Trial reportThe New England journal of medicine2021
Triple Therapy for Cystic Fibrosis
Open access · bronzeAbstract readClinical Trial, Phase IIIComparative StudyMulticenter Study
In one paragraph
Trial report in The New England journal of medicine, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 141 papers, 6 of them syntheses that pooled it.
0numbers the graph read from it
0cells of the map it votes in
141citing papers in PubMed, 6 pooled it
27.6field-weighted citation impact, top 1% of its field
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.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
141 citing papers in PubMed, 6 syntheses or guidelines pooled it, 263 citations in OpenAlex.
- A systematic review and meta-analysis of the treatment modalities available for children afflicted from cystic fibrosis.BMC pediatrics · 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
- Use of elexacaftor+tezacaftor+ivacaftor in individuals with cystic fibrosis and at least one F508del allele: a systematic review and meta-analysis.Jornal brasileiro de pneumologia : publicacao oficial da Sociedade Brasileira de Pneumologia e Tisilogia · 2024Pooled 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
- 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
- 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
- Ivacaftor for Chronic Obstructive Pulmonary Disease: Results from a Phase 2, Randomized Controlled Trial.American journal of respiratory and critical care medicine · 2025Trial
- Residual disease activity in cystic fibrosis lung disease: implications for clinical management and research.American journal of respiratory and critical care medicine · 2026Review
- CFTR regulates lipid metabolism by relieving adipose inflammation in obesity.International journal of obesity (2005) · 2026Article
- Epithelial plasma membrane transporters as drug targets.Physiological reviews · 2026Review
- Review
- Review
- Clinical Outcomes in Concurrent Cohorts of Elexacaftor/Tezacaftor/Ivacaftor (ELX/TEZ/IVA)-Treated versus ELX/TEZ/IVA-Ineligible Individuals with Cystic Fibrosis in the US during COVID-19.Pulmonary therapy · 2026Article
- Off-label treatment with elexacaftor/tezacaftor/ivacaftor in people with cystic fibrosis: real-world evidence from the German CF Registry.ERJ open research · 2026Article
- Article
- Impact of COVID-19 and CFTR Modulators on Cystic Fibrosis: A Real-World Analysis of Care Patterns.Pediatric pulmonology · 2026Observational
- Altered functional interactions between CFTR disease mutants ΔF508 and G551D and the protein kinase A catalytic subunit.The Journal of physiology · 2026Article
- The evolving understanding of cystic fibrosis-related diabetes in the highly effective modulator therapy era: a scoping review.Diabetologia · 2026Article
- Antenatal CFTR Modulators to Treat a Healthy Pregnant Woman with a Fetus Affected by Cystic Fibrosis Complicated by Meconium Ileus and Intestinal Volvulus: From a Suspicion of the Disease to a Targeted Treatment in Utero: Case Report and Narrative Review.Journal of clinical medicine · 2026Article
81 more citing papers are in PubMed but not listed here.
Corrections and comments
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Authors and funding
23 authors at 10 institutions in 6 countries.
Peter J BarryFrom Manchester University NHS Foundation Trust, Manchester, United Kingdom (P.J.B.); Charité-Universitätsmedizin Berlin, the Berlin Institute of Health, and the German Center for Lung Research, Berlin (M.A.M.), and the Division of Cystic Fibrosis, Department of Pulmonary Medicine, University Medicine Essen-Ruhrlandklinik, University of Duisburg-Essen, Essen (S.S.) - all in Germany; Vall d'Hebron Barcelona Hospital Campus, Barcelona (A.A.); Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico and the University of Milan - both in Milan (C.C.); Wilhelmina Children's Hospital-University Medical Center Utrecht, Utrecht University, Utrecht, the Netherlands (K.M.W.-G.); Assistance Publique-Hôpitaux de Paris (AP-HP) Centre-Université de Paris Hôpital Cochin AP-HP, Paris (I.F.); Rainbow Babies and Children's Hospital, Cleveland (K.A.M.); St. Vincent's University Hospital, Dublin (E.F.M.); Seattle Children's Hospital, Seattle (B.W.R.); National Jewish Health, Denver (J.L.T.-C.); St. Michael's Hospital, Toronto (E.T.); Vertex Pharmaceuticals, Boston (N.A., L.S.J., S.M.M., V.P.-C., S.T., D.W., F.X., Y.Z.); the University of Alabama at Birmingham, Birmingham (S.M.R.); and the University of Kansas Medical Center, Kansas City (D.P.).
