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Article History

Received: 23 August 2023

Accepted: 17 October 2023

First Online: 27 October 2023

Declarations

:

: This work is an ancillary study to the parent randomized, placebo-controlled OPTIMIZE clinical trial. Eligible participants were recruited from 45 Cystic Fibrosis Foundation–accredited centers in the United States. This research was performed in accordance with the Declaration of Helsinki and was approved by each of the 45 centers institutional review boards including the Colorado Multiple Institutional Review Board (CoMIRB #14–0088) where the OP samples were processed (all site review boards are listed in the supplementary section). Written informed consent and HIPPA Authorization were obtained from all patients over 17 years or from parents or legal guardians of patients younger than 18 years. Assent was obtained from patients between 10 and 17 years. A DSMB monitored the trial using pre-specified stopping guidelines for efficacy and safety, further details related to participant consent and approval can be found in the trial registration (NCT02054156).

: Not Applicable.

: The authors SDS, JKH, CER and MJS declare that they have no competing interests. BDW received honoraria for CFF DSMB participation. NM-H received honoraria for NIH DSMB participation and consulting fees from Enterprise Therapeutics. BWR received consulting fees from Vertex Pharmaceuticals, Cystetic Medicines and Sionna Therapeutics. Dr. Ramsey also serves as Chair of an NHLBI DSMB. ETZ received consulting fees from Vertex Pharmaceuticals and received honoraria from CFF. GR-B received grant funding from Vertex Pharmaceuticals.