Supplementary Material
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Supplementary methods and tables ERJ-00746-2021.Supplement
Supplementary figure S1. (a) Change from baseline in unadjusted mean (±SE) trough ppFEV1 at weeks 1 and 4 of treatment – TS and (b) LCI after 4 weeks of treatment – N2MBWS. No patients in the BI 1265162 20 μg BID treatment group were included in the N2MBWS. The BI 1265162 50 μg and 100 μg BID treatment groups only had one patient each included in the N2MBWS; accordingly, no standard error could be calculated for these groups. BI: BI 1265162; BID: twice daily; LCI: lung clearance index; N2MBWS: N2 multiple breath washout set; ppFEV1: percent predicted forced expiratory volume in 1 s; SE: standard error; TS: treated set. ERJ-00746-2021.Figure_S1
Supplementary figure S2. Change from baseline in ppFEV1 after 4 weeks of treatment for individual patients, all dose groups – TS. *: additional patient visit data added after interim analysis. BI: BI 1265162; MMRM: mixed model for repeated measures; ppFEV1: percent predicted forced expiratory volume in 1 s; TS: treated set. ERJ-00746-2021.Figure_S2
Supplementary figure S3. Sensitivity analysis for change from baseline in ppFEV1 after 4 weeks of treatment for individual patients (MMRM) – TS. Data from visits were excluded based on AEs that might have affected pulmonary function tests, compliance, and unacceptable pulmonary function test quality at baseline and/or baseline condition considered as important protocol deviation. AE: adverse event; BI: BI 1265162; MMRM: mixed model for repeated measures; ppFEV1: percent predicted forced expiratory volume in 1 s; TS: treated set. ERJ-00746-2021.Figure_S3
Supplementary figure S4. ppFEV1 subgroup analyses (MMRM) of difference between BI 1265162 and placebo from baseline – TS. BMI: body mass index; CF: cystic fibrosis; CFTR: cystic fibrosis transmembrane conductance regulator; CI: confidence interval; MMRM: mixed model for repeated measures; ppFEV1: percent predicted forced expiratory volume in 1 s; TS: treated set. ERJ-00746-2021.Figure_S4
Supplementary figure S5. Change from baseline in LCI after 4 weeks of treatment for individual patients, final analysis – N2MBWS. BI: BI 1265162; LCI: lung clearance index; N2MBWS: N2 multiple breath washout set. ERJ-00746-2021.Figure_S5
Supplementary figure S6. Correlation analysis between change in CFQ-R Respiratory Domain score and change in ppFEV1 after 4 weeks of treatment. BI: BI 1265162; CFQ-R RD: Cystic Fibrosis Questionnaire – Revised Respiratory Domains; ppFEV1: percent predicted forced expiratory volume in 1 s. ERJ-00746-2021.Figure_S6