Table 2.
Overview of SIMPLIFY study endpoints
| Primary endpoint |
| The primary endpoint in each study is the mean absolute change in ppFEV1 from visit 1 (Week 0) to visit 3 (Week 6). |
| Secondary endpoints |
| Efficacy |
| • Mean change in LCI from visit 1 (Week 0) to visit 3 (Week 6) |
| • Mean change in ppFEV1 from visit 1 (Week 0) to visit 2 (Week 2) |
| • Proportion of subjects initiating acute antibiotics from visit 1 (Week 0) to visit 3 (Week 6) |
| • Proportion of subjects hospitalized from visit 1 (Week 0) to visit 3 (Week 6) |
| • Proportion of subjects with a pulmonary exacerbation from visit 1 (Week 0) to visit 3 (Week 6), defined according to expanded Fuchs criteria (31) |
| • Mean change in CRISS (32) from visit 1 (Week 0) to visit 3 (Week 6) |
| • Mean change in the CFQR respiratory-domain score (33) from visit 1 (Week 0) to visit 3 (Week 6) |
| • Mean change in ppFEV1 from screening to visit 1 (Week 0) |
| Safety |
| • Incidence of adverse events occurring between visit 1 (Week 0) to visit 3 (Week 6) |
| • Proportion of subjects temporarily or permanently changing their assigned therapy regimen between visit 1 (Week 0) to visit 3 (Week 6) |
| Exploratory endpoints |
| • Mean change in MCC from visit 1 (Week 0) to visit 3 (Week 6) |
| • Proportions of subjects remaining on and off hypertonic saline and dornase alfa for up to 24 wk after completion of each study |
| • Proportions of subjects with acute antibiotic use up to 24 wk after completion of each study |
| • Average impact score at visit 3 (Week 6) on subject perception of how stopping hypertonic saline or dornase alfa (or both) would impact their daily life |
Definition of abbreviations: CFQR = Cystic Fibrosis Questionnaire–Revised; CRISS = Chronic Respiratory Infection Symptom Score; LCI = lung-clearance index; MCC = mucociliary clearance; ppFEV1 = percent-predicted forced expiratory volume in 1 second.