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Article

A Phase 3, Open-Label Study of Lumacaftor/Ivacaftor in Children 1 to Less Than 2 Years of Age with Cystic Fibrosis Homozygous for F508del-CFTR.

American journal of respiratory and critical care medicine - 15 Nov 2022

Rayment Jonathan H, Asfour Fadi, Rosenfeld Margaret, Higgins Mark, Liu Lingyun, Mascia Molly, Paz-Diaz Hildegarde, Tian Simon, Zahigian Rachel, McColley Susanna A

Abstract excerpt

Rationale: Previous phase 3 trials showed that treatment with lumacaftor/ivacaftor was safe and efficacious in people aged ⩾2 years with cystic fibrosis (CF) homozygous for the F508del mutation in CFTR (CF transmembrane conductance regulator) (F/F genotype). Objectives: To assess the safety, pharmacokinetics, and pharmacodynamics of lumacaftor/ivacaftor in children aged 1 to <2 years with the F/F genotype....

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Identifiers

PMID: 35771568
DOI: 10.1164/rccm.202204-0734OC
PMCID: PMC9746849

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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.

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A Phase 3, Open-Label Study of Lumacaftor/Ivacaftor in Children 1 to Less Than 2 Years of Age with Cystic Fibrosis Homozygous for F508del-CFTR.PMID 35771568Read ↗

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