ArticlePloS one2025
Evaluating elexacaftor/tezacaftor/ivacaftor (ETI; Trikafta™) for treatment of patients with non-cystic fibrosis bronchiectasis (NCFBE): A clinical study protocol.
Article in PloS one, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT05743946 (Evaluating Trikafta for the Treatment of Patients With Non-cystic Fibrosis Bronchiectasis), which is not on this map. Cited by 5 papers.
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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.
Evaluating Trikafta for the Treatment of Patients With Non-cystic Fibrosis Bronchiectasis (NCFBE)
Who cites it
5 citing papers in PubMed.
- Beyond the Surface: Novel Therapy Approach for Pancreatitis in the Setting of CFTR Dysfunction.Case reports in gastrointestinal medicine · 2026Article
- Breaking boundaries or a small piece of the puzzle: can CFTR modulators transform bronchiectasis care?ERJ open research · 2025Article
- Synthesis and Combination Studies of Novel Dipeptide Nitriles with Curcumin for a Potent Synergistic Action Against Rhodesain, Cysteine Protease ofPharmaceuticals (Basel, Switzerland) · 2025Article
- ERS Congress 2024: highlights from the Respiratory Infections Assembly.ERJ open research · 2025Article
- Cystic fibrosis: a model for research and management of respiratory diseases.Therapeutic advances in respiratory diseaseArticle
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Authors and funding
18 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
backgroundNon-cystic fibrosis bronchiectasis (NCFBE) is a disease that exhibits dilatation of airways, airflow obstruction, persistent cough, excessive sputum production, and refractory respiratory infections. NCFBE exhibits clinical and pathological manifestations similar to key features of cystic fibrosis (CF) lung disease. In CF, pathogenesis results from dysfunction of the cystic fibrosis transmembrane conductance regulator (CFTR), and diagnosis is made by demonstrating elevated sweat chloride concentrations (typically ≥60 mEq/L), two CFTR mutations known to be causal, multi-organ tissue injury, or combination(s) of these findings.
objectiveBased on a considerable body of evidence, we believe many patients with NCFBE have disease likely to benefit from drugs such as elexacaftor/tezacaftor/ivacaftor (ETI) that activate CFTR-dependent ion transport. ETI is currently prescribed solely for treatment of CF and has not been adequately tested or proposed for patients with NCFBE, many of whom exhibit decreased CFTR function. Accordingly, we are conducting a clinical trial of ETI in subjects carrying a diagnosis of NCFBE.
methodsParticipants will exhibit one disease-causing CFTR mutation and/or sweat chloride measurements of 30-59 mEq/L. Cutaneous punch biopsy or blood samples will be obtained for iPS cell differentiation into airway epithelial monolayers-which will then be tested for response to ETI. Each patient will be given CFTR modulator treatment for approximately four weeks, with monitoring of clinical endpoints that include FEV1 (forced expiratory volume in one second), sweat chloride, quality of life questionnaire, and weight. The study will evaluate response of patients with NCFBE to ETI, and test usefulness of iPSC-derived airway epithelial monolayers as a novel in vitro technology for predicting clinical benefit.
trial registrationThis trial is registered at clinicaltrials.gov (Identifier: NCT05743946. Date: 02/23/2023).
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