Conditions: Cystic Fibrosis
Keywords: ∆F508 F508del Cystic Fibrosis Transmembrane Conductance Regulator CFTR Pancreatic diseases Lung diseases Genetic disease, inborn Infant, newborn, diseases
Allocation: RANDOMIZED | Intervention model: PARALLEL
Enrollment: 93
Healthy volunteers: no

Brief summary:
The primary objective of the study was to evaluate the safety and tolerability of VX-809 in participants 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 2, randomized, double-blind, placebo-controlled, multiple-dose study of orally-administered VX-809 in participants with CF who are homozygous for the specific CFTR mutation known as ∆F508 or F508del. Enrollment was planned for 90 participants at approximately 20 centers. Participants were planned to be randomized in a 4:1 ratio to receive 1 of 4 doses of VX-809 or placebo once a day for 28 days in a parallel design. Participants were outpatients during the study, except for overnight stays on Day 1 and 28.

Arms:
arm 1: Placebo matched to VX-809 capsule orally once daily for 28 days. arm 2: VX-809, 25 milligram (mg) capsule orally once daily for 28 days. arm 3: VX-809, 50 mg capsule orally once daily for 28 days. arm 4: VX-809, 100 mg capsule orally once daily for 28 days. arm 5: VX-809, 200 mg capsule orally once daily for 28 days.

Interventions: intervention 1: VX-809 intervention 2: Placebo
Intervention descriptions: intervention 1: Capsules intervention 2: Placebo matched to VX-809 capsules.

Protocol document: