Conditions: Cystic Fibrosis, Homozygous for the F508del CFTR Mutation
Allocation: RANDOMIZED | Intervention model: PARALLEL
Enrollment: 559
Healthy volunteers: no

Brief summary:
The primary objective of the study was to evaluate the efficacy of lumacaftor in combination with ivacaftor at Week 24 in participants aged 12 years and older with cystic fibrosis (CF) who are homozygous for the F508del mutation on the CF transmembrane conductance regulator (CFTR) gene.

Detailed description:
This was a Phase 3, randomized, double-blind, placebo-controlled, parallel-group multicenter study of orally administered lumacaftor in combination with ivacaftor in participants aged 12 years and older with CF who are homozygous for the F508del-CFTR mutation.

The study included a Screening Period (Day -28 through Day -1), a Treatment Period (Day 1 \[first dose of study drug\] to Week 24 ± 5 days), and a Safety Follow-up Visit (4 weeks ± 7 days after the Week 24 Visit).

Arms:
arm 1: Placebo matched to lumacaftor (LUM, VX-809) and ivacaftor (IVA, VX-770) tablet every 12 hours (q12h), up to Week 24. arm 2: LUM 600 milligram (mg) plus IVA 250 mg fixed-dose combination (FDC) tablet in the morning and IVA 250 mg film-coated tablet in the evening, up to Week 24. arm 3: LUM 400 mg plus IVA 250 mg FDC tablet in the morning and in the evening, up to Week 24.

Interventions: intervention 1: Lumacaftor Plus Ivacaftor Combination intervention 2: Ivacaftor intervention 3: Placebo
Intervention descriptions: intervention 1: Fixed dose combination tablet intervention 2: Film-coated tablet intervention 3: Matching placebo tablet

Protocol document: