论文部分内容阅读
囊性纤维化是一种威胁生命的遗传性疾病,明显降低肺和消化系统功能,全球约有7万名患者。Concert制药公司宣布开始一项随机、平行组、双盲、安慰剂对照的Ⅱ期临床试验,评价CTP-656治疗囊性纤维化跨膜转导调节蛋白有基因突变的囊性纤维化患者的疗效和安全性。在美国多个医疗中心入选30~40名患者,在28 d试验期内分为CTP-656(1次/日)分为3个剂量组(20、100和150 mg)、安慰剂组及开放的伊伐卡托(iva-caftor)比较组。主终点是治疗28 d时与基线比较汗氯的变化,次终点是1 s用力呼气量(FEV1)的变化及修订
Cystic fibrosis is a life-threatening hereditary disease that significantly reduces lung and digestive system function in about 70,000 patients worldwide. Concert Pharmaceuticals Inc. announces the launch of a randomized, parallel-arm, double-blind, placebo-controlled Phase II clinical trial evaluating the efficacy of CTP-656 in the treatment of cystic fibrosis patients with genetically modified cystic fibrosis transmembrane transition regulatory proteins And safety. A total of 30-40 patients were enrolled in various medical centers in the United States and divided into CTP-656 (once daily) in three doses (20, 100 and 150 mg) during the 28-day trial. The placebo group and open Iva-caftor comparison group. The primary end point was changes in sweat chloride compared with baseline at 28 days of treatment, and the second end point was changes in FEV 1 and revision