Trial reportThe New England journal of medicine2017
Tezacaftor-Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del.
Registry-linked trialAbstract readClinical Trial, Phase IIIMulticenter StudyRandomized Controlled Trial
In one paragraph
Trial report in The New England journal of medicine, 2017. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT02347657 (A Phase 3, Randomized, Double Blind, Placebo Controlled, Parallel Group Study to Evaluate the Efficacy and Safety of VX-661 in Combination With Ivacaftor in Subjects Aged 12 Years and Older With Cystic Fibrosis, Homozygous for the F508del CFTR Mutation), which is not on this map. Cited by 392 papers, 6 of them syntheses that pooled it.
0numbers the graph read from it
0cells of the map it votes in
392citing papers in PubMed, 6 pooled it
–field-weighted citation impact
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.
A Phase 3, Randomized, Double Blind, Placebo Controlled, Parallel Group Study to Evaluate the Efficacy and Safety of VX-661 in Combination With Ivacaftor in Subjects Aged 12 Years and Older With Cystic Fibrosis, Homozygous for the F508del CFTR Mutation
Who cites it
392 citing papers in PubMed, 6 syntheses or guidelines pooled it.
- Prevalence of CFTR Pathogenic Variants in Pancreatitis: A Systematic Review and Meta-Analysis.Clinical and translational gastroenterology · 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
- Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants (most commonly F508del).The Cochrane database of systematic reviews · 2023Pooled it
- Efficacy and safety profile of elexacaftor-tezacaftor-ivacaftor triple therapy on cystic fibrosis: a systematic review and single arm meta-analysis.Frontiers in pharmacology · 2023Pooled it
- Brazilian guidelines for the pharmacological treatment of the pulmonary symptoms of cystic fibrosis. Official document of the Sociedade Brasileira de Pneumologia e Tisiologia (SBPT, Brazilian Thoracic Association).Jornal brasileiro de pneumologia : publicacao oficial da Sociedade Brasileira de Pneumologia e Tisilogia · 2023Guideline
- Efficacy and Safety of Triple Combination Cystic Fibrosis Transmembrane Conductance Regulator Modulators in Patients With Cystic Fibrosis: A Meta-Analysis of Randomized Controlled Trials.Frontiers in pharmacology · 2022Pooled it
- Correlates of FEV₁ deficit in pulmonary exacerbations of cystic fibrosis and associated clinical outcomes.Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society · 2026Trial
- Ivacaftor for Chronic Obstructive Pulmonary Disease: Results from a Phase 2, Randomized Controlled Trial.American journal of respiratory and critical care medicine · 2025Trial
- Adjunctive Systemic Corticosteroids for Pulmonary Exacerbations of Cystic Fibrosis.Annals of the American Thoracic Society · 2024Trial
- Long-term safety and efficacy of elexacaftor/tezacaftor/ivacaftor in people with cystic fibrosis and at least oneThe European respiratory journal · 2023Trial
- Health care costs in a randomized trial of antimicrobial duration among cystic fibrosis patients with pulmonary exacerbations.Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society · 2022Trial
- Efficacy and safety of inhaled ENaC inhibitor BI 1265162 in patients with cystic fibrosis: BALANCE-CF 1, a randomised, phase II study.The European respiratory journal · 2022Trial
- 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
- Thermophysiological and perceptual responses to exercise heat stress in people with cystic fibrosis on Elexacaftor/Tezacaftor/Ivacaftor.Experimental physiology · 2026Article
- Elexacaftor-tezacaftor-ivacaftor for individuals with residual CFTR activity: an open-label, nonrandomized clinical trial.Annals of the American Thoracic Society · 2026Article
- Combining Gene Therapy with Current Modulator Treatments for Cystic Fibrosis: A Promising Area of Research.Pharmaceutics · 2026Review
- Effects of elexacaftor/tezacaftor/ivacaftor on the nasal microbial metagenome in cystic fibrosis.Microbiology spectrum · 2026Article
- Targeted Therapy for Restoring CFTR Activity: From Experimental to Clinical Features.International journal of molecular sciences · 2026Review
- Clinical Trials in GBA1-Associated Parkinson's Disease: A Need for Molecular Classification.Movement disorders : official journal of the Movement Disorder Society · 2026Article
- Cystic Fibrosis Mortality Trends 1999-2024-A CDC Wonder Study.Advances in respiratory medicine · 2026Article
332 more citing papers are in PubMed but not listed here.
Corrections and comments
- Commented on by
Authors and funding
12 authors.
Jennifer L Taylor-CousarFrom National Jewish Health, Denver (J.L.T.-C.); Hôpital Robert Debré, Assistance Publique-Hopitaux de Paris, Paris (A. Munck); University College Dublin School of Medicine, St. Vincent's University Hospital, Dublin (E.F.M.); University Medical Center, Utrecht, the Netherlands (C.K.E.); University Children's Hospital Zurich, Zurich, Switzerland (A. Moeller); Vertex Pharmaceuticals, Boston (C.S., L.T.W., E.P.I., C.M., Y.L., J.L.-H.); and Imperial College and Royal Brompton Hospital and Harefield NHS Foundation Trust, London, and Queens University, Belfast - all in the United Kingdom (J.S.E.).
