% 0期刊文章%贡纳Buyse %一个托马斯我们% Ulrike Schara % 1月Verschuuren %一个阿甘Bernert玛丽亚德安杰洛% %一个让-玛丽•Cuisset %理查德·芬克尔%娜塔莉Goemans %克雷格·麦克唐纳%基督徒Rummey %托马斯·迈耶% T杜氏肌萎缩症等物质的减少损失的呼吸功能,结果第三阶段的双盲,随机,安慰剂对照试验(提洛岛)(PL2.003) % D J神经病学2015% % P PL2.003 % V 84% N % X 14补充目标和背景:心肺衰竭死亡的主要原因是杜氏肌肉营养不良症(DMD)。首页临床前和第二阶段的证据后,疗效和安全性等的评估在10 - 18岁的多中心3期试验DMD患者没有服用伴随糖皮质激素(提洛岛,ClinicalTrials.gov NCT01027884)。设计/方法:患者随机1:1接收等(Raxone®/连锁®)900毫克/天为52周或安慰剂。主要终点是最大呼气流量的变化[percnt]预测(PEF [percnt] p)从基线到52周。二次呼吸结果措施包括用力肺活量(FVC),在1秒用力呼气量(FEV1),每周家庭PEF测量,咳嗽流峰值(PCF)和最大压力。重复测量混合模型用于疗效分析。ITT人口由64例(31等物质,33安慰剂)。修改ITT的人口(手套)前瞻性定义不包括7个病人。结果:等显著降低PEF下降[percnt]米特·p(主要终点:PEF下降[percnt]的3•05 (percnt) p p等物质与9•01 [percnt] p安慰剂,52周的治疗效果5•96 [percnt] p;p = 0•04)和ITT人口(2×57 (percnt) p与8×84 (percnt) p, 52周6•27 [percnt] p的治疗效果; p=0•03). A significant Idebenone effect was also observed in PEF (L/min), weekly home-based PEF as well as analyses of FVC and FEV1. The effect of Idebenone on PEF[percnt]p, FVC[percnt]p and FEV1[percnt]p were comparable between patients with previous corticosteroid use and steroid naïve patients. Results of respiratory function analyses were supported by responder analyses and clinical observations. No significant differences were observed in maximal mouth pressures and PCF. Treatment with Idebenone was safe and well tolerated. Conclusions: The data showed that Idebenone was safe and well tolerated, significantly reduced loss of respiratory function, and represents a new treatment option for patients with DMD. Funding: Study supported by Santhera Pharmaceuticals.Disclosure: Dr. Buyse has received personal compensation for activities with Santhera Pharmaceuticals as a consultant. Dr. Voit has received personal compensation for activities with Prosensa. Dr. Schara has nothing to disclose. Dr. Verschuuren has nothing to disclose. Dr. D'Angelo has nothing to disclose. Dr. Bernert has nothing to disclose. Dr. Cuisset has nothing to disclose. Dr. Finkel has received personal compensation for activities with Isis Pharmaceuticals and Voyager Therapeutics as a consultant and/or speaker. Dr. Goemans has nothing to disclose. Dr. McDonald has received personal compensation for activities with PTC Therapeutics and Sarepta Therapeutics as an advisory board participant and/or consultant. Dr. Rummey has nothing to disclose. Dr. Meier has received personal compensation for activities with Santhera Pharmaceuticals as an employee.Friday, April 24 2015, 12:00 pm-1:30 pm %U