeks 37, 49, 73, 109, 157, 205 in Study 202)
- Change From Baseline in Quantitative Muscle Testing (QMT) for Elbow Extensors Versus Matched Historical Controls(Baseline 201, Weeks 37, 49, 73, 109, 157, 205 in Study 202)
- Change From Baseline in Quantitative Muscle Testing (QMT) for Knee Flexors Versus Matched Historical Controls(Baseline 201, Weeks 37, 49, 73, 109, 157, 205 in Study 202)
- Change From Baseline in Quantitative Muscle Testing (QMT) for Knee Extensors Versus Matched Historical Controls(Baseline 201, Weeks 37, 49, 73, 109, 157, 205 in Study 202)