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

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

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.

Publication types

  • Research Support, Non-U.S. Gov't
  • Research Support, N.I.H., Extramural

MeSH terms

  • Aminophenols / adverse effects
  • Anti-Infective Agents*
  • Benzodioxoles / adverse effects
  • Cystic Fibrosis Transmembrane Conductance Regulator / genetics
  • Cystic Fibrosis* / drug therapy
  • Cystic Fibrosis* / genetics
  • Humans
  • Mutation

Substances

  • elexacaftor
  • ivacaftor
  • tezacaftor
  • Anti-Infective Agents
  • Cystic Fibrosis Transmembrane Conductance Regulator
  • Benzodioxoles
  • Aminophenols

Associated data

  • ClinicalTrials.gov/NCT03350828