Trial reportJournal of cystic fibrosis : official journal of the European Cystic Fibrosis Society2019
An open-label extension study of ivacaftor in children with CF and a CFTR gating mutation initiating treatment at age 2-5 years (KLIMB).
Trial report in Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to 3 registered trials, which are not on this map. Cited by 62 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 3, 2-Part, Open-Label Study to Evaluate the Safety, Pharmacokinetics and Pharmacodynamics of Ivacaftor in Subjects With Cystic Fibrosis Who Are 2 Through 5 Years of Age and Have a CFTR Gating Mutation
A Phase 3, 2-Arm, Roll-Over Study to Evaluate the Long-term Safety and Pharmacodynamics of Ivacaftor Treatment in Pediatric Subjects With Cystic Fibrosis and a CFTR Gating Mutation
The Return of the Pancreas: Evaluating Impact of CFTR Modulators on Pancreatic Function
Who cites it
62 citing papers in PubMed, 2 syntheses or guidelines pooled it, 132 citations in OpenAlex.
- European guidelines for the diagnosis and treatment of pancreatic exocrine insufficiency: UEG, EPC, EDS, ESPEN, ESPGHAN, ESDO, and ESPCG evidence-based recommendations.United European gastroenterology journal · 2025Guideline
- 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
- Ivacaftor in Infants Aged 4 to <12 Months with Cystic Fibrosis and a Gating Mutation. Results of a Two-Part Phase 3 Clinical Trial.American journal of respiratory and critical care medicine · 2021Trial
- Cystic fibrosis care today-a narrative review based on views of experts, patients and the public.Wiener klinische Wochenschrift · 2026Review
- Impact of Elexacaftor/Tezacaftor/Ivacaftor on Fat-Soluble Vitamin Status in 2 to 5 Year-Old Children Using a Cystic Fibrosis-Specific Multivitamin Formulation.Children (Basel, Switzerland) · 2026Article
- Considerations for early life genetic therapies in cystic fibrosis.American journal of physiology. Lung cellular and molecular physiology · 2026Review
- Utility of Fecal Elastase-1 in Estimating Exocrine Pancreatic Function in Cystic Fibrosis: A Scoping Review.Pediatric pulmonology · 2026Article
- Impact of elexacaftor/tezacaftor/ivacaftor on gastrointestinal outcomes, inflammation, exocrine pancreatic function and fat malabsorption: Report of PROMISE pediatric substudy.Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society · 2026Observational
- Review
- Cystic Fibrosis of the Pancreas: In Vitro Duct Models for CFTR-Targeted Translational Research.International journal of molecular sciences · 2026Review
- Role of elexacaftor-tezacaftor-ivacaftor therapy on fecal elastase-1 and fat-soluble vitamins in children with cystic fibrosis.European journal of pediatrics · 2026Article
- Article
- Fecal microbiota changes in people with cystic fibrosis after 6 months of elexacaftor/tezacaftor/ivacaftor: Findings from the promise study.Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society · 2025Observational
- Impacts and New Challenges with Highly Effective Modulator Therapies in Younger Children with Cystic Fibrosis.Journal of clinical medicine · 2025Review
- Impact of Elexacaftor/Tezacaftor/Ivacaftor on Fecal Elastase-1 in Children With Cystic Fibrosis.Pediatric pulmonology · 2025Article
- Unraveling the Mechanism of Action, Binding Sites, and Therapeutic Advances of CFTR Modulators: A Narrative Review.Current issues in molecular biology · 2025Review
- Allergic Bronchopulmonary Aspergillosis (ABPA) in the Era of Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) Modulators.Journal of fungi (Basel, Switzerland) · 2024Review
- Pancreatic enzyme prescription following ivacaftor licensing: A retrospective analysis of the US and UK cystic fibrosis registries.Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society · 2024Article
- Current and future therapeutic approaches of CFTR and airway dysbiosis in an era of personalized medicine.Journal of family medicine and primary care · 2024Review
- Changes in vitamins and trace elements after initiation of highly effective CFTR modulator therapy in children and adults with cystic fibrosis - a real-life insight.Molecular and cellular pediatrics · 2024Article
2 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
13 authors at 11 institutions in 3 countries.
Funding
Abstract
backgroundKIWI (NCT01705145) was a 24-week, single-arm, pharmacokinetics, safety, and efficacy study of ivacaftor in children aged 2 to 5 years with cystic fibrosis (CF) and a CFTR gating mutation. Here, we report the results of KLIMB (NCT01946412), an 84-week, open-label extension of KIWI.
methodsChildren received age- and weight-based ivacaftor dosages for 84 weeks. The primary outcome was safety. Other outcomes included sweat chloride, growth parameters, and measures of pancreatic function.
resultsAll 33 children who completed KIWI enrolled in KLIMB; 28 completed 84 weeks of treatment. Most adverse events were consistent with those reported during KIWI. Ten (30%) children had transaminase elevations >3 × upper limit of normal (ULN), leading to 1 discontinuation in a child with alanine aminotransferase >8 × ULN. Improvements in sweat chloride, weight, and body mass index z scores and fecal elastase-1 observed during KIWI were maintained during KLIMB; there was no further improvement in these parameters.
conclusionsIvacaftor was generally well tolerated for up to 108 weeks in children aged 2 to 5 years with CF and a gating mutation, with safety consistent with the KIWI study. Improvements in sweat chloride and growth parameters during the initial 24 weeks of treatment were maintained for up to an additional 84 weeks of treatment. Prevalence of raised transaminases remained stable and did not increase with duration of exposure during the open-label extension.
Indexed as
Identifiers
What OpenQuestion holds
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.