Marcus A MallFrom Manchester University NHS Foundation Trust, Manchester, United Kingdom (P.J.B.); Charité-Universitätsmedizin Berlin, the Berlin Institute of Health, and the German Center for Lung Research, Berlin (M.A.M.), and the Division of Cystic Fibrosis, Department of Pulmonary Medicine, University Medicine Essen-Ruhrlandklinik, University of Duisburg-Essen, Essen (S.S.) - all in Germany; Vall d'Hebron Barcelona Hospital Campus, Barcelona (A.A.); Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico and the University of Milan - both in Milan (C.C.); Wilhelmina Children's Hospital-University Medical Center Utrecht, Utrecht University, Utrecht, the Netherlands (K.M.W.-G.); Assistance Publique-Hôpitaux de Paris (AP-HP) Centre-Université de Paris Hôpital Cochin AP-HP, Paris (I.F.); Rainbow Babies and Children's Hospital, Cleveland (K.A.M.); St. Vincent's University Hospital, Dublin (E.F.M.); Seattle Children's Hospital, Seattle (B.W.R.); National Jewish Health, Denver (J.L.T.-C.); St. Michael's Hospital, Toronto (E.T.); Vertex Pharmaceuticals, Boston (N.A., L.S.J., S.M.M., V.P.-C., S.T., D.W., F.X., Y.Z.); the University of Alabama at Birmingham, Birmingham (S.M.R.); and the University of Kansas Medical Center, Kansas City (D.P.).
Antonio ÁlvarezFrom Manchester University NHS Foundation Trust, Manchester, United Kingdom (P.J.B.); Charité-Universitätsmedizin Berlin, the Berlin Institute of Health, and the German Center for Lung Research, Berlin (M.A.M.), and the Division of Cystic Fibrosis, Department of Pulmonary Medicine, University Medicine Essen-Ruhrlandklinik, University of Duisburg-Essen, Essen (S.S.) - all in Germany; Vall d'Hebron Barcelona Hospital Campus, Barcelona (A.A.); Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico and the University of Milan - both in Milan (C.C.); Wilhelmina Children's Hospital-University Medical Center Utrecht, Utrecht University, Utrecht, the Netherlands (K.M.W.-G.); Assistance Publique-Hôpitaux de Paris (AP-HP) Centre-Université de Paris Hôpital Cochin AP-HP, Paris (I.F.); Rainbow Babies and Children's Hospital, Cleveland (K.A.M.); St. Vincent's University Hospital, Dublin (E.F.M.); Seattle Children's Hospital, Seattle (B.W.R.); National Jewish Health, Denver (J.L.T.-C.); St. Michael's Hospital, Toronto (E.T.); Vertex Pharmaceuticals, Boston (N.A., L.S.J., S.M.M., V.P.-C., S.T., D.W., F.X., Y.Z.); the University of Alabama at Birmingham, Birmingham (S.M.R.); and the University of Kansas Medical Center, Kansas City (D.P.).
Carla ColomboFrom Manchester University NHS Foundation Trust, Manchester, United Kingdom (P.J.B.); Charité-Universitätsmedizin Berlin, the Berlin Institute of Health, and the German Center for Lung Research, Berlin (M.A.M.), and the Division of Cystic Fibrosis, Department of Pulmonary Medicine, University Medicine Essen-Ruhrlandklinik, University of Duisburg-Essen, Essen (S.S.) - all in Germany; Vall d'Hebron Barcelona Hospital Campus, Barcelona (A.A.); Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico and the University of Milan - both in Milan (C.C.); Wilhelmina Children's Hospital-University Medical Center Utrecht, Utrecht University, Utrecht, the Netherlands (K.M.W.-G.); Assistance Publique-Hôpitaux de Paris (AP-HP) Centre-Université de Paris Hôpital Cochin AP-HP, Paris (I.F.); Rainbow Babies and Children's Hospital, Cleveland (K.A.M.); St. Vincent's University Hospital, Dublin (E.F.M.); Seattle Children's Hospital, Seattle (B.W.R.); National Jewish Health, Denver (J.L.T.-C.); St. Michael's Hospital, Toronto (E.T.); Vertex Pharmaceuticals, Boston (N.A., L.S.J., S.M.M., V.P.-C., S.T., D.W., F.X., Y.Z.); the University of Alabama at Birmingham, Birmingham (S.M.R.); and the University of Kansas Medical Center, Kansas City (D.P.).
Karin M de Winter-de GrootFrom Manchester University NHS Foundation Trust, Manchester, United Kingdom (P.J.B.); Charité-Universitätsmedizin Berlin, the Berlin Institute of Health, and the German Center for Lung Research, Berlin (M.A.M.), and the Division of Cystic Fibrosis, Department of Pulmonary Medicine, University Medicine Essen-Ruhrlandklinik, University of Duisburg-Essen, Essen (S.S.) - all in Germany; Vall d'Hebron Barcelona Hospital Campus, Barcelona (A.A.); Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico and the University of Milan - both in Milan (C.C.); Wilhelmina Children's Hospital-University Medical Center Utrecht, Utrecht University, Utrecht, the Netherlands (K.M.W.-G.); Assistance Publique-Hôpitaux de Paris (AP-HP) Centre-Université de Paris Hôpital Cochin AP-HP, Paris (I.F.); Rainbow Babies and Children's Hospital, Cleveland (K.A.M.); St. Vincent's University Hospital, Dublin (E.F.M.); Seattle Children's Hospital, Seattle (B.W.R.); National Jewish Health, Denver (J.L.T.-C.); St. Michael's Hospital, Toronto (E.T.); Vertex Pharmaceuticals, Boston (N.A., L.S.J., S.M.M., V.P.-C., S.T., D.W., F.X., Y.Z.); the University of Alabama at Birmingham, Birmingham (S.M.R.); and the University of Kansas Medical Center, Kansas City (D.P.).
