A Phase III Open-Label, Single Group Extension Study of the Safety and Tolerability of Idebenone in the Treatment of Friedreich's Ataxia Patients
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 68
- 试验地点
- 2
- 主要终点
- Change in ICARS
研究概览
简要总结
This study is meant to assess the safety and tolerability of idebenone in patients with Friedreich's Ataxia over a 12 months period.
详细描述
The study involves 6 clinic visits.
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
- 8 Years 至 18 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- 未提供
排除标准
- 未提供
研究组 & 干预措施
1
high dose Idebenone
干预措施: Idebenone (Drug)
结局指标
主要结局
Change in ICARS
时间窗: baseline and 12 months
International Cooperative Ataxia Rating Scale (ICARS): ICARS consists of a one-hundred-point semi-quantitative scale based upon 19 simple testing manoeuvres compartmentalized into postural and stance disorders, limb ataxia, dysarthria and oculomotor disorders and has been previously used in this patient population with good inter-rater reliability. A total score of 0 points represents the best possible score, and a value of 100 points is the worst possible score. Therefore, a negative change in ICARS score represents an improvement. A higher score also indicates more disability.
次要结局
- Nature of Adverse Events(12 Months)
- Frequency/Number of Mild, Moderate, and Severe Adverse Events(12 months)
- FARS (Friedreich's Ataxia Rating Scale)(baseline and 12 Months)