An Open-Label Safety, Tolerability, and Efficacy Study of Eteplirsen in Patients With Duchenne Muscular Dystrophy Who Have Completed Study 4658-102
试验速览
- 阶段
- 2 期
- 状态
- 终止
- 入组人数
- 15
- 试验地点
- 4
- 主要终点
- Number of Participants Experiencing Adverse Events (AEs)
研究概览
简要总结
The purpose of this extension study is to evaluate the ongoing safety and tolerability of additional treatment with eteplirsen administered once weekly by intravenous (IV) infusion in male participants with DMD who have successfully completed the 96-week eteplirsen Study 4658-102.
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
- 年龄范围
- 2 Years 至 5 Years（Child）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Participant successfully completes 96 weeks of treatment in Study 4658-102.
排除标准
- •Participant has a prior or ongoing medical condition that, in the Investigator's opinion, could adversely affect the safety of the participant, or make it unlikely that the course of treatment or follow-up would be completed, or impair the assessment of study results.
- •Other inclusion/exclusion criteria apply.
研究组 & 干预措施
Eteplirsen
Participants will receive eteplirsen via IV infusions, once weekly, for up to 284 weeks.
干预措施: Eteplirsen (Drug)
结局指标
主要结局
Number of Participants Experiencing Adverse Events (AEs)
时间窗: Up to 162 weeks
A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' section.
Number of Participants Experiencing Death Due to Adverse Events
时间窗: Up to 162 weeks
A summary of all deaths regardless of causality is located in the 'Reported Adverse Events' section.
Number of Participants Experiencing Adverse Events of Special Interest (AESIs)
时间窗: Up to 162 weeks
AESIs were defined as any AE that was of scientific and medical concern specific to study treatment, for which ongoing and rapid communication by the Investigator to the sponsor was appropriate. AESIs included findings potentially indicative of hepatic and renal abnormalities, hypersensitivity, and thrombocytopenia. A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' section.
次要结局
未报告次要终点