囊性纤维化患者接受 elexacaftor/tezacaftor/ivacaftor（ETI）治疗愿意参加随机调节剂和吸入性抗菌药物临床试验的意愿。
Willingness of people with cystic fibrosis receiving elexacaftor/tezacaftor/ivacaftor (ETI) to participate in randomized modulator and inhaled antimicrobial clinical trials.
机构信息
Case Western Reserve University School of Medicine, Cleveland, OH United States.
University of Colorado, Anschutz Medical Campus, Aurora CO United States.
出版信息
J Cyst Fibros. 2023 Jul;22(4):652-655. doi: 10.1016/j.jcf.2023.04.007. Epub 2023 Apr 24.
OBJECTIVE
To assess the feasibility of enrolling people with CF (pwCF) taking the CFTR modulator elexacaftor/tezacaftor/ivacaftor (ETI) in clinical trials of a new modulator.
METHODS
PwCF receiving ETI at CHEC-SC study (NCT03350828) enrollment were surveyed for interest in 2-week to 6-month placebo- (PC) and active-comparator (AC) modulator studies. Those taking inhaled antimicrobials (inhABX) were surveyed for interest in PC inhABX studies.
RESULTS
Of 1791 respondents, 75% [95% CI 73, 77] would enroll in a 2-week PC modulator study versus 51% [49, 54] for a 6-month study; 82% [81, 84] and 63% [61, 65] would enroll in 2-week and 6 month AC studies; 77% [74, 80] of 551 taking inhABX would enroll in a 2-week PC inhABX study versus 59% [55, 63] for a 6-month study. Previous clinical trial experience increased willingness.
CONCLUSIONS
Study designs will affect feasibility of future clinical trials of new modulators and inhABX in people receiving ETI.
目的
评估招募接受 CFTR 调节剂 elexacaftor/tezacaftor/ivacaftor（ETI）治疗的 CF（pwCF）患者参加新调节剂临床试验的可行性。
方法
在 CHEC-SC 研究（NCT03350828）入组时接受 ETI 治疗的 pwCF 对为期 2 周至 6 个月的安慰剂（PC）和活性对照（AC）调节剂研究的兴趣进行了调查。接受吸入性抗生素（inhABX）治疗的患者对 PC inhABX 研究的兴趣进行了调查。
结果
在 1791 名应答者中，75%[95%CI 73，77]愿意参加为期 2 周的 PC 调节剂研究，而 51%[49，54]愿意参加为期 6 个月的研究；82%[81，84]和 63%[61，65]愿意参加为期 2 周和 6 个月的 AC 研究；551 名接受 inhABX 治疗的患者中，77%[74，80]愿意参加为期 2 周的 PC inhABX 研究，而 59%[55，63]愿意参加为期 6 个月的研究。先前的临床试验经验增加了意愿。
结论
研究设计将影响未来接受 ETI 治疗的患者新调节剂和 inhABX 的临床试验的可行性。
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