Table 1.
Summary of clinical trials: ivacaftor monotherapy
| Measure, Test, and Assay |
||||||||||
|---|---|---|---|---|---|---|---|---|---|---|
| Study Name | Trial Phase No. | Mutations | Age Group, yr | Sample Size | ppFEV1 | BMI | CFQ-R | SwCl | Conclusion (Failed or Approved) | Reference |
| STRIVE | 3 | G551D | ≥12 | 161 | +10.6% (P < 0.001) [24] | +2.7 kg (P < 0.001) [24] | +3.2 points (P < 0.001) [24] | −47.9 mmol/l (P < 0.001) [24] | Approved | (23) |
| ENVISION | 3 | G551D | 6–11 | 52 | +12.5% (P < 0.001) [24] | +1.9 kg (P < 0.001) [24] | +6.0 points (P > 0.05 [24] | −54.3 mmol/l (P < 0.0010 [24] | (24) | |
| KONNECTION | 3 | G1244E; G1349; G178R; G511S; G970R; S1251N; S1255P; S549N; S549R | ≥6 | 39 | +10.7% (P < 0.0001) [8] | +0.7 kg/m2 (P < 0.0001) [8] | +9.6 points (P < 0.0004) [8] | −49.2 mmol/l (P < 0.0001) [8] | (25) | |
| KONDUCT | 3 | R117H | ≥6 yr | 69 | +2.1% (P = 0.2) [24] | +0.26 kg/m2 (P = 0.78) [24] | +8.4 points (P = 0.009) [24] | −24.0 mmol/l (P < 0.001) [24] | (27) | |
| KIWI | 3 | G551D; S549N | 2–5 | 34 | +0.4 kg/m2 (P < 0.0001) [24] | −46.9 mmol/l (P < 0.0001) [24] | (28) | |||
| DISCOVER | 2 | Homozygous F508de1 | ≥12 | 140 | +1.7% (P = 0.15) [16] | −0.04 kg/m2 (P > 0.05) [16] | +1.3 points (P > 0.05) [16] | −2.9 mmol/l (P = 0.04) [16] | Failed | (29) |
Numbers within brackets indicate n. SwCl, sweat chloride levels.