Trial reportAmerican journal of respiratory and critical care medicine2022
A Phase 3, Open-Label Study of Lumacaftor/Ivacaftor in Children 1 to Less Than 2 Years of Age with Cystic Fibrosis Homozygous for
Trial report in American journal of respiratory and critical care medicine, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 19 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.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
19 citing papers in PubMed, 2 syntheses or guidelines pooled it, 33 citations in OpenAlex.
- Pharmacokinetics of Ivacaftor, Tezacaftor, Elexacaftor, and Lumacaftor in Special Cystic Fibrosis Populations: A Systematic Review.Clinical pharmacokinetics · 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
- Early CFTR modulation and clinical outcomes in preschool cystic fibrosis.Pediatric research · 2026Article
- Targeted Therapy for Restoring CFTR Activity: From Experimental to Clinical Features.International journal of molecular sciences · 2026Review
- Real-life effectiveness and safety of lumacaftor/ivacaftor in preschool children with cystic fibrosis: data from an italian multicentre study.Pediatric research · 2026Article
- Utility of Fecal Elastase-1 in Estimating Exocrine Pancreatic Function in Cystic Fibrosis: A Scoping Review.Pediatric pulmonology · 2026Article
- Gut-lung microbial dynamics with lumacaftor/ivacaftor in children with cystic fibrosis: a prospective multicenter study.Pediatric research · 2026Article
- Role of elexacaftor-tezacaftor-ivacaftor therapy on fecal elastase-1 and fat-soluble vitamins in children with cystic fibrosis.European journal of pediatrics · 2026Article
- Post-marketing safety profile of lumacaftor/ivacaftor in cystic fibrosis treatment: a pharmacovigilance analysis based on FAERS.Frontiers in medicine · 2026Article
- Fecal calprotectin as a marker of intestinal inflammation in pediatric cystic fibrosis patients.European journal of pediatrics · 2025Article
- Impact of Elexacaftor/Tezacaftor/Ivacaftor on Fecal Elastase-1 in Children With Cystic Fibrosis.Pediatric pulmonology · 2025Article
- Body composition changes and clinical outcomes in pediatric cystic fibrosis during 24 months of lumacaftor ivacaftor therapy based on real-world data.Scientific reports · 2025Observational
- In utero and postnatal ivacaftor/lumacaftor therapy rescues multiorgan disease in CFTR-F508del ferrets.JCI insight · 2024Article
- ROCK STUDY in CF: sustained anti-inflammatory effects of lumacaftor-ivacaftor in sputum and peripheral blood samples of adult patients with cystic fibrosis-an observational study.BMJ open respiratory research · 2023Observational
- A year in review (2022): Modulators and COVID19, the story goes on….Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society · 2023Review
- Ocular development after highly effective modulator treatment early in life.Frontiers in pharmacology · 2023Review
- Mechanism and potential treatments for gastrointestinal dysfunction in patients with COVID-19.World journal of gastroenterology · 2022Review
- A New Era for Cystic Fibrosis and Cystic Fibrosis Transmembrane Conductance Regulator Modulator Trials in Infants.American journal of respiratory and critical care medicine · 2022Article
- Dual CFTR modulator therapy efficacy in the real world: lessons for the future.ERJ open research · 2022Article
Corrections and comments
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Authors and funding
10 authors at 5 institutions in 2 countries.
Funding
Abstract
PubMed holds no abstract for this paper.
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.