A Study Evaluating the Safety and Efficacy of VX-440 Combination Therapy in Subjects With Cystic Fibrosis
Summary
This is a Phase 2, randomized, double-blind, placebo- and active-controlled, parallel group, multicenter study to evaluate the safety, tolerability, and efficacy of VX-440 in dual and triple combination with tezacaftor (TEZ; VX-661) and ivacaftor (IVA; VX-770) in subjects with cystic fibrosis (CF) who are homozygous for the F508del mutation of the CF transmembrane conductance regulator (CFTR) gene (F508del/F508del), or who are heterozygous for the F508del mutation and a minimal function (MF) CFTR mutation not likely to respond to TEZ and/or IVA therapy (F508del/MF).
Timeline
- Start
- 2016-10
- Primary completion
- 2017-08
- Completion
- 2017-08
Outcome
Met primary endpoint
registry analysis (superiority test); Part 1: Placebo - Cohort 1A and 1B Combined vs Part 1: TC-1A/ TC-1B-low Pooled; p = 0.0016 NCT02951182 ↗
registry p = 0.0016; Least Squares (LS) Mean Difference 8.6 (95% CI 3.5 to 13.8) NCT02951182 ↗
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | IVA | Unknown | 150 mg | — |
| Subject | IVA | Unknown | 300 mg | — |
| Subject | OLACAFTOR | Small molecule | 200 mg | — |
| Subject | OLACAFTOR | Small molecule | 600 mg | — |
| Subject | TEZ | Unknown | 50 mg | — |
| Subject | TEZ | Unknown | 100 mg | — |