Trial reportAnnals of the American Thoracic Society2017
Lumacaftor/Ivacaftor Treatment of Patients with Cystic Fibrosis Heterozygous for F508del-CFTR.
Trial report in Annals of the American Thoracic Society, 2017. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT06837181 (Studying the Presence of CFRD Complications With Thoughtful Recruitment), which is not on this map. Cited by 56 papers, 6 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.
Studying the Presence of CFRD Complications With Thoughtful Recruitment (SPeCTRuM)
Who cites it
56 citing papers in PubMed, 6 syntheses or guidelines pooled it, 88 citations in OpenAlex.
- CFTR modulator monotherapy for people with cystic fibrosis with class II CFTR gene variants (most commonly F508del).The Cochrane database of systematic reviews · 2026Pooled 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
- Academy of Nutrition and Dietetics: 2020 Cystic Fibrosis Evidence Analysis Center Evidence-Based Nutrition Practice Guideline.Journal of the Academy of Nutrition and Dietetics · 2021Guideline
- 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 · 2020Pooled it
- From Ivacaftor to Triple Combination: A Systematic Review of Efficacy and Safety of CFTR Modulators in People with Cystic Fibrosis.International journal of molecular sciences · 2020Pooled it
- A Systematic Review of the Clinical Efficacy and Safety of CFTR Modulators in Cystic Fibrosis.Scientific reports · 2019Pooled it
- 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.Lancet (London, England) · 2019Trial
- Elexacaftor-Tezacaftor-Ivacaftor for Cystic Fibrosis with a Single Phe508del Allele.The New England journal of medicine · 2019Trial
- Analytical and biological variation in repeated sweat chloride concentrations in clinical trials for CFTR modulator therapy.Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society · 2018Trial
- Tezacaftor-Ivacaftor in Residual-Function Heterozygotes with Cystic Fibrosis.The New England journal of medicine · 2017Trial
- Comparative efficacy and safety of CFTR modulators for people with cystic fibrosis with phe508del mutation: a systematic review and bayesian network meta-analysis.EClinicalMedicine · 2025Article
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- Article
- Cystic Fibrosis Treatment Landscape: Progress, Challenges, and Future Directions.Turkish archives of pediatrics · 2025Article
- TMEM16 proteins: CaInternational journal of molecular medicine · 2024Review
- Delivering a New Future for People With Cystic Fibrosis.Pediatrics · 2023Article
- CFTR Function Restoration upon Elexacaftor/Tezacaftor/Ivacaftor Treatment in Patient-Derived Intestinal Organoids with RareInternational journal of molecular sciences · 2023Article
- Dry Powder Inhalation for Lung Delivery in Cystic Fibrosis.Pharmaceutics · 2023Review
- Crosslink between SARS-CoV-2 replication and cystic fibrosis hallmarks.Frontiers in microbiology · 2023Review
- Cystic Fibrosis Patients with F508del/Minimal Function Genotype: Laboratory and Nutritional Evaluations after One Year of Elexacaftor/Tezacaftor/Ivacaftor Treatment.Journal of clinical medicine · 2022Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
10 authors at 7 institutions in 3 countries.
Funding
Abstract
rationaleIn a prior study, lumacaftor/ivacaftor treatment (≤28 d) in patients with cystic fibrosis (CF) heterozygous for F508del-CFTR did not improve lung function.
objectivesTo evaluate an optimized lumacaftor/ivacaftor dosing regimen with a longer duration in a cohort of patients heterozygous for F508del-CFTR.
methodsPatients aged 18 years or older with a confirmed CF diagnosis and percent predicted FEV MEASUREMENTS AND MAIN
resultsOf 126 patients, 119 (94.4%) completed the study. Lumacaftor/ivacaftor was well tolerated, although chest tightness and dyspnea occurred more frequently with active treatment than with placebo (27.4% vs. 14.3% and 14.5% vs. 6.3%, respectively). Mean (SD) ppFEV
conclusionsSweat chloride and respiratory symptom scores improved with lumacaftor/ivacaftor, though no meaningful benefit was seen in ppFEV
Indexed as
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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.