Isabelle FajacFrom Manchester University NHS Foundation Trust, Manchester, United Kingdom (P.J.B.); Charité-Universitätsmedizin Berlin, the Berlin Institute of Health, and the German Center for Lung Research, Berlin (M.A.M.), and the Division of Cystic Fibrosis, Department of Pulmonary Medicine, University Medicine Essen-Ruhrlandklinik, University of Duisburg-Essen, Essen (S.S.) - all in Germany; Vall d'Hebron Barcelona Hospital Campus, Barcelona (A.A.); Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico and the University of Milan - both in Milan (C.C.); Wilhelmina Children's Hospital-University Medical Center Utrecht, Utrecht University, Utrecht, the Netherlands (K.M.W.-G.); Assistance Publique-Hôpitaux de Paris (AP-HP) Centre-Université de Paris Hôpital Cochin AP-HP, Paris (I.F.); Rainbow Babies and Children's Hospital, Cleveland (K.A.M.); St. Vincent's University Hospital, Dublin (E.F.M.); Seattle Children's Hospital, Seattle (B.W.R.); National Jewish Health, Denver (J.L.T.-C.); St. Michael's Hospital, Toronto (E.T.); Vertex Pharmaceuticals, Boston (N.A., L.S.J., S.M.M., V.P.-C., S.T., D.W., F.X., Y.Z.); the University of Alabama at Birmingham, Birmingham (S.M.R.); and the University of Kansas Medical Center, Kansas City (D.P.).
Kimberly A McBennettFrom Manchester University NHS Foundation Trust, Manchester, United Kingdom (P.J.B.); Charité-Universitätsmedizin Berlin, the Berlin Institute of Health, and the German Center for Lung Research, Berlin (M.A.M.), and the Division of Cystic Fibrosis, Department of Pulmonary Medicine, University Medicine Essen-Ruhrlandklinik, University of Duisburg-Essen, Essen (S.S.) - all in Germany; Vall d'Hebron Barcelona Hospital Campus, Barcelona (A.A.); Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico and the University of Milan - both in Milan (C.C.); Wilhelmina Children's Hospital-University Medical Center Utrecht, Utrecht University, Utrecht, the Netherlands (K.M.W.-G.); Assistance Publique-Hôpitaux de Paris (AP-HP) Centre-Université de Paris Hôpital Cochin AP-HP, Paris (I.F.); Rainbow Babies and Children's Hospital, Cleveland (K.A.M.); St. Vincent's University Hospital, Dublin (E.F.M.); Seattle Children's Hospital, Seattle (B.W.R.); National Jewish Health, Denver (J.L.T.-C.); St. Michael's Hospital, Toronto (E.T.); Vertex Pharmaceuticals, Boston (N.A., L.S.J., S.M.M., V.P.-C., S.T., D.W., F.X., Y.Z.); the University of Alabama at Birmingham, Birmingham (S.M.R.); and the University of Kansas Medical Center, Kansas City (D.P.).
Edward F McKoneFrom Manchester University NHS Foundation Trust, Manchester, United Kingdom (P.J.B.); Charité-Universitätsmedizin Berlin, the Berlin Institute of Health, and the German Center for Lung Research, Berlin (M.A.M.), and the Division of Cystic Fibrosis, Department of Pulmonary Medicine, University Medicine Essen-Ruhrlandklinik, University of Duisburg-Essen, Essen (S.S.) - all in Germany; Vall d'Hebron Barcelona Hospital Campus, Barcelona (A.A.); Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico and the University of Milan - both in Milan (C.C.); Wilhelmina Children's Hospital-University Medical Center Utrecht, Utrecht University, Utrecht, the Netherlands (K.M.W.-G.); Assistance Publique-Hôpitaux de Paris (AP-HP) Centre-Université de Paris Hôpital Cochin AP-HP, Paris (I.F.); Rainbow Babies and Children's Hospital, Cleveland (K.A.M.); St. Vincent's University Hospital, Dublin (E.F.M.); Seattle Children's Hospital, Seattle (B.W.R.); National Jewish Health, Denver (J.L.T.-C.); St. Michael's Hospital, Toronto (E.T.); Vertex Pharmaceuticals, Boston (N.A., L.S.J., S.M.M., V.P.-C., S.T., D.W., F.X., Y.Z.); the University of Alabama at Birmingham, Birmingham (S.M.R.); and the University of Kansas Medical Center, Kansas City (D.P.).
