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艾地苯醌治疗弗里德赖希共济失调的一项3期、双盲、安慰剂对照试验。

A phase 3, double-blind, placebo-controlled trial of idebenone in friedreich ataxia.

作者信息

Lynch David R, Perlman Susan L, Meier Thomas

机构信息

Division of Neurology, Children's Hospital of Philadelphia, 502 Abramson Bldg, Philadelphia, PA 19104-4318, USA.

出版信息

Arch Neurol. 2010 Aug;67(8):941-7. doi: 10.1001/archneurol.2010.168.

Abstract

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 < or = or >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. Trial Registration clinicaltrials.gov Identifier: NCT00537680.

摘要

目的

评估艾地苯醌对弗里德赖希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

费城儿童医院和加利福尼亚大学洛杉矶分校。

参与者

70名能够行走的儿科患者(年龄8 - 18岁),国际合作共济失调评定量表(ICARS)基线评分为10至54分。

干预措施

参与者被随机分为3个治疗组之一:每天服用450或900毫克艾地苯醌(分别适用于体重≤或>45千克者;n = 22);每天服用1350或2250毫克艾地苯醌(n = 24);或安慰剂(n = 24)。

主要结局指标

ICARS评分从基线到第24周的平均变化是主要疗效变量。弗里德赖希共济失调评定量表(FARS)评分、功能指标和日常生活活动的平均变化是次要疗效变量。

结果

与基线相比,接受艾地苯醌治疗的患者ICARS平均评分提高了2.5分,而安慰剂组患者提高了1.3分。服用艾地苯醌的患者FARS评分也提高了1.6分,而服用安慰剂的患者下降了0.6分。对于这两个终点,艾地苯醌组和安慰剂组之间的差异无统计学意义。

结论

在为期6个月的研究中,艾地苯醌未显著改变弗里德赖希共济失调患者的神经功能。可能需要进行更大规模、更长时间的研究来评估候选药物对弗里德赖希共济失调患者神经功能的治疗潜力。试验注册 clinicaltrials.gov标识符:NCT00537680。

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