PT -期刊文章盟贡纳Buyse AU -托马斯我们盟Ulrike Schara AU - Jan Verschuuren AU -玛丽亚德安杰洛AU -冈瑟Bernert盟-让-玛丽•Cuisset AU -理查德·芬克尔盟娜塔莉Goemans AU -克雷格·麦克唐纳盟-基督教Rummey AU -托马斯·迈耶TI -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) DP - 2015年4月06 TA -神经病学PG - PL2.003 VI - 84 IP - 14补充4099 - //www.ez-admanager.com/content/84/14_Supplement/PL2.003.short 4100 - //www.ez-admanager.com/content/84/14_Supplement/PL2.003.full所以Neurology2015 4月06;首页84 AB -客观,背景:心肺衰竭死亡的主要原因是杜氏肌肉营养不良症(DMD)。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。 Results: Idebenone significantly reduced the decline in PEF[percnt]p in the mITT (primary endpoint: decline in PEF[percnt]p of 3•05[percnt]p for Idebenone versus 9•01[percnt]p for placebo, week 52 treatment effect of 5•96[percnt]p; p=0•04) and ITT population (2•57[percnt]p versus 8•84 [percnt]p, week 52 treatment effect of 6•27[percnt]p; 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