NCT00758225已完成2 期
A Phase II Open-label Extension Study to Obtain Long-term Safety, Tolerability and Efficacy Data of Idebenone in the Treatment of Duchenne Muscular Dystrophy - Extension to Study SNT-II-001
适应症
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 21
- 试验地点
- 1
- 主要终点
- Measures of safety and tolerability of idebenone: - Nature and frequency of AEs - Laboratory parameters (haematology, biochemistry and urinalysis) - Physical examinations and vital signs - ECGs
研究概览
简要总结
The scientific aim of the present extension study is to monitor long-term safety and tolerability of idebenone in patients with DMD. Furthermore, the long-term effect on respiratory, cardiac and motor functions, and skeletal muscle strength/function will be assessed.
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
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Completion of study SNT-II-001
- •Body weight ≥ 25 kg
- •Glucocorticosteroids and ACE-inhibitors are allowed, if on stable dosage within 2 months prior to inclusion
- •Eligibility to participate in the present extension study as confirmed by the investigator
排除标准
- •Safety or tolerability issues arising during the course of SNT-II-001 which in the opinion of the investigator preclude further treatment with idebenone
- •Clinically significant abnormalities of haematology or biochemistry
- •Abuse of drugs or alcohol
- •Use of coenzyme Q10 or idebenone within 30 days prior to inclusion
- •Intake of any investigational drug within 30 days prior to inclusion
- •Symptomatic heart failure
- •Previous history of ventricular arrhythmias (other than isolated ventricular extrasystole); ventricular arrhythmias presented at baseline
- •Known individual hypersensitivity to idebenone or to any of the excipients
研究组 & 干预措施
only one arm
Experimental
干预措施: Idebenone (Drug)
结局指标
主要结局
Measures of safety and tolerability of idebenone: - Nature and frequency of AEs - Laboratory parameters (haematology, biochemistry and urinalysis) - Physical examinations and vital signs - ECGs
时间窗: Month 0, 3, 6, 12, 18, 24, FU
次要结局
- Measures of efficacy of idebenone: - Respiratory Function Testing - Motor Function Measure - Quantitative Muscle Testing - Hand-Held Myometry - Echocardiography and Color Doppler Myocardial Imaging - Cardiac biomarkers(Month 0, 6, 12, 18, 24)
研究者
研究点 (1)
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