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. 2023 Jul;22(4):652-655.
doi: 10.1016/j.jcf.2023.04.007. Epub 2023 Apr 24.

Willingness of people with cystic fibrosis receiving elexacaftor/tezacaftor/ivacaftor (ETI) to participate in randomized modulator and inhaled antimicrobial clinical trials

D R VanDevanter  1 , E T Zemanick  2 , M W Konstan  3 , C L Ren  4 , K Odem-Davis  5 , I Emerman  5 , J Young  5 , N Mayer-Hamblett  6 ; CHEC-SC Study Group
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Willingness of people with cystic fibrosis receiving elexacaftor/tezacaftor/ivacaftor (ETI) to participate in randomized modulator and inhaled antimicrobial clinical trials

D R VanDevanter et al. J Cyst Fibros. 2023 Jul.

Abstract

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.

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Conflict of interest statement

Declaration of Competing Interest The authors have no competing or conflicting interests to report.

Figures

Figure 1.
Figure 1.. Proportions of survey respondents willing to participate in clinical trials of different design.
Panel A, studies of a new CFTR modulator comparable to ETI of varying durations including 1:1 allocation to either an active (gray circles) or placebo (black circles) comparator. Panel B, 1:1 randomized placebo-controlled studies of varying durations of an inhaled antimicrobial. Respondent numbers are shown in parentheses. Bars are 95% confidence intervals.
See this image and copyright information in PMC

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References

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