Bonnie W RamseyFrom Manchester University NHS Foundation Trust, Manchester, United Kingdom (P.J.B.); Charité-Universitätsmedizin Berlin, the Berlin Institute of Health, and the German Center for Lung Research, Berlin (M.A.M.), and the Division of Cystic Fibrosis, Department of Pulmonary Medicine, University Medicine Essen-Ruhrlandklinik, University of Duisburg-Essen, Essen (S.S.) - all in Germany; Vall d'Hebron Barcelona Hospital Campus, Barcelona (A.A.); Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico and the University of Milan - both in Milan (C.C.); Wilhelmina Children's Hospital-University Medical Center Utrecht, Utrecht University, Utrecht, the Netherlands (K.M.W.-G.); Assistance Publique-Hôpitaux de Paris (AP-HP) Centre-Université de Paris Hôpital Cochin AP-HP, Paris (I.F.); Rainbow Babies and Children's Hospital, Cleveland (K.A.M.); St. Vincent's University Hospital, Dublin (E.F.M.); Seattle Children's Hospital, Seattle (B.W.R.); National Jewish Health, Denver (J.L.T.-C.); St. Michael's Hospital, Toronto (E.T.); Vertex Pharmaceuticals, Boston (N.A., L.S.J., S.M.M., V.P.-C., S.T., D.W., F.X., Y.Z.); the University of Alabama at Birmingham, Birmingham (S.M.R.); and the University of Kansas Medical Center, Kansas City (D.P.).
Sivagurunathan SutharsanFrom Manchester University NHS Foundation Trust, Manchester, United Kingdom (P.J.B.); Charité-Universitätsmedizin Berlin, the Berlin Institute of Health, and the German Center for Lung Research, Berlin (M.A.M.), and the Division of Cystic Fibrosis, Department of Pulmonary Medicine, University Medicine Essen-Ruhrlandklinik, University of Duisburg-Essen, Essen (S.S.) - all in Germany; Vall d'Hebron Barcelona Hospital Campus, Barcelona (A.A.); Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico and the University of Milan - both in Milan (C.C.); Wilhelmina Children's Hospital-University Medical Center Utrecht, Utrecht University, Utrecht, the Netherlands (K.M.W.-G.); Assistance Publique-Hôpitaux de Paris (AP-HP) Centre-Université de Paris Hôpital Cochin AP-HP, Paris (I.F.); Rainbow Babies and Children's Hospital, Cleveland (K.A.M.); St. Vincent's University Hospital, Dublin (E.F.M.); Seattle Children's Hospital, Seattle (B.W.R.); National Jewish Health, Denver (J.L.T.-C.); St. Michael's Hospital, Toronto (E.T.); Vertex Pharmaceuticals, Boston (N.A., L.S.J., S.M.M., V.P.-C., S.T., D.W., F.X., Y.Z.); the University of Alabama at Birmingham, Birmingham (S.M.R.); and the University of Kansas Medical Center, Kansas City (D.P.).
Jennifer L Taylor-CousarFrom Manchester University NHS Foundation Trust, Manchester, United Kingdom (P.J.B.); Charité-Universitätsmedizin Berlin, the Berlin Institute of Health, and the German Center for Lung Research, Berlin (M.A.M.), and the Division of Cystic Fibrosis, Department of Pulmonary Medicine, University Medicine Essen-Ruhrlandklinik, University of Duisburg-Essen, Essen (S.S.) - all in Germany; Vall d'Hebron Barcelona Hospital Campus, Barcelona (A.A.); Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico and the University of Milan - both in Milan (C.C.); Wilhelmina Children's Hospital-University Medical Center Utrecht, Utrecht University, Utrecht, the Netherlands (K.M.W.-G.); Assistance Publique-Hôpitaux de Paris (AP-HP) Centre-Université de Paris Hôpital Cochin AP-HP, Paris (I.F.); Rainbow Babies and Children's Hospital, Cleveland (K.A.M.); St. Vincent's University Hospital, Dublin (E.F.M.); Seattle Children's Hospital, Seattle (B.W.R.); National Jewish Health, Denver (J.L.T.-C.); St. Michael's Hospital, Toronto (E.T.); Vertex Pharmaceuticals, Boston (N.A., L.S.J., S.M.M., V.P.-C., S.T., D.W., F.X., Y.Z.); the University of Alabama at Birmingham, Birmingham (S.M.R.); and the University of Kansas Medical Center, Kansas City (D.P.).
