Long-term Use of Viltolarsen in Boys With Duchenne Muscular Dystrophy in Clinical Practice (VILT-502)
试验速览
- 阶段
- 4 期
- 状态
- 进行中（未招募）
- 入组人数
- 9
- 试验地点
- 5
- 主要终点
- Number of participants with treatment related Adverse Events as assessed by CTCAE v4.0
研究概览
简要总结
The VILT-502 study is Non-interventional Study(United States)/Low-intervention Clinical Trial (Canada) of Viltolarsen administered intravenously once weekly for 10 years to boys with DMD who complete the NS-065/NCNP-01-202 study.
详细描述
The VILT-502 study is an open-label, single-arm study to assess the long-term safety and effectiveness of viltolarsen, an exon skipping therapy for the treatment of DMD. Patients who complete the Phase II long-term extension study and meet the additional inclusion and exclusion criteria of the present protocol will be invited to enroll. Viltolarsen will be administered through weekly IV infusions, at the study site or at home.
The VILT-502 study will be conducted as a non-interventional study in the US, and as a low-intervention clinical trial in Canada where viltolarsen is not yet commercially available, owing to differences in the stage of regulatory approval in the two countries.
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, patient's parent or legal guardian have provided written informed consent/medical record release authorization prior to any extension study-specific procedures, and the patient has provided assent appropriate for his age and developmental status.
- •Patient completed the NS-065/NCNP-01-202 study and was judged by the investigator as appropriate to participate in the VILT-502 study.
- •Patient and parent or legal guardian are willing and able to comply with scheduled visits, study treatment administration plan, and study procedures.
排除标准
- •Patient has an allergy or hypersensitivity to the study drug or to any of its constituents.
- •Patient has severe behavioral or cognitive problems that preclude participation in the study, in the opinion of the investigator.
- •Patient has previous or ongoing medical condition, medical history, physical findings or laboratory abnormalities that could affect safety, make it unlikely that treatment and observation will be correctly completed or impair the assessment of study results, in the opinion of the investigator.
- •Patient had a treatment which was made for the purpose of dystrophin or its related protein induction after completing the NS-065/NCNP-01-202 study.
- •Patient took any other investigational drugs after completing the NS-065/NCNP-01-202 study.
- •Patient plans to participate in another clinical trial.
- •Patient was judged by the investigator and/or the Sponsor as not appropriate to participate in the study for reasons other than #1 - #6 above.
研究组 & 干预措施
Non-interventional (United States) / Low-interventional (Canada) : Viltolarsen
Patients will receive viltolarsen (recommended dose 80mg/kg/week) during a treatment period up to 120 months.
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 v4.0
时间窗: baseline to up to 120 months of treatment
Change in Time to Stand (TTSTAND)
时间窗: baseline to up to 120 months of treatment
Change in Performance of Upper Limb (PUL)
时间窗: baseline to up to 120 months of treatment
The Performance of the Upper Limb (PUL) scale is a specifically designed for assessing upper limb function in ambulant and non-ambulant patients with DMD. It consists of 22 items subdivided into shoulder level (6 items), mid-level (9 items) and distal level (7 items) dimension. The item ranges from score 0 -no useful hand function -to score 6 full shoulder abduction.
Loss of Ambulation (LOA)
时间窗: baseline to up to 120 months of treatment
Loss of Ambulation (LOA) is defined by the inability to complete the Time to Run/Walk 10 meters (TTRW) in less than 30 seconds.
Change in Time to Run/Walk 10 meters (TTRW)
时间窗: baseline to up to 120 months of treatment
次要结局
未报告次要终点