From: Targeted therapies to improve CFTR function in cystic fibrosis
Study name and reference | Flume et al. 2012 [54] | KONNECTION: De Boeck et al. 2014 [51] | KONDUCT: Moss et al. 2015 [53] |
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Type of study | Phase II RCT with open label extension | Phase III randomized crossover trial with open label extension | Phase III RCT |
Number of participants | n = 104 | n = 39 | n = 69 |
Ivacaftor 34; placebo 35 | |||
Duration | 16 weeks (96-week extension) | 24 weeks (total) | 24 weeks |
8 weeks placebo/ivacaftor | |||
8 weeks ivacaftor/placebo | |||
12 weeks ivacaftor | |||
Inclusion criteria | ≥12 years | ≥6 years | ≥6 years |
Phe508del homozygous | >1 non-Gly551Asp gating mutation | >1 Arg117His mutation | |
FEV_{1} > 40 % | FEV_{1} > 40 % | FEV_{1} > 40–90 % (>12 years) | |
FEV_{1} > 40–105 % (6–11 years) | |||
Weight >15 kg | |||
Outcome measure | Treatment effect | Treatment effect after 8 weeks | Treatment effect |
Mean FEV_{1} (percentage predicted) | +1.7 (P = 0.15) | +10.7 (P < 0.0001) | All ages: +2.1 (P = 0.2) |
>18 years: +5 (P = 0.01) | |||
6–11 years: −6.3 (P = 0.03) | |||
Sweat chloride levels (mmol/L) | −2.9 (P = 0.04) | −49.2 (P < 0.0001) | −24 (P < 0.0001) |
CFQ-R score (points) | No significant differences | +9.6 (P = 0.0004) | +8.4 (P = 0.009) |
Weight (kg) | No significant differences | – | – |
BMI | No significant differences | BMI-for-age z-score 0.28 (P = 0.001) | – |