Elizabeth TullisFrom Manchester University NHS Foundation Trust, Manchester, United Kingdom (P.J.B.); Charité-Universitätsmedizin Berlin, the Berlin Institute of Health, and the German Center for Lung Research, Berlin (M.A.M.), and the Division of Cystic Fibrosis, Department of Pulmonary Medicine, University Medicine Essen-Ruhrlandklinik, University of Duisburg-Essen, Essen (S.S.) - all in Germany; Vall d'Hebron Barcelona Hospital Campus, Barcelona (A.A.); Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico and the University of Milan - both in Milan (C.C.); Wilhelmina Children's Hospital-University Medical Center Utrecht, Utrecht University, Utrecht, the Netherlands (K.M.W.-G.); Assistance Publique-Hôpitaux de Paris (AP-HP) Centre-Université de Paris Hôpital Cochin AP-HP, Paris (I.F.); Rainbow Babies and Children's Hospital, Cleveland (K.A.M.); St. Vincent's University Hospital, Dublin (E.F.M.); Seattle Children's Hospital, Seattle (B.W.R.); National Jewish Health, Denver (J.L.T.-C.); St. Michael's Hospital, Toronto (E.T.); Vertex Pharmaceuticals, Boston (N.A., L.S.J., S.M.M., V.P.-C., S.T., D.W., F.X., Y.Z.); the University of Alabama at Birmingham, Birmingham (S.M.R.); and the University of Kansas Medical Center, Kansas City (D.P.).
Neil AhluwaliaFrom Manchester University NHS Foundation Trust, Manchester, United Kingdom (P.J.B.); Charité-Universitätsmedizin Berlin, the Berlin Institute of Health, and the German Center for Lung Research, Berlin (M.A.M.), and the Division of Cystic Fibrosis, Department of Pulmonary Medicine, University Medicine Essen-Ruhrlandklinik, University of Duisburg-Essen, Essen (S.S.) - all in Germany; Vall d'Hebron Barcelona Hospital Campus, Barcelona (A.A.); Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico and the University of Milan - both in Milan (C.C.); Wilhelmina Children's Hospital-University Medical Center Utrecht, Utrecht University, Utrecht, the Netherlands (K.M.W.-G.); Assistance Publique-Hôpitaux de Paris (AP-HP) Centre-Université de Paris Hôpital Cochin AP-HP, Paris (I.F.); Rainbow Babies and Children's Hospital, Cleveland (K.A.M.); St. Vincent's University Hospital, Dublin (E.F.M.); Seattle Children's Hospital, Seattle (B.W.R.); National Jewish Health, Denver (J.L.T.-C.); St. Michael's Hospital, Toronto (E.T.); Vertex Pharmaceuticals, Boston (N.A., L.S.J., S.M.M., V.P.-C., S.T., D.W., F.X., Y.Z.); the University of Alabama at Birmingham, Birmingham (S.M.R.); and the University of Kansas Medical Center, Kansas City (D.P.).
Lucy S JunFrom Manchester University NHS Foundation Trust, Manchester, United Kingdom (P.J.B.); Charité-Universitätsmedizin Berlin, the Berlin Institute of Health, and the German Center for Lung Research, Berlin (M.A.M.), and the Division of Cystic Fibrosis, Department of Pulmonary Medicine, University Medicine Essen-Ruhrlandklinik, University of Duisburg-Essen, Essen (S.S.) - all in Germany; Vall d'Hebron Barcelona Hospital Campus, Barcelona (A.A.); Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico and the University of Milan - both in Milan (C.C.); Wilhelmina Children's Hospital-University Medical Center Utrecht, Utrecht University, Utrecht, the Netherlands (K.M.W.-G.); Assistance Publique-Hôpitaux de Paris (AP-HP) Centre-Université de Paris Hôpital Cochin AP-HP, Paris (I.F.); Rainbow Babies and Children's Hospital, Cleveland (K.A.M.); St. Vincent's University Hospital, Dublin (E.F.M.); Seattle Children's Hospital, Seattle (B.W.R.); National Jewish Health, Denver (J.L.T.-C.); St. Michael's Hospital, Toronto (E.T.); Vertex Pharmaceuticals, Boston (N.A., L.S.J., S.M.M., V.P.-C., S.T., D.W., F.X., Y.Z.); the University of Alabama at Birmingham, Birmingham (S.M.R.); and the University of Kansas Medical Center, Kansas City (D.P.).
