A Phase III Open-Label Extension Study to Assess the Long-Term Safety and Efficacy of Idebenone in Patients With Duchenne Muscular Dystrophy (DMD) Who Completed the SIDEROS Study
试验速览
- 阶段
- 3 期
- 状态
- 终止
- 入组人数
- 161
- 试验地点
- 39
- 主要终点
- Number of patients with premature discontinuations of study treatment due to adverse events.
研究概览
简要总结
The purpose of the study is to assess the long-term safety and efficacy of idebenone in patients with Duchenne muscular dystrophy (DMD) who completed the SIDEROS study.
详细描述
The study is an open-label, single-group, multi-center extension study in patients with DMD receiving glucocorticoid steroids who participated in the SIDEROS study and who meet all the inclusion criteria and none of the exclusion criteria for this extension study.
The study consists of 4 study visits scheduled every 6 months (Visit 1/Baseline, Visit 2/Week 26, Visit 3/ Week 52 and Visit 4/ Week 78), and a follow-up visit 4 weeks after treatment discontinuation. Visit 8/Week 78 in SIDEROS study is also SIDEROS-E Visit 1/Baseline.
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
- 11 Years 至 —（Child, Adult, Older Adult）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Completion of the SIDEROS study at Visit 8/ Week 78
- •Signed and dated Informed Consent Form for SIDEROS-E
排除标准
- •Patients who discontinued SIDEROS study prematurely (i.e. did not attend all visits from V1 to V8)
- •Safety, tolerability or other issues arising during the course of the SIDEROS study which in the opinion of the Investigator may put the patient at significant risk or may interfere significantly with the patient's participation in the SIDEROS-E study
- •Use of any investigational drug other than the study medication
研究组 & 干预措施
idebenone 150 mg film-coated tablets
900 mg idebenone/day (2 tablets to be taken 3 times a day with meal)
干预措施: idebenone 150 mg film-coated tablets (Drug)
结局指标
主要结局
Number of patients with premature discontinuations of study treatment due to adverse events.
时间窗: From baseline until visit 4 (week 78)
To assess the long-term safety of idebenone in DMD patients who completed the SIDEROS study.
Number of patients with abnormal vital signs.
时间窗: 4 weeks after discontinuation of treatment
To assess the long-term safety of idebenone in DMD patients who completed the SIDEROS study.
Incidence and severity of adverse events, as per ICH Topic E2A
时间窗: 4 weeks after discontinuation of treatment
To assess the long-term safety of idebenone in DMD patients who completed the SIDEROS study.
Number of patients with abnormal safety laboratory parameters.
时间窗: 4 weeks after discontinuation of treatment
To assess the long-term safety of idebenone in DMD patients who completed the SIDEROS study.
Number of patients with abnormal ECG.
时间窗: From baseline until visit 4 (week 78)
To assess the long-term safety of idebenone in DMD patients who completed the SIDEROS study.
次要结局
- Change from Baseline in Peak Expiratory Flow (PEF) as percent of predicted (PEF%p)(From baseline until visit 4 (week 78))
- Change from Baseline in Forced Expiratory Volume in 1 second (FEV1) as percent of predicted (FEV1%p)(From baseline until visit 4 (week 78))
- Change from Baseline in Forced Vital Capacity (FVC) as percent of predicted (FVC%p).(From baseline until visit 4 (week 78))