Trial reportLancet (London, England)2019
Efficacy and safety of the elexacaftor plus tezacaftor plus ivacaftor combination regimen in people with cystic fibrosis homozygous for the F508del mutation: a double-blind, randomised, phase 3 trial.
Trial report in Lancet (London, England), 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. An erratum has been issued. It is linked to 3 registered trials, which are not on this map. Cited by 685 papers, 4 of them syntheses that pooled it.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Functional and Phenotypic Characteristics of Blood Neutrophils in Cystic Fibrosis
TAAI Erasmus Research Initiative to Fight CF: Monitoring Inflammation in CF Lung Disease Into a New Era
Studying the Presence of CFRD Complications With Thoughtful Recruitment (SPeCTRuM)
Who cites it
685 citing papers in PubMed, 4 syntheses or guidelines pooled it.
- A systematic review of the impact of Elexacaftor/Tezacaftor/Ivacaftor on body composition in people with cystic fibrosis.European journal of clinical nutrition · 2025Pooled it
- Pharmacokinetics of Ivacaftor, Tezacaftor, Elexacaftor, and Lumacaftor in Special Cystic Fibrosis Populations: A Systematic Review.Clinical pharmacokinetics · 2025Pooled it
- Ivacaftor-tezacaftor-elexacaftor, tezacaftor-ivacaftor and lumacaftor-ivacaftor for treating cystic fibrosis: a systematic review and economic evaluation.Health technology assessment (Winchester, England) · 2025Pooled it
- Exploring the utilisation and effectiveness of implementation science strategies by cystic fibrosis registries for healthcare improvement: a systematic review.European respiratory review : an official journal of the European Respiratory Society · 2025Pooled it
- Safety and efficacy of elexacaftor/tezacaftor/ivacaftor in adolescents and adults with cystic fibrosis and F508del-gating and F508del-residual function genotypes: results from an open-label extension study.Annals of the American Thoracic Society · 2026Trial
- Impact of 2 years of treatment with elexacaftor/tezacaftor/ivacaftor on longitudinal changes in structural lung disease in people with cystic fibrosis: results from the RECOVER trial.Annals of the American Thoracic Society · 2026Trial
- Evaluation of the drug interaction between rifabutin and elexacaftor/tezacaftor/ivacaftor (ETI).Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society · 2026Trial
- Elexacaftor/tezacaftor/ivacaftor in children aged ≥6 years with cystic fibrosis heterozygous forThe European respiratory journal · 2025Trial
- Vanzacaftor-tezacaftor-deutivacaftor versus elexacaftor-tezacaftor-ivacaftor in individuals with cystic fibrosis aged 12 years and older (SKYLINE Trials VX20-121-102 and VX20-121-103): results from two randomised, active-controlled, phase 3 trials.The Lancet. Respiratory medicine · 2025Trial
- Vanzacaftor-tezacaftor-deutivacaftor for children aged 6-11 years with cystic fibrosis (RIDGELINE Trial VX21-121-105): an analysis from a single-arm, phase 3 trial.The Lancet. Respiratory medicine · 2025Trial
- Low-Dose Ketone Monoester Administration in Adults with Cystic Fibrosis: A Pilot and Feasibility Study.Nutrients · 2024Trial
- The dipeptidyl peptidase 1 inhibition landscape in bronchiectasis and its future direction.European respiratory review : an official journal of the European Respiratory Society · 2026Review
- Long-term effectiveness and safety of cystic fibrosis transmembrane conductance regulator modulators and their impact on the lives of people with cystic fibrosis.American journal of respiratory and critical care medicine · 2026Article
- Residual disease activity in cystic fibrosis lung disease: implications for clinical management and research.American journal of respiratory and critical care medicine · 2026Review
- How cystic fibrosis modulator therapy could transformMicrobiology (Reading, England) · 2026Review
- Anti-inflammatory and phosphorylation effects of CFTR modulator triple therapy in cystic fibrosis.iScience · 2026Article
- Article
- Thermophysiological and perceptual responses to exercise heat stress in people with cystic fibrosis on Elexacaftor/Tezacaftor/Ivacaftor.Experimental physiology · 2026Article
- Elastic Fibers in the Pathogenesis of Cystic Fibrosis: Mechanisms, Consequences, and Therapeutic Implications.Life (Basel, Switzerland) · 2026Review
- Elexacaftor-tezacaftor-ivacaftor for individuals with residual CFTR activity: an open-label, nonrandomized clinical trial.Annals of the American Thoracic Society · 2026Article
625 more citing papers are in PubMed but not listed here.
Corrections and comments
- Commented on by
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- Commented on by
- Erratum issued
Authors and funding
21 authors.
Funding
Abstract
backgroundCystic fibrosis transmembrane conductance regulator (CFTR) modulators correct the basic defect caused by CFTR mutations. Improvements in health outcomes have been achieved with the combination of a CFTR corrector and potentiator in people with cystic fibrosis homozygous for the F508del mutation. The addition of elexacaftor (VX-445), a next-generation CFTR corrector, to tezacaftor plus ivacaftor further improved F508del-CFTR function and clinical outcomes in a phase 2 study in people with cystic fibrosis homozygous for the F508del mutation.
methodsThis phase 3, multicentre, randomised, double-blind, active-controlled trial of elexacaftor in combination with tezacaftor plus ivacaftor was done at 44 sites in four countries. Eligible participants were those with cystic fibrosis homozygous for the F508del mutation, aged 12 years or older with stable disease, and with a percentage predicted forced expiratory volume in 1 s (ppFEV
findingsBetween Aug 3 and Dec 28, 2018, 113 participants were enrolled. Following the run-in, 107 participants were randomly assigned (55 in the elexacaftor plus tezacaftor plus ivacaftor group and 52 in the tezacaftor plus ivacaftor group) and completed the 4-week treatment period. The elexacaftor plus tezacaftor plus ivacaftor group had improvements in the primary outcome of ppFEV
interpretationElexacaftor plus tezacaftor plus ivacaftor provided clinically robust benefit compared with tezacaftor plus ivacaftor alone, with a favourable safety profile, and shows the potential to lead to transformative improvements in the lives of people with cystic fibrosis who are homozygous for the F508del mutation.
fundingVertex Pharmaceuticals.
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What OpenQuestion holds
Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.