TABLE 3.
Secondary efficacy end-points (F508del–minimal function (F/MF) genotypes)
| Parent Study 445-102 (F/MF genotypes) through Week 24 |
Study 445-105 at Extended Week 144
(F/MF genotypes) |
|||
|
Placebo
(n=203) |
ELX/TEZ/IVA
(n=200) |
Placebo
→ ELX/TEZ/IVA (n=203) |
ELX/TEZ/IVA
→ ELX/TEZ/IVA (n=196) |
|
| Absolute change in FEV1 % pred, percentage points | −0.4 (−1.5–0.7) | 13.9 (12.8–15.0) | 14.8 (13.3–16.3) n=161 |
14.1 (12.6–15.6) n=166 |
| Absolute change in sweat chloride concentration, mmol·L−1 | −0.4 (−2.2–1.4) | −42.2 (−44.0– −40.4) | −50.5 (−53.4– −47.7) n=146 |
−47.2 (−49.9– −44.4) n=160 |
| Absolute change in CFQ-R respiratory domain score, points | −2.7 (−4.6 to −0.8) | 17.5 (15.6–19.5) | 17.6 (14.9–20.2) n=175 |
19.1 (16.4–21.8) n=171 |
| Absolute change in BMI, kg·m−2 | 0.09 (−0.05–0.22)# | 1.13 (0.99–1.26)# | 1.76 (1.48–2.05) n=167 |
1.61 (1.32–1.90) n=169 |
| Estimated pulmonary exacerbation event rate per 48 weeks | 0.98 | 0.37 | 0.20 (0.16–0.24)¶ | |
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). ELX/TEZ/IVA: elexacaftor/tezacaftor/ivacaftor; FEV1: forced expiratory volume in 1 s; CFQ-R: Cystic Fibrosis Questionnaire-Revised; BMI: body mass index. #: for BMI, parent study result represents mean absolute change from baseline at Week 24; ¶: calculated from cumulative ELX/TEZ/IVA exposure in the parent study (Study 445-102) and in the extension study (Study 445-105).