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Table 3 Summary of clinical studies investigating the efficacy of ivacaftor in patients with cystic fibrosis mutations other than Gly551Asp

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]
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
FEV1 > 40 % FEV1 > 40 % FEV1 > 40–90 % (>12 years)
FEV1 > 40–105 % (6–11 years)
Weight >15 kg
Outcome measure Treatment effect Treatment effect after 8 weeks Treatment effect
Mean FEV1 (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)
  1. BMI body mass index (the weight in kilograms divided by the square of the height in meters), CFQ-R revised Cystic Fibrosis Questionnaire, FEV 1 percentage predicted forced expiratory volume in 1 second for age, sex and height, RCT randomized controlled trial