Samuel M MoskowitzFrom Manchester University NHS Foundation Trust, Manchester, United Kingdom (P.J.B.); Charité-Universitätsmedizin Berlin, the Berlin Institute of Health, and the German Center for Lung Research, Berlin (M.A.M.), and the Division of Cystic Fibrosis, Department of Pulmonary Medicine, University Medicine Essen-Ruhrlandklinik, University of Duisburg-Essen, Essen (S.S.) - all in Germany; Vall d'Hebron Barcelona Hospital Campus, Barcelona (A.A.); Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico and the University of Milan - both in Milan (C.C.); Wilhelmina Children's Hospital-University Medical Center Utrecht, Utrecht University, Utrecht, the Netherlands (K.M.W.-G.); Assistance Publique-Hôpitaux de Paris (AP-HP) Centre-Université de Paris Hôpital Cochin AP-HP, Paris (I.F.); Rainbow Babies and Children's Hospital, Cleveland (K.A.M.); St. Vincent's University Hospital, Dublin (E.F.M.); Seattle Children's Hospital, Seattle (B.W.R.); National Jewish Health, Denver (J.L.T.-C.); St. Michael's Hospital, Toronto (E.T.); Vertex Pharmaceuticals, Boston (N.A., L.S.J., S.M.M., V.P.-C., S.T., D.W., F.X., Y.Z.); the University of Alabama at Birmingham, Birmingham (S.M.R.); and the University of Kansas Medical Center, Kansas City (D.P.).
Valentin Prieto-CenturionFrom Manchester University NHS Foundation Trust, Manchester, United Kingdom (P.J.B.); Charité-Universitätsmedizin Berlin, the Berlin Institute of Health, and the German Center for Lung Research, Berlin (M.A.M.), and the Division of Cystic Fibrosis, Department of Pulmonary Medicine, University Medicine Essen-Ruhrlandklinik, University of Duisburg-Essen, Essen (S.S.) - all in Germany; Vall d'Hebron Barcelona Hospital Campus, Barcelona (A.A.); Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico and the University of Milan - both in Milan (C.C.); Wilhelmina Children's Hospital-University Medical Center Utrecht, Utrecht University, Utrecht, the Netherlands (K.M.W.-G.); Assistance Publique-Hôpitaux de Paris (AP-HP) Centre-Université de Paris Hôpital Cochin AP-HP, Paris (I.F.); Rainbow Babies and Children's Hospital, Cleveland (K.A.M.); St. Vincent's University Hospital, Dublin (E.F.M.); Seattle Children's Hospital, Seattle (B.W.R.); National Jewish Health, Denver (J.L.T.-C.); St. Michael's Hospital, Toronto (E.T.); Vertex Pharmaceuticals, Boston (N.A., L.S.J., S.M.M., V.P.-C., S.T., D.W., F.X., Y.Z.); the University of Alabama at Birmingham, Birmingham (S.M.R.); and the University of Kansas Medical Center, Kansas City (D.P.).
Simon TianFrom Manchester University NHS Foundation Trust, Manchester, United Kingdom (P.J.B.); Charité-Universitätsmedizin Berlin, the Berlin Institute of Health, and the German Center for Lung Research, Berlin (M.A.M.), and the Division of Cystic Fibrosis, Department of Pulmonary Medicine, University Medicine Essen-Ruhrlandklinik, University of Duisburg-Essen, Essen (S.S.) - all in Germany; Vall d'Hebron Barcelona Hospital Campus, Barcelona (A.A.); Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico and the University of Milan - both in Milan (C.C.); Wilhelmina Children's Hospital-University Medical Center Utrecht, Utrecht University, Utrecht, the Netherlands (K.M.W.-G.); Assistance Publique-Hôpitaux de Paris (AP-HP) Centre-Université de Paris Hôpital Cochin AP-HP, Paris (I.F.); Rainbow Babies and Children's Hospital, Cleveland (K.A.M.); St. Vincent's University Hospital, Dublin (E.F.M.); Seattle Children's Hospital, Seattle (B.W.R.); National Jewish Health, Denver (J.L.T.-C.); St. Michael's Hospital, Toronto (E.T.); Vertex Pharmaceuticals, Boston (N.A., L.S.J., S.M.M., V.P.-C., S.T., D.W., F.X., Y.Z.); the University of Alabama at Birmingham, Birmingham (S.M.R.); and the University of Kansas Medical Center, Kansas City (D.P.).
David WaltzFrom Manchester University NHS Foundation Trust, Manchester, United Kingdom (P.J.B.); Charité-Universitätsmedizin Berlin, the Berlin Institute of Health, and the German Center for Lung Research, Berlin (M.A.M.), and the Division of Cystic Fibrosis, Department of Pulmonary Medicine, University Medicine Essen-Ruhrlandklinik, University of Duisburg-Essen, Essen (S.S.) - all in Germany; Vall d'Hebron Barcelona Hospital Campus, Barcelona (A.A.); Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico and the University of Milan - both in Milan (C.C.); Wilhelmina Children's Hospital-University Medical Center Utrecht, Utrecht University, Utrecht, the Netherlands (K.M.W.-G.); Assistance Publique-Hôpitaux de Paris (AP-HP) Centre-Université de Paris Hôpital Cochin AP-HP, Paris (I.F.); Rainbow Babies and Children's Hospital, Cleveland (K.A.M.); St. Vincent's University Hospital, Dublin (E.F.M.); Seattle Children's Hospital, Seattle (B.W.R.); National Jewish Health, Denver (J.L.T.-C.); St. Michael's Hospital, Toronto (E.T.); Vertex Pharmaceuticals, Boston (N.A., L.S.J., S.M.M., V.P.-C., S.T., D.W., F.X., Y.Z.); the University of Alabama at Birmingham, Birmingham (S.M.R.); and the University of Kansas Medical Center, Kansas City (D.P.).
