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Table 2 Summary of clinical studies investigating the efficacy of ivacaftor in patients with cystic fibrosis and the Gly551Asp mutation

From: Targeted therapies to improve CFTR function in cystic fibrosis

Study name and reference Accurso et al. 2010 [42] STRIVE: Ramsey et al. 2011 [6] ENVISION: Davies et al. 2013 [43] Davies et al. 2013 [44] Barry et al. 2014 [45]
Type of study Phase II RCT Phase III RCT Phase III RCT Phase III RCT Case–control study
Number of participants n = 39 n = 161 n = 52 n = 21 n = 56
Ivacaftor: 31; placebo: 8 Ivacaftor: 83; placebo: 78 Ivacaftor: 26; placebo: 22   Ivacaftor: 21; placebo: 35
Duration 28 days 48 weeks 48 weeks 29 days 9 months
Inclusion criteria ≥18 years ≥12 years 6–11 years ≥6 years ≥18 years
≥1 Gly551Asp allele ≥1 Gly551Asp allele ≥1 Gly551AspP allele ≥1 Gly551Asp allele ≥1 Gly551Asp allele
FEV1 > 40 % FEV1 40–90 % FEV1 40–105 % FEV1 > 90 % FEV1 < 40 %
Weight ≥15 kg LCI >7.4 and/or actively listed for lung transplant
Weight ≥15 kg
Outcome measure Median change from baseline with 150 mg Treatment effect Treatment effect Treatment effect Changes within treated patients Treated patients versus controls
Mean FEV1 (percentage predicted) +8.7 (P = 0.008) 24 weeks: +10.6 (P < 0.001) 24 weeks: +12.5 (P < 0.001) +4.2 (P = 0.0068) +3.8 versus 0.6 (P = 0.009; median)
48 weeks: +10.5 (P < 0.001) 48 weeks: +10 (P < 0.0006)
Sweat chloride levels (mmol/L) −59.5 (P = 0.008) −47.9 (P < 0.001) −54.3 (P < 0.001)
CFQ-R score (points) +8.3 (P = 0.06) +8.6 (P < 0.001) +6.1 (P = 0.109)
Nasal potential difference (mV) −3.5 (P = 0.02)
Weight (kg) +2.7 (P < 0.001) +2.8 (P < 0.001) +1.8 (P = 0.0058; median) +2.3 versus 0.6 (P = 0.25; median)
BMI BMI-for-age z-score: 0.45 (P < 0.001) +1.1 kg/m2 (P = 0.010; median) +0.84 versus 0.2 kg/m2 (P = 0.234; median)
Time on intravenous antibiotics (days per year) −36 (P = 0.0016; median) −36 versus +10 (P = 0.0003; median)
Pulmonary exacerbations 55 % risk reduction No significant difference
(0.455 hazard ratio: P = 0.001)
LCI −2.16 (P < 0.0001)
  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, LCI lung clearance index, RCT randomized controlled trial