Table 2.
Efficacy Results.*
| End Point | Elexacaftor–Tezacaftor–Ivacaftor (N = 132) | Active Control: Ivacaftor or Tezacaftor–Ivacaftor (N = 126) |
|---|---|---|
| Percentage of predicted FEV1 | ||
| Value at baseline | 67.1±15.7 | 68.1±16.4 |
| Absolute change from baseline through wk 8 | ||
| Sample size† | 115 | 114 |
| Least-squares mean change (95% CI) | 3.7 (2.8 to 4.6)‡§ | 0.2 (−0.7 to 1.1) |
| Between-group difference (95% CI) | 3.5 (2.2 to 4.7)¶‖ | |
| Sweat chloride concentration | ||
| Value at baseline — mmol/liter | 59.5±27.0 | 56.4±25.5 |
| Absolute change from baseline through wk 8 | ||
| Sample size† | 120 | 119 |
| Least-squares mean change (95% CI) — mmol/liter | −22.3 (−24.5 to −20.2)§¶ | 0.7 (−1.4 to 2.8) |
| Between-group difference (95% CI) — mmol/liter | −23.1 (−26.1 to −20.1)¶ | |
| CFQ-R respiratory domain score** | ||
| Value at baseline | 76.5±16.6 | 77.3±15.8 |
| Absolute change from baseline through wk 8 | ||
| Sample size†† | 130 | 126 |
| Least-squares mean change (95% CI) | 10.3 (8.0 to 12.7) | 1.6 (−0.8 to 4.1) |
| Between-group difference (95% CI) | 8.7 (5.3 to 12.1) | |
Plus–minus values are means ±SD. Baseline, considered to be the end of the run-in period, was defined as the most recent nonmissing measurement before the first dose of trial drug in the treatment period.
The sample size is the number of patients through week 8 with in-clinic data that could be evaluated.
The primary end point was the absolute change in the percentage of predicted FEV1 from baseline through week 8 in the elexacaftor–tezacaftor–ivacaftor group.
P<0.001 for the within-group change from baseline.
The key secondary end points, in hierarchical order, were the absolute change in sweat chloride concentration from baseline through week 8 in the elexacaftor–tezacaftor–ivacaftor group, the absolute change in the percentage of predicted FEV1 from baseline through week 8 for elexacaftor–tezacaftor–ivacaftor as compared with active control, and the absolute change in sweat chloride concentration from baseline through week 8 for elexacaftor–tezacaftor–ivacaftor as compared with active control.
P<0.001 for the between-group difference in the change from baseline.
Pooled CFQ-R data were obtained both in the clinic and in the home and were based on both the “Children Ages 12 and 13” and “Adolescents and Adults” versions. The minimal clinically important difference for the CFQ-R respiratory domain score is 4 points.
The sample size is the number of patients through week 8 with in-clinic or in-home data that could be evaluated.