Fengjuan XuanFrom Manchester University NHS Foundation Trust, Manchester, United Kingdom (P.J.B.); Charité-Universitätsmedizin Berlin, the Berlin Institute of Health, and the German Center for Lung Research, Berlin (M.A.M.), and the Division of Cystic Fibrosis, Department of Pulmonary Medicine, University Medicine Essen-Ruhrlandklinik, University of Duisburg-Essen, Essen (S.S.) - all in Germany; Vall d'Hebron Barcelona Hospital Campus, Barcelona (A.A.); Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico and the University of Milan - both in Milan (C.C.); Wilhelmina Children's Hospital-University Medical Center Utrecht, Utrecht University, Utrecht, the Netherlands (K.M.W.-G.); Assistance Publique-Hôpitaux de Paris (AP-HP) Centre-Université de Paris Hôpital Cochin AP-HP, Paris (I.F.); Rainbow Babies and Children's Hospital, Cleveland (K.A.M.); St. Vincent's University Hospital, Dublin (E.F.M.); Seattle Children's Hospital, Seattle (B.W.R.); National Jewish Health, Denver (J.L.T.-C.); St. Michael's Hospital, Toronto (E.T.); Vertex Pharmaceuticals, Boston (N.A., L.S.J., S.M.M., V.P.-C., S.T., D.W., F.X., Y.Z.); the University of Alabama at Birmingham, Birmingham (S.M.R.); and the University of Kansas Medical Center, Kansas City (D.P.).
Yaohua ZhangFrom Manchester University NHS Foundation Trust, Manchester, United Kingdom (P.J.B.); Charité-Universitätsmedizin Berlin, the Berlin Institute of Health, and the German Center for Lung Research, Berlin (M.A.M.), and the Division of Cystic Fibrosis, Department of Pulmonary Medicine, University Medicine Essen-Ruhrlandklinik, University of Duisburg-Essen, Essen (S.S.) - all in Germany; Vall d'Hebron Barcelona Hospital Campus, Barcelona (A.A.); Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico and the University of Milan - both in Milan (C.C.); Wilhelmina Children's Hospital-University Medical Center Utrecht, Utrecht University, Utrecht, the Netherlands (K.M.W.-G.); Assistance Publique-Hôpitaux de Paris (AP-HP) Centre-Université de Paris Hôpital Cochin AP-HP, Paris (I.F.); Rainbow Babies and Children's Hospital, Cleveland (K.A.M.); St. Vincent's University Hospital, Dublin (E.F.M.); Seattle Children's Hospital, Seattle (B.W.R.); National Jewish Health, Denver (J.L.T.-C.); St. Michael's Hospital, Toronto (E.T.); Vertex Pharmaceuticals, Boston (N.A., L.S.J., S.M.M., V.P.-C., S.T., D.W., F.X., Y.Z.); the University of Alabama at Birmingham, Birmingham (S.M.R.); and the University of Kansas Medical Center, Kansas City (D.P.).
Steven M RoweFrom Manchester University NHS Foundation Trust, Manchester, United Kingdom (P.J.B.); Charité-Universitätsmedizin Berlin, the Berlin Institute of Health, and the German Center for Lung Research, Berlin (M.A.M.), and the Division of Cystic Fibrosis, Department of Pulmonary Medicine, University Medicine Essen-Ruhrlandklinik, University of Duisburg-Essen, Essen (S.S.) - all in Germany; Vall d'Hebron Barcelona Hospital Campus, Barcelona (A.A.); Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico and the University of Milan - both in Milan (C.C.); Wilhelmina Children's Hospital-University Medical Center Utrecht, Utrecht University, Utrecht, the Netherlands (K.M.W.-G.); Assistance Publique-Hôpitaux de Paris (AP-HP) Centre-Université de Paris Hôpital Cochin AP-HP, Paris (I.F.); Rainbow Babies and Children's Hospital, Cleveland (K.A.M.); St. Vincent's University Hospital, Dublin (E.F.M.); Seattle Children's Hospital, Seattle (B.W.R.); National Jewish Health, Denver (J.L.T.-C.); St. Michael's Hospital, Toronto (E.T.); Vertex Pharmaceuticals, Boston (N.A., L.S.J., S.M.M., V.P.-C., S.T., D.W., F.X., Y.Z.); the University of Alabama at Birmingham, Birmingham (S.M.R.); and the University of Kansas Medical Center, Kansas City (D.P.).
