Ratjen Felix, Koker Paul, Geller David E, Langellier-Cocteaux Berengere, Le Maulf Florence, Kattenbeck Sabine, Moroni-Zentgraf Petra, Elborn J Stuart
Division of Respiratory Medicine, Department of Pediatrics, and Program in Physiology and Experimental Medicine, SickKids Research Institute, The Hospital for Sick Children, and University of Toronto, Toronto, Ontario, Canada.
Boehringer Ingelheim Pharmaceuticals Inc, Ridgefield, CT, United States.
J Cyst Fibros. 2015 Sep;14(5):608-14. doi: 10.1016/j.jcf.2015.03.004. Epub 2015 Mar 26.
BACKGROUND: Tiotropium Respimat improved lung function in a phase 2 trial in patients with cystic fibrosis (CF). We investigated its efficacy and safety in a phase 3 trial, including a pre-specified pooled analysis of the phase 2 and 3 trials. METHODS: 12-week, randomized, double-blind, placebo-controlled trial of tiotropium Respimat 5 μg once daily in patients with CF (N=463). RESULTS: Co-primary efficacy endpoints showed no statistical difference between tiotropium and placebo: percent-predicted forced expiratory volume in 1s (FEV1) area under the curve from 0-4h (AUC0-4h) (95% CI): 1.64% (0.27,3.55; p=0.092); percent-predicted trough FEV1 (95% CI) 1.40% (0.50,3.30; p=0.15). Adverse events were similar between groups. Pooled phase 2/3 trial results showed a treatment difference in favor of tiotropium: percent-predicted FEV1 AUC0-4h (95% CI): 2.62% (1.34,3.90). CONCLUSION: Tiotropium was well tolerated in patients with CF; lung function improvements compared with placebo were not statistically significant in the phase 3 trial. CLINICAL TRIALS: These studies are registered with clinical trial identifier numbers NCT00737100 and NCT01179347 NCT00737100 NCT01179347. These studies are also registered with the EudraCT number: 2008-001156-43 and 2010-019802-17.
背景：噻托溴铵软雾吸入剂在一项囊性纤维化（CF）患者的2期试验中改善了肺功能。我们在一项3期试验中研究了其疗效和安全性，包括对2期和3期试验进行预先指定的汇总分析。 方法：对CF患者（N = 463）进行为期12周的随机、双盲、安慰剂对照试验，每日一次吸入5μg噻托溴铵软雾吸入剂。 结果：共同主要疗效终点显示噻托溴铵与安慰剂之间无统计学差异：0至4小时曲线下1秒用力呼气容积（FEV1）预测值百分比（AUC0 - 4h）（95% CI）：1.64%（0.27, 3.55；p = 0.092）；FEV1谷值预测值百分比（95% CI）1.40%（0.50, 3.30；p = 0.15）。两组不良事件相似。2/3期试验汇总结果显示治疗效果有利于噻托溴铵：FEV1 AUC0 - 4h预测值百分比（95% CI）：2.62%（1.34, 3.90）。 结论：CF患者对噻托溴铵耐受性良好；在3期试验中，与安慰剂相比，肺功能改善无统计学意义。 临床试验：这些研究已在临床试验标识符编号NCT00737100和NCT01179347注册。这些研究也已在欧洲临床试验数据库（EudraCT）编号：2008 - 001156 - 43和2010 - 019802 - 17注册。
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