A Study of VX-445 Combination Therapy in CF Subjects Homozygous for F508del (F/F)

Phase 3 Completed 113 enrolled Vertex Pharmaceuticals Incorporated

RandomizedParallel-groupQuadruple-blindTreatment

Summary

This study will evaluate the efficacy of VX-445 in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in subjects with cystic fibrosis (CF) who are homozygous for the F508del mutation (F/F)

Timeline

Start: 2018-08-03
Primary completion: 2018-12-28
Completion: 2018-12-28

Outcome

Met primary endpoint

paper The elexacaftor plus tezacaftor plus ivacaftor group had improvements in the primary outcome of ppFEV1 PMID 31679946 ↗

registry analysis (superiority test); TEZ/IVA vs VX-445/TEZ/IVA TC; p <0.0001; Least squares (LS) mean difference 10 (95% CI 7.4 to 12.6) NCT03525548 ↗

Publications

Heijerman HGM, McKone EF, Downey DG, Van Braeckel E, Rowe SM, Tullis E, Mall MA, Welter JJ, Ramsey BW, McKee CM, Marigowda G, Moskowitz SM, Waltz D, Sosnay PR, Simard C, Ahluwalia N, Xuan F, Zhang Y, Taylor-Cousar JL, McCoy KS; VX17-445-103 Trial Group. Efficacy and safety of the elexacaftor plus tezacaftor plus ivacaftor combination regimen in people with cystic fibrosis homozygous for the F508del mutation: a double-blind, randomised, phase 3 trial. Lancet. 2019 Nov 23;394(10212):1940-1948. doi: 10.1016/S0140-6736(19)32597-8. Epub 2019 Oct 31.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	elexacaftor	Small molecule	200 mg	Oral
Background	Ivacaftor	Other / unclassified	150 mg	Oral
Comparator	tezacaftor	Other / unclassified	100 mg	Oral