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. Author manuscript; available in PMC: 2017 Apr 22.
Published in final edited form as: Expert Rev Precis Med Drug Dev. 2016 Apr 22;1(3):235–243. doi: 10.1080/23808993.2016.1175299

Table 2.

Summary of phase 2 (Boyle et al [49]) and phase 3 (Wainwright et al [52]) studies with ivacaftor-lumacaftor combination treatment in CF patients ages 12 and older who are homozygous for F508del-CFTR.

Study FEV1* BMI CFQ-R Pulmonary exacerbations§ Number of participants
Phase 2 study, 8 weeks [37]
Lumacaftor 200 mg/day 3.8 (−0.4 to 8.1) 23
Ivacaftor 250 mg twice/day P= 0.077
Lumacaftor 400 mg/day 2.7 (−1.7 to 7.0) 21
Ivacaftor 250 mg twice/day P= 0.225
Lumacaftor 600 mg/day 5.6 (1.2 to 10.0) 21
Ivacaftor 250 mg twice/day P= 0.013
Lumacaftor 400 mg twice/day 4.2 (−1.3 to 9.6) 11
Ivacaftor 250 mg twice/day P= 0.132
Phase 3 study, 24 weeks [38]
TRAFFIC
Lumacaftor 600 mg/day 4.0 (2.6 to 5.4) 0.16 (−0.04 to 0.35) 3.9 (0.7 to 7.1) 0.72 (0.52 to 1.00) 183
Ivacaftor 250 mg twice/day P< 0.001 P= 0.11 P= 0.02 P= 0.05
Lumacaftor 400 mg twice/day 2.6 (1.2 to 4.0) 0.13 (−0.07 to 0.32) 1.5 (−1.7 to 4.7) 0.66 (0.47 to 0.93) 182
Ivacaftor 250 mg twice/day P< 0.001 P= 0.19 P= 0.36 P= 0.02
TRANSPORT
Lumacaftor 600 mg/day 2.6 (1.2 to 4.1) 0.41 (0.23 to 0.59) 2.2 (−0.9 to 5.3) 0.69 (0.52 to 0.92) 185
Ivacaftor 250 mg twice/day P< 0.001 P< 0.001 P= 0.17 P= 0.01
Lumacaftor 400 mg twice/day 3.0 (1.6 to 4.4) 0.36 (0.17 to 0.54) 2.9 (−0.3 to 6.0) 0.57 (0.42 to 0.76) 187
Ivacaftor 250 mg twice/day P< 0.001 P< 0.001 P= 0.07 P< 0.001
TRAFFIC + TRANSPORT Pooled
Lumacaftor 600 mg/day 3.3 (2.3 to 4.3) 0.28 (0.15 to 0.41) 3.1 (0.8 to 5.3) 0.70 (0.56 to 0.87) 368
Ivacaftor 250 mg twice/day P< 0.001 P< 0.001 P= 0.007 P= 0.001
Lumacaftor 400 mg twice/day 2.8 (1.8 to 3.8) 0.24 (0.11 to 0.37) 2.2 (0.0 to 4.5) 0.61 (0.49 to 0.76) 369
Ivacaftor 250 mg twice/day P< 0.001 P< 0.001 P= 0.05 P< 0.001
*

Difference versus placebo for absolute change in percentage predicted FEV1 (percentage points; 95% CI).

Difference versus placebo in absolute change from baseline BMI; mean (95% CI).

Difference versus placebo in absolute change from baseline in CFQ-R respiratory domain; mean (95% CI). The minimal clinically important difference for stable patients on this scale is 4.0 points [53].

§

Rate ratio (95% CI)