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Article History

Received: 29 May 2026

Accepted: 3 July 2026

First Online: 16 July 2026

Declarations

Conflict of Interest: Doreen Addrizzo-Harris: clinical trial support to the institution from AN2 Therapeutics, Armata Pharmaceuticals, Chiesi, Boehringer Ingelheim, Hillrom, Insmed Incorporated, Novartis, Sanofi, Verona, and Zambon; consulting fees from AstraZeneca, Boehringer Ingelheim, Clarametyx, MannKind, Paratex, and Sanofi; and member of the data and safety monitoring board for MannKind. James D. Chalmers: grants and personal fees from AstraZeneca, Boehringer Ingelheim, GSK, Insmed Incorporated, and Zambon; a grant from Gilead; and personal fees from Chiesi and Novartis. Stefano Aliberti: grant support from GSK; consulting fees from AN2 Therapeutics, AstraZeneca, Boehringer Ingelheim, BRAHMS GmbH, Chiesi Farmaceutica SpA, CSL Behring GmbH, Fondazione Internazionale Menarini, GlaxoSmithKline SpA, Insmed Incorporated, Menarini, Moderna, MSD Italia Srl, Pfizer, PhysioAssist SAS, Verona Pharma PLC, Vertex Pharmaceuticals, and Zambon SpA; speaking fees from Boehringer Ingelheim, Fondazione Internazionale Menarini, GlaxoSmithKline SpA, Insmed Incorporated, Vertex Pharmaceuticals, and Zambon; data monitoring committee work with AstraZeneca, Insmed Incorporated, MSD Italia Srl, and Verona Pharma PLC. Pierre-Régis Burgel: grants from GSK and Vertex, outside the submitted work; clinical trials advisory activity for AstraZeneca, Chiesi, GSK, Insmed Incorporated, MSD, Vertex, Viatris, and Zambon; substantial financial contributions to the budget of his institution from Association Vaincre la Mucoviscidose, Filière MUCO-CFTR, GSK, Société Française de la Mucoviscidose, and Vertex. Pierre-Régis Burgel is an editorial board member of Pulmonary Therapy. Pierre-Régis Burgel was not involved in the selection of peer reviewers for the manuscript nor any of the subsequent editorial decisions. Brian M. Morrissey: grants from the Cystic Fibrosis Foundation and clinical trial support to the institution from Boehringer Ingelheim, CSL Behring, Insmed Incorporated, US National Institutes of Health, and Vertex Pharmaceuticals. Xiangmin Zhang, Chunpeng Fan, Melanie Lauterio, Sebastian Fucile, Dianne Griffis, and Ariel Teper: employees and shareholders of Insmed Incorporated.

Ethical Approval: The trial was conducted in accordance with the principles of the Declaration of Helsinki, the Good Clinical Practice guidelines of the International Council for Harmonisation, and applicable regulatory requirements. An institutional review board or independent ethics committee approved the protocol at each participating site (details provided as supplementary material). All participants provided their written informed consent before starting the study. Adolescents provided their signed assent if required per local requirements, and a parent or legal guardian provided their signed informed consent.