Anne MunckFrom National Jewish Health, Denver (J.L.T.-C.); Hôpital Robert Debré, Assistance Publique-Hopitaux de Paris, Paris (A. Munck); University College Dublin School of Medicine, St. Vincent's University Hospital, Dublin (E.F.M.); University Medical Center, Utrecht, the Netherlands (C.K.E.); University Children's Hospital Zurich, Zurich, Switzerland (A. Moeller); Vertex Pharmaceuticals, Boston (C.S., L.T.W., E.P.I., C.M., Y.L., J.L.-H.); and Imperial College and Royal Brompton Hospital and Harefield NHS Foundation Trust, London, and Queens University, Belfast - all in the United Kingdom (J.S.E.).
Edward F McKoneFrom National Jewish Health, Denver (J.L.T.-C.); Hôpital Robert Debré, Assistance Publique-Hopitaux de Paris, Paris (A. Munck); University College Dublin School of Medicine, St. Vincent's University Hospital, Dublin (E.F.M.); University Medical Center, Utrecht, the Netherlands (C.K.E.); University Children's Hospital Zurich, Zurich, Switzerland (A. Moeller); Vertex Pharmaceuticals, Boston (C.S., L.T.W., E.P.I., C.M., Y.L., J.L.-H.); and Imperial College and Royal Brompton Hospital and Harefield NHS Foundation Trust, London, and Queens University, Belfast - all in the United Kingdom (J.S.E.).
Cornelis K van der EntFrom National Jewish Health, Denver (J.L.T.-C.); Hôpital Robert Debré, Assistance Publique-Hopitaux de Paris, Paris (A. Munck); University College Dublin School of Medicine, St. Vincent's University Hospital, Dublin (E.F.M.); University Medical Center, Utrecht, the Netherlands (C.K.E.); University Children's Hospital Zurich, Zurich, Switzerland (A. Moeller); Vertex Pharmaceuticals, Boston (C.S., L.T.W., E.P.I., C.M., Y.L., J.L.-H.); and Imperial College and Royal Brompton Hospital and Harefield NHS Foundation Trust, London, and Queens University, Belfast - all in the United Kingdom (J.S.E.).
Alexander MoellerFrom National Jewish Health, Denver (J.L.T.-C.); Hôpital Robert Debré, Assistance Publique-Hopitaux de Paris, Paris (A. Munck); University College Dublin School of Medicine, St. Vincent's University Hospital, Dublin (E.F.M.); University Medical Center, Utrecht, the Netherlands (C.K.E.); University Children's Hospital Zurich, Zurich, Switzerland (A. Moeller); Vertex Pharmaceuticals, Boston (C.S., L.T.W., E.P.I., C.M., Y.L., J.L.-H.); and Imperial College and Royal Brompton Hospital and Harefield NHS Foundation Trust, London, and Queens University, Belfast - all in the United Kingdom (J.S.E.).
Christopher SimardFrom National Jewish Health, Denver (J.L.T.-C.); Hôpital Robert Debré, Assistance Publique-Hopitaux de Paris, Paris (A. Munck); University College Dublin School of Medicine, St. Vincent's University Hospital, Dublin (E.F.M.); University Medical Center, Utrecht, the Netherlands (C.K.E.); University Children's Hospital Zurich, Zurich, Switzerland (A. Moeller); Vertex Pharmaceuticals, Boston (C.S., L.T.W., E.P.I., C.M., Y.L., J.L.-H.); and Imperial College and Royal Brompton Hospital and Harefield NHS Foundation Trust, London, and Queens University, Belfast - all in the United Kingdom (J.S.E.).
Linda T WangFrom National Jewish Health, Denver (J.L.T.-C.); Hôpital Robert Debré, Assistance Publique-Hopitaux de Paris, Paris (A. Munck); University College Dublin School of Medicine, St. Vincent's University Hospital, Dublin (E.F.M.); University Medical Center, Utrecht, the Netherlands (C.K.E.); University Children's Hospital Zurich, Zurich, Switzerland (A. Moeller); Vertex Pharmaceuticals, Boston (C.S., L.T.W., E.P.I., C.M., Y.L., J.L.-H.); and Imperial College and Royal Brompton Hospital and Harefield NHS Foundation Trust, London, and Queens University, Belfast - all in the United Kingdom (J.S.E.).
Edward P IngenitoFrom National Jewish Health, Denver (J.L.T.-C.); Hôpital Robert Debré, Assistance Publique-Hopitaux de Paris, Paris (A. Munck); University College Dublin School of Medicine, St. Vincent's University Hospital, Dublin (E.F.M.); University Medical Center, Utrecht, the Netherlands (C.K.E.); University Children's Hospital Zurich, Zurich, Switzerland (A. Moeller); Vertex Pharmaceuticals, Boston (C.S., L.T.W., E.P.I., C.M., Y.L., J.L.-H.); and Imperial College and Royal Brompton Hospital and Harefield NHS Foundation Trust, London, and Queens University, Belfast - all in the United Kingdom (J.S.E.).
