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. 2014 Jul;39(7):500-511.

Table 3.

Lumacaftor Clinical Trials

Reference
Design 		CFTR Mutation Population Treatment
Duration 		Results
Clancy (2012)48
Double-blind, placebo-controlled 		Homozygous F508del Age 18–54 years
N = 89
FEV1 ≥ 40%		 LUM 25 mg q.d.
LUM 50 mg q.d.
LUM 100 mg q.d.
LUM 200 mg q.d.
PBO 28 days
  • Mean change in sweat chloride from baseline to day 28 compared with PBO:
    • ○ LUM 25 mg: 0.1 mmol/L
    • ○ LUM 50 mg: −4.61 mmol/L
    • ○ LUM 100 mg: −6.13 mmol/L (95% CI, −12.25 to −0.01; P < 0.05)
    • ○ LUM 200 mg: −8.21 mmol/L (95% CI, −14.33 to −2.1; P < 0.01)
  • Number of pulmonary exacerbations (P = 0.62):
    • ○ LUM, 17%; PBO, 12%
  • Mean change in FEV1 from baseline to 28 days (P = NS):
    • ○ LUM 25 mg: −2.46%
    • ○ LUM 50 mg: −2.15%
    • ○ LUM 100 mg: 0.32%
    • ○ LUM 200 mg: 0.47%
    • ○ PBO: 0.07%
  • CFQ-R change from baseline to 28 days:
    • ○ LUM 25 mg: −5.2 pts
    • ○ LUM 50 mg: −6.3 pts
    • ○ LUM 100 mg: −1.3 pts
    • ○ LUM 200 mg: 2.2 pts
    • ○ PBO: 4.5 pts
Boyle (2011)50 Randomized, double-blind, placebo-controlled, multicenter, cohort Homozygous F508del Age > 18 years
N = 62
FEV1 ≥ 40%		 Part 1: LUM 200 mg q.d. or PBO 14 days Part 2: LUM 200 mg q.d. + IVA 150 mg b.i.d., or LUM 200 mg q.d. + LUM 250 mg b.i.d., or PBO 7 days of treatment
  • Change in FEV1 percentage from day 1 to day 14:
    • ○ LUM 200 mg, −0.34%; PBO, 1.73%
  • Change in FEV1 percentage from day 14 to day 21:
    • ○ LUM + IVA 150 mg b.i.d.: 3.42% (P < 0.05 compared with baseline)
    • ○ LUM + IVA 250 mg b.i.d.: 0.57%
    • ○ PBO: −1.47%
  • Mean change in sweat chloride from day 1 to day 14:
    • ○ LUM 200 mg, −4.21 mmol/L; PBO, −2.86 mmol/L
  • Mean change in sweat chloride from day 14 to day 28:
    • ○ LUM + IVA 150 mg b.i.d.: −1.65 mmol/L
    • ○ LUM + IVA 250 mg b.i.d.: −8.96 mmol/L (P < 0.05 compared with PBO)
    • ○ PBO: 0.86 mmol/L
Boyle (2012)51 Randomized, placebo-controlled Homozygous F508del Age > 18 years
N = 82		 Period 1: LUM 200 mg, 400 mg, 600 mg q.d. or PBO 28 days Period 2: period 1 treat- ment + IVA 250 mg b.i.d. or PBO 28 days
  • Change in absolute FEV1 percent predicted from baseline at 28 days of combination treatment (end of period 2):
    • ○ LUM 200 mg + IVA: 1.9%
    • ○ LUM 400 mg + IVA: 0.6%
    • ○ LUM 600 mg + IVA: 3.4%;
    • ○ PBO: −3.3%
  • Sweat chloride change compared to PBO in LUM 600-mg group:
    • ○ End of period 1: −6.41 mmol/L
    • ○ Additional reduction at end of period 2: −2.82 mmol/L
Heterozygous F508del Age > 18 years
N = 27		 Period 1: LUM 600 mg q.d. or PBO 28 days Period 2: period 1 treat- ment + IVA 250 mg b.i.d. or PBO 28 days
  • Change in absolute FEV1 percent predicted from baseline at 28 days of combination treatment (end of period 2):
    • ○ LUM 600 mg + IVA: −1.3%
    • ○ PBO: −3.7%

b.i.d. = twice daily; CFTR = cystic fibrosis transmembrane conductance regulator gene; CFQ-R = Cystic Fibrosis Questionnaire-Revised; CI = confidence interval; FEV1 = forced expiratory volume in 1 second; IVA = ivacaftor; LUM = lumacaftor; NS = nonsignificant; PBO = placebo; pts = points,