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Article History

Received: 17 January 2025

Accepted: 12 March 2025

First Online: 8 April 2025

Declarations

:

: Charles L. Daley: grant support, advisory board fees, and consulting fees from Insmed Incorporated. Dr. Daley also reports grant support from AN2 Therapeutics, Bugworks, Paratek Pharmaceuticals, Juvabis, FDA, NIH, PCORI, Cystic Fibrosis Foundation, COPD Foundation, and Renovion; advisory board work with AN2 Therapeutics, AstraZeneca, Cepheid, Galapagos, Hyfe, MannKind, Matinas Biopharma, NobHill, Spero Therapeutics, and Zambon; consulting with Galapagos, Genentech, and Pfizer; data monitoring committee work with Otsuka and Bill & Melinda Gates Foundation. James D. Chalmers: grant support from AstraZeneca, Boehringer Ingelheim, Genentech, Gilead Sciences, Grifols, GSK, Trudell, and Insmed Incorporated; consulting fees from Antabio, AstraZeneca, Boehringer Ingelheim, Chiese, Genentech, GSK, Insmed Incorporated, Pfizer, Trudell, and Zambon. Patrick A. Flume: grant support and consulting fees from Insmed Incorporated. David E. Griffith: Consulting fees, personal fees, and advisory board fees from Insmed Incorporated. Dr Griffith also reports consulting and advisory board fees from AN2 Therapeutics and Paratek Pharmaceuticals. Naoki Hasegawa: consulting fees, advisory board fees, and clinical trial design or participation from AN2 Therapeutics and Janssen Pharmaceuticals; consulting fees, advisory board fees, and personal fees from Insmed Incorporated; consulting fees and clinical trial design or participation from MannKind. Kozo Morimoto: consulting fees, personal fees, and advisory board fees from Boehringer Ingelheim and Insmed Incorporated. Kevin L. Winthrop: grant support and consulting fees from AN2 Therapeutics, Insmed Incorporated, MannKind, Paratek Pharmaceuticals, Renovion, and Spero Therapeutics. Korkut Avsar: personal fees from Insmed Incorporated. Luigi Ruffo Codecasa: consulting fees from Cepheid and Dia Sorin. Chau-Chyun Sheu and Dario Andrisani have nothing to report. Dayton W. Yuen, Mariam Hassan, Marie-Laure Nevoret, and Kevin Mange are employees and shareholders in Insmed Incorporated. Charles L. Daley, James D. Chalmers, Patrick A. Flume, David E. Griffith, Naoki Hasegawa, Kozo Morimoto, Kevin L. Winthrop, Luigi Ruffo Codecasa, Chau-Chyun Sheu, Korkut Avsar, and Dario Andrisani were investigators in the ARISE trial.

: A data monitoring committee periodically monitored the safety of patients in the study. ARISE was approved by the Advarra Institutional Review Board under protocol reference number Pro00045468 on 12 August 2020. ARISE also received ethics approval from all study sites. Participants and/or their legally authorized representative were informed that their participation was voluntary. Participants or their legally authorized representative were required to sign a statement of informed consent that met the requirements of 21 CFR 50, local regulations, ICH guidelines, HIPAA requirements. Participants did not consent for publication as no individual patient data are included. ARISE was conducted in compliance with its protocol and the ethical principles derived from international guidelines (Declaration of Helsinki, the Council for International Organizations of Medical Sciences International Ethical Guidelines), and applicable International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use Good Clinical Practice Guidelines, as well as applicable local laws and regulatory requirements.