. Author manuscript; available in PMC: 2022 Jun 5.

Published in final edited form as: J Cyst Fibros. 2020 Jan 23;19(4):595–601. doi: 10.1016/j.jcf.2020.01.007

Table 2.

Primary and secondary efficacy results.

Efficacy endpoint	Endpoint hierarchy	LS mean	Difference (ataluren minus placebo)	Std error	95% CI	p-Value

ppFEV₁ change from baseline ^a	Primary
Ataluren		−1.396		0.6996	−2.7735, −0.0180
Placebo		−1.992		0.6777	−3.3271, −0.6576
Ataluren vs Placebo			0.597	0.957	−1.2881, 2.4813	0.5336
PEx rate, events/48 weeks	1st Secondary
Ataluren		0.950 (1.4038)^b		0.1195	0.7136, 1.1862
Placebo		1.127 (2.5241)^b		0.2164	0.6986, 1.5547
Ataluren/Placebo		0.8567^c				0.4008
BMI change from baseline, kg/m²	2nd Secondary
Ataluren		0.296		0.093	0.1126, 0.4789
Placebo		0.361		0.0938	0.1759, 0.5455
Ataluren vs Placebo			−0.065	0.1312	−0.3233, 0.1934	0.6208
Respiratory HRQL change from baseline	3rd Secondary
Ataluren		−0.76		1.3691	−3.4566, 1.9364
Placebo		−1.032		1.3733	−3.7368, 1.6728
Ataluren vs Placebo			0.272	1.93	−3.5292, 4.0731	0.8881

ppFEV₁, forced expiratory volume in 1 s; PEx, pulmonary exacerbation; BMI, body mass index; HQRL, health-related quality of life.

Mean of Week 40 and 48 minus baseline.

Mean (SD).

Ratio of ataluren to placebo rate.