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Clinical Trial
. 2021 Feb 1;203(3):381-385.
doi: 10.1164/rccm.202008-3176LE.

Safety and Efficacy of Elexacaftor/Tezacaftor/Ivacaftor for 24 Weeks or Longer in People with Cystic Fibrosis and One or More F508del Alleles: Interim Results of an Open-Label Phase 3 Clinical Trial

Matthias Griese  1   2 , Stefano Costa  3 , Rachel W Linnemann  4 , Marcus A Mall  5   6 , Edward F McKone  7 , Deepika Polineni  8 , Bradley S Quon  9   10 , Felix C Ringshausen  11   12 , Jennifer L Taylor-Cousar  13 , Nicholas J Withers  14 , Samuel M Moskowitz  15 , Cori L Daines  16
Affiliations
Clinical Trial

Safety and Efficacy of Elexacaftor/Tezacaftor/Ivacaftor for 24 Weeks or Longer in People with Cystic Fibrosis and One or More F508del Alleles: Interim Results of an Open-Label Phase 3 Clinical Trial

Matthias Griese et al. Am J Respir Crit Care Med. .
No abstract available

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Figures

Figure 1.
Figure 1.
Mixed-effects model for repeated measures analysis of absolute change from baseline in FEV1% predicted (ppFEV1), sweat chloride (SwCl), body mass index (BMI), and Cystic Fibrosis Questionnaire-Revised respiratory domain (CFQ-R RD) by visit. The graphed data represent the least squares mean (SE) absolute change from parent study baseline by mixed-effects model for repeated measures at each visit. The data labels on each plot represent the least squares mean (95% confidence interval) absolute change from parent study baseline and the number of evaluable participants for that visit. For participants heterozygous for the F508del-CFTR mutation and a minimal function CFTR mutation (F/MF) (5), the data labels correspond with the Week 24 visit of the F/MF pivotal study and the Week 24 visit of the open-label extension. For participants homozygous for the F508del-CFTR mutation (F/F) (6), the data labels correspond with the Week 4 visit of the F/F pivotal study and the Week 24 (SwCl and CFQ-R RD) or Week 36 visit (ppFEV1 and BMI) of the open-label extension. Baseline for F/F participants occurred after a 4-week run-in with tezacaftor (TEZ)/ivacaftor (IVA). SwCl and CFQ-R RD were assessed through Week 24 in all participants. The ppFEV1 and BMI were assessed through Week 24 in participants with F/MF genotypes and through Week 36 in participants with the F/F genotype. For participants with F/MF genotypes, n = 203 for those who received placebo in the 24-week F/MF pivotal study and n = 196 for those treated with elexacaftor (ELX)/TEZ/IVA. For participants with the F/F genotype, n = 52 for those treated with TEZ/IVA and n = 55 for those treated with ELX/TEZ/IVA in the 4-week F/F pivotal study. OLE = open-label extension; PBO = placebo.
See this image and copyright information in PMC

References

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    1. Middleton PG, Mall MA, Dřevínek P, Lands LC, McKone EF, Polineni D, et al. VX17-445-102 Study Group. Elexacaftor-tezacaftor-ivacaftor for cystic fibrosis with a single Phe508del allele. N Engl J Med. 2019;381:1809–1819. - PMC - PubMed

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