A Study Evaluating the Safety and Efficacy of VX-659 Combination Therapy in Subjects With Cystic Fibrosis

• Status: Completed
• Trial end date: 2018-02-28
• Target enrollment: 0
• Participant gender: All

Summary

This is a Phase 2, randomized, double-blind, placebo- and tezacaftor/ivacaftor (TEZ/IVA)-controlled, parallel-group, 3-part, multicenter study designed to evaluate the safety and efficacy of VX-659 in triple combination (TC) with TEZ and IVA in subjects with cystic fibrosis (CF) who are homozygous for the F508del mutation of the CF transmembrane conductance regulator (CFTR) gene (F/F genotype), or who are heterozygous for the F508del mutation and a minimal function (MF) CFTR mutation not likely to respond to TEZ, IVA, or TEZ/IVA (F/MF genotypes).

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Vertex Pharmaceuticals Incorporated
• Treatments: Ivacaftor; VX-659

Criteria

Key Inclusion Criteria:

• Body weight ≥35 kg.

• Subjects must have an eligibleCFTR genotype.

• Part 1 and Part 3: Heterozygous for F508del and an MF mutation (F/MF)

• Part 2: Homozygous for F508del (F/F)

• FEV1 value ≥40% and ≤90% of predicted mean for age, sex, and height

Key Exclusion Criteria:

• History of clinically significant cirrhosis with or without portal hypertension.

• Glucose-6-phosphate dehydrogenase (G6PD) deficiency

• Lung infection with organisms associated with a more rapid decline in pulmonary status.

• History of solid organ or hematological transplantation.

Other protocol defined Inclusion/Exclusion criteria may apply.