Efficacy and Safety of Elexacaftor/Tezacaftor/Ivacaftor in Children 6 Through 11 Years of Age with Cystic Fibrosis Heterozygous for F508del and a Minimal Function Mutation: A Phase 3b, Randomized, Placebo-controlled Study.

doi:10.1164/rccm.202202-0392OC

Loading publication...

Noodle always counts identifier-free feature events for service operation. With your permission, Google Analytics can also estimate audience and engagement. Search text, publication identifiers, cookies, IP addresses and browser fingerprints are never included in Noodle's first-party event counters.

Privacy details

Community

Back to search

Article

Efficacy and Safety of Elexacaftor/Tezacaftor/Ivacaftor in Children 6 Through 11 Years of Age with Cystic Fibrosis Heterozygous for F508del and a Minimal Function Mutation: A Phase 3b, Randomized, Placebo-controlled Study.

American journal of respiratory and critical care medicine - 1 Dec 2022

Mall Marcus A, Brugha Rossa, Gartner Silvia, Legg Julian, Moeller Alexander, Mondejar-Lopez Pedro, Prais Dario, Pressler Tacjana, Ratjen Felix, Reix Philippe, Robinson Paul D, Selvadurai Hiran, Stehling Florian, Ahluwalia Neil, Arteaga-Solis Emilio, Bruinsma Bote G, Jennings Mark, Moskowitz Samuel M, Noel Sabrina, Tian Simon, Weinstock Tanya G, Wu Pan, Wainwright Claire E, Davies Jane C

Abstract excerpt

Rationale: The triple-combination regimen elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) was shown to be safe and efficacious in children aged 6 through 11 years with cystic fibrosis and at least one F508del-CFTR allele in a phase 3, open-label, single-arm study. Objectives: To further evaluate the efficacy and safety of ELX/TEZ/IVA in children 6 through 11 years of age with cystic fibrosis heterozygous for...

Read the complete abstract on PubMed

Topics

Share this publication in a Topic to start or enrich a Post.

Share to Aminophenols

Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

Share to Benzodioxoles

Share to Child

Share to Chloride Channel Agonists

Share to Cystic Fibrosis

Share to Cystic Fibrosis Transmembrane Conductance Regulator

Share to Forced Expiratory Volume

Share to Mutation

Source

Open in PubMed Open full text

Identifiers

PMID: 35816621
DOI: 10.1164/rccm.202202-0392OC
PMCID: PMC9746869

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-08.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

Related research

Semantic proximity does not establish scientific evidence.

Research trail→

Click a neighbor to travelStep 1 · 12 closest

Efficacy and Safety of Elexacaftor/Tezacaftor/Ivacaftor in Children 6 Through 11 Years of Age with Cystic Fibrosis Heterozygous for F508del and a Minimal Function Mutation: A Phase 3b, Randomized, Placebo-controlled Study.PMID 35816621Read ↗

A Phase 3 Open-Label Study of Elexacaftor/Tezacaftor/Ivacaftor in Children 6 through 11 Years of Age with Cystic Fibrosis and at Least One F508del Allele.Semantic proximity Read ↗

Phase 3 Open-Label Clinical Trial of Elexacaftor/Tezacaftor/Ivacaftor in Children Aged 2-5 Years with Cystic Fibrosis and at Least One F508del Allele.Semantic proximity Read ↗

Long-Term Safety and Efficacy of Elexacaftor/Tezacaftor/Ivacaftor in Children ≥6 Years with Cystic Fibrosis and at Least One F508del Allele: A 192-Week, Phase 3, Open-Label Extension Study.Semantic proximity Read ↗

Long-Term Safety and Efficacy of Elexacaftor/Tezacaftor/Ivacaftor in Adults and Adolescents with Cystic Fibrosis and at Least One F508del Allele: A Phase 3 Open-Label Extension StudySemantic proximity Read ↗

Effectiveness and safety of elexacaftor/tezacaftor/ivacaftor in patients with cystic fibrosis and advanced lung disease with the Phe508del/minimal function genotype.Semantic proximity Read ↗

A phase 3, double-blind, parallel-group study to evaluate the efficacy and safety of tezacaftor in combination with ivacaftor in participants 6 through 11 years of age with cystic fibrosis homozygous for F508del or heterozygous for the F508del-CFTR mutation and a residual function mutation.Semantic proximity Read ↗

Long-Term Safety and Efficacy of Elexacaftor/Tezacaftor/Ivacaftor in Children Aged ⩾6 Years with Cystic Fibrosis and at Least One F508del Allele: A Phase 3, Open-Label Clinical Trial.Semantic proximity Read ↗

Long-term safety and efficacy of elexacaftor/tezacaftor/ivacaftor in people with cystic fibrosis and at least one F508del allele: 144-week interim results from a 192-week open-label extension study.Semantic proximity Read ↗

Safety and Efficacy of Triple Therapy in Cystic Fibrosis in Pediatric Patients of above 6 Years of Age: A Meta-Analysis of TRIKAFTA (Elexacaftor/Tezacaftor/Ivacaftor)Semantic proximity Read ↗

Elexacaftor-Tezacaftor-Ivacaftor for Cystic Fibrosis with a Single Phe508del Allele.Semantic proximity Read ↗

Efficacy and safety of elexacaftor plus tezacaftor plus ivacaftor versus tezacaftor plus ivacaftor in people with cystic fibrosis homozygous for F508del-CFTR: a 24-week, multicentre, randomised, double-blind, active-controlled, phase 3b trial.Semantic proximity Read ↗

A retrospective analysis of clinical parameters, safety and tolerability of Elexacaftor/Tezacaftor/Ivacaftor in a cohort of patients aged 6-17 years with cystic fibrosisSemantic proximity Read ↗

⌁Select a neighboring publication to make it the new centre.