Charlotte McKeeFrom National Jewish Health, Denver (J.L.T.-C.); Hôpital Robert Debré, Assistance Publique-Hopitaux de Paris, Paris (A. Munck); University College Dublin School of Medicine, St. Vincent's University Hospital, Dublin (E.F.M.); University Medical Center, Utrecht, the Netherlands (C.K.E.); University Children's Hospital Zurich, Zurich, Switzerland (A. Moeller); Vertex Pharmaceuticals, Boston (C.S., L.T.W., E.P.I., C.M., Y.L., J.L.-H.); and Imperial College and Royal Brompton Hospital and Harefield NHS Foundation Trust, London, and Queens University, Belfast - all in the United Kingdom (J.S.E.).
Yimeng LuFrom National Jewish Health, Denver (J.L.T.-C.); Hôpital Robert Debré, Assistance Publique-Hopitaux de Paris, Paris (A. Munck); University College Dublin School of Medicine, St. Vincent's University Hospital, Dublin (E.F.M.); University Medical Center, Utrecht, the Netherlands (C.K.E.); University Children's Hospital Zurich, Zurich, Switzerland (A. Moeller); Vertex Pharmaceuticals, Boston (C.S., L.T.W., E.P.I., C.M., Y.L., J.L.-H.); and Imperial College and Royal Brompton Hospital and Harefield NHS Foundation Trust, London, and Queens University, Belfast - all in the United Kingdom (J.S.E.).
Julie Lekstrom-HimesFrom National Jewish Health, Denver (J.L.T.-C.); Hôpital Robert Debré, Assistance Publique-Hopitaux de Paris, Paris (A. Munck); University College Dublin School of Medicine, St. Vincent's University Hospital, Dublin (E.F.M.); University Medical Center, Utrecht, the Netherlands (C.K.E.); University Children's Hospital Zurich, Zurich, Switzerland (A. Moeller); Vertex Pharmaceuticals, Boston (C.S., L.T.W., E.P.I., C.M., Y.L., J.L.-H.); and Imperial College and Royal Brompton Hospital and Harefield NHS Foundation Trust, London, and Queens University, Belfast - all in the United Kingdom (J.S.E.).
J Stuart ElbornFrom National Jewish Health, Denver (J.L.T.-C.); Hôpital Robert Debré, Assistance Publique-Hopitaux de Paris, Paris (A. Munck); University College Dublin School of Medicine, St. Vincent's University Hospital, Dublin (E.F.M.); University Medical Center, Utrecht, the Netherlands (C.K.E.); University Children's Hospital Zurich, Zurich, Switzerland (A. Moeller); Vertex Pharmaceuticals, Boston (C.S., L.T.W., E.P.I., C.M., Y.L., J.L.-H.); and Imperial College and Royal Brompton Hospital and Harefield NHS Foundation Trust, London, and Queens University, Belfast - all in the United Kingdom (J.S.E.).
Funding
No grant is acknowledged in the PubMed record.
Abstract
backgroundCombination treatment with the cystic fibrosis transmembrane conductance regulator (CFTR) modulators tezacaftor (VX-661) and ivacaftor (VX-770) was designed to target the underlying cause of disease in patients with cystic fibrosis.
methodsIn this phase 3, randomized, double-blind, multicenter, placebo-controlled, parallel-group trial, we evaluated combination therapy with tezacaftor and ivacaftor in patients 12 years of age or older who had cystic fibrosis and were homozygous for the CFTR Phe508del mutation. Patients were randomly assigned in a 1:1 ratio to receive either 100 mg of tezacaftor once daily and 150 mg of ivacaftor twice daily or matched placebo for 24 weeks. The primary end point was the absolute change in the percentage of the predicted forced expiratory volume in 1 second (FEV
resultsOf the 510 patients who underwent randomization, 509 received tezacaftor-ivacaftor or placebo, and 475 completed 24 weeks of the trial regimen. The mean FEV
conclusionsThe combination of tezacaftor and ivacaftor was efficacious and safe in patients 12 years of age or older who had cystic fibrosis and were homozygous for the CFTR Phe508del mutation. (Funded by Vertex Pharmaceuticals; EVOLVE ClinicalTrials.gov number, NCT02347657 .).
Indexed as
AdolescentAdultAminophenolsBenzodioxolesBody Mass IndexChildCystic FibrosisCystic Fibrosis Transmembrane Conductance RegulatorDouble-Blind MethodDrug CombinationsFemaleForced Expiratory VolumeHomozygoteHumansIndolesMaleAminophenolsBenzodioxolesCFTR protein, humanCystic Fibrosis Transmembrane Conductance RegulatorDrug CombinationsIndolesivacaftorQuinolonestezacaftor
Identifiers
PMID
29099344What OpenQuestion holds
Textmetadata
Read underepoch 390
Registered trials
NCT02347657ClinicalTrials.gov
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.