Trial reportJournal of cystic fibrosis : official journal of the European Cystic Fibrosis Society2014
Sweat chloride as a biomarker of CFTR activity: proof of concept and ivacaftor clinical trial data.
Trial report in Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society, 2014. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT00457821 (A Phase 2a, Randomized, Double-Blind, Placebo-Controlled Study of VX-770 to Evaluate Safety, Pharmacokinetics, and Biomarkers of CFTR Activity in Cystic Fibrosis), which is not on this map. Cited by 65 papers, 2 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.
A Phase 2a, Randomized, Double-Blind, Placebo-Controlled Study of VX-770 to Evaluate Safety, Pharmacokinetics, and Biomarkers of CFTR Activity in Cystic Fibrosis (CF) Subjects With Genotype G551D
Who cites it
65 citing papers in PubMed, 2 syntheses or guidelines pooled 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
- Potentiators (specific therapies for class III and IV mutations) for cystic fibrosis.The Cochrane database of systematic reviews · 2019Pooled it
- Ivacaftor for Chronic Obstructive Pulmonary Disease: Results from a Phase 2, Randomized Controlled Trial.American journal of respiratory and critical care medicine · 2025Trial
- A Phase 3, Open-Label Study of Lumacaftor/Ivacaftor in Children 1 to Less Than 2 Years of Age with Cystic Fibrosis Homozygous forAmerican journal of respiratory and critical care medicine · 2022Trial
- Triple Therapy for Cystic FibrosisThe New England journal of medicine · 2021Trial
- Clinical Effectiveness of Lumacaftor/Ivacaftor in Patients with Cystic Fibrosis Homozygous for F508del-CFTR. A Clinical Trial.Annals of the American Thoracic Society · 2021Trial
- 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
- In vivo and in vitro ivacaftor response in cystic fibrosis patients with residual CFTR function: N-of-1 studies.Pediatric pulmonology · 2017Trial
- A novel treatment of cystic fibrosis acting on-target: cysteamine plus epigallocatechin gallate for the autophagy-dependent rescue of class II-mutated CFTR.Cell death and differentiation · 2016Trial
- Restoration of CFTR function in patients with cystic fibrosis carrying the F508del-CFTR mutation.Autophagy · 2014Trial
- Early CFTR modulation and clinical outcomes in preschool cystic fibrosis.Pediatric research · 2026Article
- Sweat components as a promising monitoring tool for systemic diseases.The journal of physiological sciences : JPS · 2026Review
- Association of depressive symptoms with sweat chloride variability and medication possession ratio during long-term Elexacaftor/Tezacaftor/Ivacaftor therapy in adolescents and young adults with cystic fibrosis.Frontiers in pediatrics · 2026Article
- Non pathological sweat test, pancreatic insufficiency and Cystic Fibrosis: an unusual case in a child with F508del-duplication of exons 1-3 CFTR genotype.BMC pediatrics · 2024Article
- Ivacaftor ameliorates mucus burden, bacterial load, and inflammation in acute but not chronic P. aeruginosa infection in hG551D rats.Respiratory research · 2024Article
- Establishment of a conditionally reprogrammed primary eccrine sweat gland culture for evaluation of tissue-specific CFTR function.Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society · 2024Article
- A Systematic Review of the Effect of Cystic Fibrosis Treatments on the Nasal Potential Difference Test in Animals and Humans.Diagnostics (Basel, Switzerland) · 2023Review
- Impact of elexacaftor/tezacaftor/ivacaftor on lung function, nutritional status, pulmonary exacerbation frequency and sweat chloride in people with cystic fibrosis: real-world evidence from the German CF Registry.The Lancet regional health. Europe · 2023Article
- Characterizing CFTR modulated sweat chloride response across the cf population: Initial results from the CHEC-SC study.Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society · 2023Article
- Precision sirolimus dosing in children: The potential for model-informed dosing and novel drug monitoring.Frontiers in pharmacology · 2023Review
5 more citing papers are in PubMed but not listed here.
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
14 authors.
Funding
Abstract
backgroundWe examined data from a Phase 2 trial {NCT00457821} of ivacaftor, a CFTR potentiator, in cystic fibrosis (CF) patients with aG551D mutation to evaluate standardized approaches to sweat chloride measurement and to explore the use of sweat chloride and nasal potential difference (NPD) to estimate CFTR activity.
methodsSweat chloride and NPD were secondary endpoints in this placebo-controlled, multicenter trial. Standardization of sweat collection, processing,and analysis was employed for the first time. Sweat chloride and chloride ion transport (NPD) were integrated into a model of CFTR activity.
resultsWithin-patient sweat chloride determinations showed sufficient precision to detect differences between dose-groups and assess ivacaftor treatment effects. Analysis of changes in sweat chloride and NPD demonstrated that patients treated with ivacaftor achieved CFTR activity equivalent to approximately 35%–40% of normal.
conclusionsSweat chloride is useful in multicenter trials as a biomarker of CFTR activity and to test the effect of CFTR potentiators.
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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.