A Phase 3, Double-blind, Placebo-Controlled Trial of Idebenone in Friedreich Ataxia
A Phase 3, Double-blind, Placebo-Controlled Trial of Idebenone in Friedreich Ataxia
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DOI：
10.1001/archneurol.2010.168
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发表时间：
2010-08-01
影响因子：
--
通讯作者：
Meier, Thomas
中科院分区：
文献类型：
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作者：
Lynch, David R.;Perlman, Susan L.;Meier, Thomas
目的：评价艾地苯酮对Friedreich共济失调患者神经功能的疗效。设计：随机、双盲、安慰剂对照干预试验。地点：费城儿童医院和加州大学洛杉矶分校。参与者：70名门诊儿科患者(年龄8-18岁)，国际合作共济失调评定量表(ICars)基线评分10-54分。干预：参与者被随机分为3组：每天服用450或900 mg艾地苯尼(体重分别为45公斤；n=22)；每天服用1350或2250 mg艾地苯尼(n=24)；或安慰剂(n=24)。主要观察指标：iCars评分从基线到24周的平均变化是主要疗效变量。次要疗效变量是Friedreich共济失调评定量表(FARS)评分的平均变化、操作测量和日常生活能力。结果：与基线相比，接受艾地苯酮治疗的患者iCars平均评分提高了2.5分，而安慰剂组患者的平均iCars评分提高了1.3分。服用艾地苯酮的患者在FARS上的得分也提高了1.6分，而服用安慰剂的患者下降了0.6分。对于两个终点，艾地苯酮组和安慰剂组之间的差异在统计学上没有差异。结论：在为期6个月的研究中，艾地苯酮没有显著改变Friedreich共济失调患者的神经功能。可能需要进行更大规模、持续时间更长的研究，以评估候选药物对Friedreich共济失调患者神经功能的治疗潜力。
Objective: To assess the efficacy of idebenone on neurological function in patients with Friedreich ataxia.Design: Randomized, double-blind, placebo-controlled intervention trial.Setting: Children's Hospital of Philadelphia and the University of California at Los Angeles.Participants: Seventy ambulatory pediatric patients (age, 8-18 years) with a baseline International Cooperative Ataxia Rating Scale (ICARS) score of 10 to 54.Interventions: Participants were randomized into 1 of 3 treatment arms: 450 or 900 mg of idebenone per day (in those with a body weight 45 kg, respectively; n=22); 1350 or 2250 mg of idebenone per day (n=24); or placebo (n= 24).Main Outcome Measures: Mean change from baseline to week 24 in ICARS score was the primary efficacy variable. Mean change in Friedreich Ataxia Rating Scale (FARS) score, performance measures, and activities of daily living were the secondary efficacy variables.Results: Patients who received idebenone improved by 2.5 points on mean ICARS score compared with baseline, while patients in the placebo group improved by 1.3 points. Patients who took idebenone also improved by 1.6 points on the FARS, while patients taking placebo declined by 0.6 points. For both end points, the difference between the idebenone and placebo groups was not statistically different.Conclusions: Idebenone did not significantly alter neurological function in Friedreich ataxia during the 6-month study. Larger studies of longer duration may be needed to assess the therapeutic potential of drug candidates on neurological function in Friedreich ataxia.