Long-term, Open-label Extension Study for Patients With Duchenne Muscular Dystrophy Enrolled in Clinical Trials Evaluating Casimersen or Golodirsen
试验速览
- 阶段
- 3 期
- 状态
- 终止
- 入组人数
- 171
- 试验地点
- 50
- 主要终点
- Number of Participants With Treatment-emergent Serious Adverse Events (TESAEs)
研究概览
简要总结
The main objective of this study is to evaluate the safety and tolerability of long-term treatment with casimersen or golodirsen 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
- 年龄范围
- 7 Years 至 23 Years（Child, Adult）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Completed a clinical trial evaluating casimersen or golodirsen, per protocol.
- •Is between 7 and 23 years of age, inclusive, at enrollment.
- •Other inclusion/exclusion criteria apply.
排除标准
- 未提供
研究组 & 干预措施
Casimersen
Patients amenable to exon 45 skipping who have completed a clinical trial evaluating casimersen will receive open-label casimersen intravenous (IV) infusions, weekly, at 30 mg/kg for up to 144 Weeks.
干预措施: Casimersen (Drug)
Golodirsen
Patients amenable to exon 53 skipping who have completed a clinical trial evaluating golodirsen will receive open-label golodirsen intravenous (IV) infusions, weekly, at 30 mg/kg for up to 144 Weeks.
干预措施: Golodirsen (Drug)
结局指标
主要结局
Number of Participants With Treatment-emergent Serious Adverse Events (TESAEs)
时间窗: Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
A treatment-emergent adverse event (TEAE) was any untoward medical occurrence in a clinical study participant that did not necessarily have a causal relationship with the study drug. A TEAE could, therefore, be any unfavorable and unintended symptom, sign, disease, condition, or test abnormality that occurred during or after administration of the study drug, whether or not considered related to the study drug. A TESAE was any TEAE that resulted in any of the following outcomes: death, a life-threatening event, required or prolonged inpatient hospitalization, persistent or significant disability/incapacity, or an important medical event (that is, may have jeopardized the participant and may have required medical or surgical intervention to prevent one of the previously mentioned outcomes). A summary of serious and all other non-serious TEAEs regardless of causality is located in the Reported Adverse Events module.
次要结局
未报告次要终点