TABLE 4.
Secondary efficacy end-points (F508del–F508del (F/F) genotype)
|
Parent Study 445-103 (F/F genotype)
at Week 4 # |
Study 445-105 at Extended Week 144
(F/F genotype) |
|||
|
TEZ/IVA
(n=52) |
ELX/TEZ/IVA
(n=55) |
TEZ/IVA
→ ELX/TEZ/IVA (n=52) |
ELX/TEZ/IVA
→ ELX/TEZ/IVA (n=55) |
|
| Absolute change in FEV1 % pred, percentage points | 0.4 (−1.4–2.3) | 10.4 (8.6–12.2) | 12.0 (9.5–14.5) n=44 |
11.6 (9.1–14.0) n=48 |
| Absolute change in sweat chloride concentration, mmol·L−1 | 1.7 (−1.9–5.3) | −43.4 (−46.9– −40.0) | −53.4 (−57.7– −49.0) n=42 |
−49.9 (−54.1– −45.7) n=47 |
| Absolute change in CFQ-R respiratory domain score, points | −1.4 (−5.4–2.6) | 16.0 (12.1–19.9) | 13.9 (9.2–18.6) n=45 |
18.2 (13.6–22.7) n=48 |
| Absolute change in BMI, kg·m−2 | −0.07 (−0.21–0.06) | 0.53 (0.39–0.66) | 1.50 (1.11–1.89) n=44 |
1.74 (1.36–2.12) n=48 |
| Estimated pulmonary exacerbation event rate per 48 weeks ¶ | NA | NA | 0.18 (0.12–0.26) | |
Date are presented as least squares mean absolute change (95% CI) from baseline of the parent study, except for pulmonary exacerbation event rate (95% CI). TEZ/IVA: tezacaftor/ivacaftor; ELX/TEZ/IVA: elexacaftor/tezacaftor/ivacaftor; FEV1: forced expiratory volume in 1 s; CFQ-R: Cystic Fibrosis Questionnaire-Revised; BMI: body mass index; NA: not available. #: participants in Study 445-103 had a 4-week TEZ/IVA run-in period prior to baseline; ¶: calculated from cumulative ELX/TEZ/IVA exposure in the parent study (Study 445-103) and in the extension study (Study 445-105).