A Phase III Double-Blind, Randomized, Placebo-Controlled Study of the Efficacy, 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
- 70
- 试验地点
- 2
- 主要终点
- Change in the International Cooperative Ataxia Rating Scale (ICARS) From Baseline Assessment to Week 24
研究概览
简要总结
This study is meant to assess the effectiveness of idebenone on neurological outcome measures in patients with Friedreich's Ataxia over a 6 months period.
详细描述
The study involves 6 clinic visits and upon completion the possibility to join a 12 months extension study where all patients will receive high dose Idebenone.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 8 Years 至 17 Years（Child）
- 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
Arm 1 - 900 mg/day Idebenone
mid dose Idebenone
干预措施: Idebenone (Drug)
Arm 2 - up to 2250 mg/day Idebenone
high dose Idebenone
干预措施: Idebenone (Drug)
Arm 3 - Placebo
Placebo
干预措施: Placebo (Drug)
结局指标
主要结局
Change in the International Cooperative Ataxia Rating Scale (ICARS) From Baseline Assessment to Week 24
时间窗: baseline and 6 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. Minimum score: 0, maximum score 100, higher score indicates greater impairment Calculation details: ICARS score at week 24 minus ICARS score baseline
次要结局
- Change in the International Cooperative Ataxia Rating Scale (ICARS) From Baseline Assessment to Week 12(baseline and 12 weeks)
- Absolute Change From Baseline to Week 24 in Left Ventricular Mass Index (LVMI)(baseline and 6 months)
- Change in Friedreich's Ataxia Rating Scale (FARS) Scores From Baseline to Week 24(baseline and 6 months)
- Activities of Daily Living (ADL) of Friedreich's Ataxia Rating Scale (FARS), Change in ADL (Total Score ) From Baseline to Week 24(baseline and 6 months)
- Percent Change From Baseline to Week 24 in the Relative Wall Thickness (RWT) of the Left Ventricle(baseline and 6 months)
- Absolute Change From Baseline to Week 24 in the Relative Wall Thickness (RWT) of the Left Ventricle(baseline and 6 months)
- Percent Change From Baseline to Week 24 in Left Ventricular Mass Index(baseline and 6 months)
- Absolute Change From Baseline to Week 24 in the Thickness of the Posterior Wall (PW) of the Left Ventricle(baseline and 6 months)
- Percent Change From Baseline to Week 24 in the Thickness of the Posterior Wall (PW) of the Left Ventricle(baseline and 6 months)
- Absolute Change From Baseline to Week 24 in the Thickness of the Interventricular Septum (IVS)(baseline and 6 months)
- Percent Change From Baseline to Week 24 in the Thickness of the Interventricular Septum (IVS)(baseline and 24 weeks)