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Table 5 Summary of clinical studies investigating the efficacy of lumacaftor and ivacaftor in patients with Phe508del mutations

From: Targeted therapies to improve CFTR function in cystic fibrosis

Study name and reference Boyle et al. 2014 [70] TRAFFIC and TRANSPORT: Wainwright et al. 2015 [8]
Type of study Phase II RCT Phase III RCT
Number of participants and study design Cohort 1: Cohort 1:
64 homozygotes n = 368
Lumacaftor 200 mg/day for 14 days Lumacaftor 600 mg/day plus ivacaftor 250 mg every 12 h
Followed by: Cohort 2:
Ivacaftor 150 mg/250 mg every 12 h for 7 days n = 369
OR Lumacaftor 400 mg every 12 h plus ivacaftor 250 mg every 12 h
Placebo for 21 days Cohort 3:
Cohorts 2 and 3: n = 371
96 homozygotes Placabo plus placebo
28 compound heterozygotes  
Cohort 2:
Lumacaftor 200 mg, 400 mg, 600 mg/day for 56 days
Cohort 3:
Lumacaftor 400 mg every 12 h for 56 days
Followed by:
Ivacaftor 250 mg every 12 h after 28 days
OR
Placebo for 56 days
Duration Cohort 1: 21 days 24 weeks
Cohorts 2 and 3: 56 days
Inclusion criteria ≥18 years ≥12 years
>1 Phe508del allele Phe508del homozygous
FEV1 > 40 % FEV1 40–90 %
Outcome measure Treatment effect Pooled analysis of treatment effect in TRAFFIC and TRANSORT
Mean FEV1 (percentage predicted) Cohort 2 with lumacaftor 600 mg/day: +5.6 (P = 0.013) Cohort 1: +3.3 (P < 0.001)
Cohort 3: no significant differences Cohort 2: +2.8 (P < 0.001)
Sweat chloride levels (mmol/L) Cohort 1 with 250 mg ivacaftor: −9.1 mmol/L (P < 0.001) Cohorts 2 and 3: no significant differences
CFQ-R score (points) Cohort 1: 3.1 (P = 0.007)
Cohort 2: 2.2 (P = 0.05)
BMI Cohort 1: 0.28 (P < 0.001)
Cohort 2: 0.24 (P < 0.001)
Pulmonary exacerbations Cohort 1: rate ratio 0.7 (P = 0.001)
Cohort 2: rate ratio 0.61 (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