TABLE 3.
Change in trough percentage predicted forced expiratory volume in 1 s (ppFEV1) and lung clearance index (LCI) after 4 weeks of treatment with BI 1265162 (BI) 200 µg twice daily: final analysis
| Patients, n | Trough ppFEV1 (TS)¶ | LCI (ANCOVA)+ | p-value# | |||
| Change from baseline | 95% CI# | Change from baseline | 95% CI# | |||
| MMRM¶ | ||||||
| Placebo | 18 | –1.0±1.70 | –4.5–2.4 | |||
| BI 200 µg | 16 | 0.5±1.77 | –3.2–4.1 | |||
| BI 200 µg versus placebo | 1.5±2.45 | –3.5–6.5 | 0.5468 | |||
| N2MBWS+ | ||||||
| Placebo | 6 | –1.3±1.01 | –3.7–1.2 | |||
| BI 200 µg | 3 | 0.8±1.46 | –2.8–4.4 | |||
| BI 200 µg versus placebo | 2.1±1.83 | –2.4–6.5 | 0.3039 | |||
| Sensitivity analyses | ||||||
| MMRM, pre-specified¶,§ | ||||||
| Placebo | 17 | –0.4±1.65 | –3.8–3.0 | |||
| BI 200 µg | 15 | 2.3±1.78 | –1.4–6.0 | |||
| BI 200 µg versus placebo | 2.7±2.43 | –2.3–7.7 | 0.2761 | |||
| Quantile regression, post hoc | ||||||
| All visitsƒ | ||||||
| Placebo | 17 | 54.6±2.2## | 50.2–59.0 | |||
| BI 200 µg | 16 | 58.6±1.8## | 54.8–62.3 | |||
| BI 200 µg versus placebo | 4.0±2.8## | –1.8–9.7 | 0.1728 | |||
| Visit data excluded§,ƒ | ||||||
| Placebo | 14 | 53.8±2.5## | 48.7–58.9 | |||
| BI 200 µg | 12 | 59.4±2.0## | 55.3–63.5 | |||
| BI 200 µg versus placebo | 5.7±3.5## | –1.6–12.9 | 0.1193 | |||
Data are presented as adjusted mean±se, unless otherwise stated. MMRM: mixed model for repeated measures; TS: treated set; ANCOVA: analysis of covariance; N2MBWS: nitrogen multiple-breath washout set. #: confidence intervals and p-values are provided for reference only and inference should not be drawn; ¶: adjusted mean based on MMRM with fixed effects for baseline, visit, treatment, treatment-by-visit interaction, baseline-by-visit interaction, and random effect for patient; +: adjusted mean based on ANCOVA with fixed effects for baseline and treatment; §: data from visits were excluded based on adverse events 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; ƒ: estimate of median is using overall median of baseline; ##: data presented as median estimate±se.