Deepika PolineniFrom Manchester University NHS Foundation Trust, Manchester, United Kingdom (P.J.B.); Charité-Universitätsmedizin Berlin, the Berlin Institute of Health, and the German Center for Lung Research, Berlin (M.A.M.), and the Division of Cystic Fibrosis, Department of Pulmonary Medicine, University Medicine Essen-Ruhrlandklinik, University of Duisburg-Essen, Essen (S.S.) - all in Germany; Vall d'Hebron Barcelona Hospital Campus, Barcelona (A.A.); Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico and the University of Milan - both in Milan (C.C.); Wilhelmina Children's Hospital-University Medical Center Utrecht, Utrecht University, Utrecht, the Netherlands (K.M.W.-G.); Assistance Publique-Hôpitaux de Paris (AP-HP) Centre-Université de Paris Hôpital Cochin AP-HP, Paris (I.F.); Rainbow Babies and Children's Hospital, Cleveland (K.A.M.); St. Vincent's University Hospital, Dublin (E.F.M.); Seattle Children's Hospital, Seattle (B.W.R.); National Jewish Health, Denver (J.L.T.-C.); St. Michael's Hospital, Toronto (E.T.); Vertex Pharmaceuticals, Boston (N.A., L.S.J., S.M.M., V.P.-C., S.T., D.W., F.X., Y.Z.); the University of Alabama at Birmingham, Birmingham (S.M.R.); and the University of Kansas Medical Center, Kansas City (D.P.).
VX18-445-104 Study Group
Assistance Publique – Hôpitaux de Paris · FRRuhrlandklinik · DEVertex Pharmaceuticals (United States) · USFondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico · ITGerman Center for Lung Research · DESeattle Children's Hospital · USUniversity of Alabama at Birmingham · USUniversity of Colorado Denver · USUniversity of Duisburg-Essen · DEUniversity of Kansas Medical Center · US
Funding
National Dissemination of I-Corps@NCATS: Accelerating Translation through CommercializationUL1TR003096 · NCATS · UNIVERSITY OF ALABAMA AT BIRMINGHAM · PI GUTIERREZ, ORLANDO M, KIMBERLY, ROBERT P. · 2019 to 2023
$43.6MUAB CF Research and Translation Core CenterP30DK072482 · NIDDK · UNIVERSITY OF ALABAMA AT BIRMINGHAM · PI Brian R. Davis · 2007 to 2026
$23.0MUsing Integrated Omics to Identify Dysfunctional Genetic Mechanisms Influencing Schizophrenia and Sleep DisturbancesP20GM130423 · NIGMS · UNIVERSITY OF KANSAS MEDICAL CENTER · PI Kalyani Pyaram, Steven Allan Soper · 2019 to 2026
$21.5MTranslational Research Center to Expedite Novel Therapies in Cystic FibrosisP30DK089507 · NIDDK · SEATTLE CHILDREN'S HOSPITAL · PI Christopher Hooper Goss · 2010 to 2026
$21.4MTranslational Program in CFTR-Related Airway DiseasesR35HL135816 · NHLBI · UNIVERSITY OF ALABAMA AT BIRMINGHAM · PI ROWE, STEVEN MARK · 2017 to 2022
$6.3MNCATS NIH HHS UL1 TR003096NHLBI NIH HHS R35 HL135816NIDDK NIH HHS P30 DK072482NIDDK NIH HHS P30 DK089507NIGMS NIH HHS P20 GM130423
Abstract
backgroundElexacaftor-tezacaftor-ivacaftor is a small-molecule cystic fibrosis transmembrane conductance regulator (CFTR) modulator regimen shown to be efficacious in patients with at least one
methodsWe conducted a phase 3, double-blind, randomized, active-controlled trial involving patients 12 years of age or older with cystic fibrosis and
resultsAfter the run-in period, 132 patients received elexacaftor-tezacaftor-ivacaftor and 126 received active control. Elexacaftor-tezacaftor-ivacaftor resulted in a percentage of predicted FEV
conclusionsElexacaftor-tezacaftor-ivacaftor was efficacious and safe in patients with
Indexed as
AdolescentAdultAminophenolsBenzodioxolesChildChloride Channel AgonistsChloridesCystic FibrosisCystic Fibrosis Transmembrane Conductance RegulatorDouble-Blind MethodDrug CombinationsFemaleGenotypeHumansIndolesMaleAminophenolsBenzodioxolesCFTR protein, humanChloride Channel AgonistsChloridesCystic Fibrosis Transmembrane Conductance RegulatorDrug Combinationselexacaftor, ivacaftor, tezacaftor drug combinationIndolesPyrazolesPyridinesQuinolines
Identifiers
What OpenQuestion holds
Textmetadata
LicenceTDM
Read underepoch 390
Registered trials
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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.