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Article

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 - 1 Mar 2021

Davies Jane C, Wainwright Claire E, Sawicki Gregory S, Higgins Mark N, Campbell Daniel, Harris Christopher, Panorchan Paul, Haseltine Eric, Tian Simon, Rosenfeld Margaret

Abstract excerpt

Rationale: We previously reported that ivacaftor was safe and well tolerated in cohorts aged 12 to <24 months with cystic fibrosis and gating mutations in the ARRIVAL study; here, we report results for cohorts aged 4 to <12 months.Objectives: To evaluate the safety, pharmacokinetics, and pharmacodynamics of ivacaftor in infants aged 4 to <12 months with one or more gating mutations.Methods: ARRIVAL is a...

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Identifiers

PMID: 33023304
DOI: 10.1164/rccm.202008-3177OC
PMCID: PMC7924576

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-07.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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