Willingness of people with cystic fibrosis receiving elexacaf tor/tezacaf tor/ivacaf tor (ETI) to participate in randomized modulator and inhaled antimicrobial clinical trials

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. & COPY; 2023 European Cystic Fibrosis Society. Published by Elsevier B.V. All rights reserved.

Automated summary

This study assessed the feasibility of enrolling CF patients using ETI in clinical trials of new modulators and inhaled antimicrobials (inhABX). The results showed that study designs and previous clinical trial experience influenced the willingness of participants. This has implications for the feasibility of future clinical trials.