A Phase 3 Study of VX-659 Combination Therapy in Subjects With Cystic Fibrosis Heterozygous for the F508del Mutation and a Minimal Function Mutation (F/MF)

Summary

This study will evaluate the efficacy of VX-659 in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in subjects with cystic fibrosis (CF) who are heterozygous for F508del and a minimal function mutation (F/MF subjects).

Timeline

Start

2018-03-07

Primary completion

2019-02-05

Completion

2019-02-05

Outcome

Met primary endpoint

registry analysis (superiority test); Placebo vs VX-659/TEZ/IVA TC; p <0.0001; Least Squares (LS) Mean Difference 14.0 (95% CI 12.4 to 15.7) NCT03447249 ↗

release “14.0 percentage points from baseline at week 4 in people with one F508del mutation and one minimal function mutation compared to placebo” vrtx.com ↗

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	BAMOCAFTOR	Small molecule	240 mg	Oral
Subject	Ivacaftor	Other / unclassified	150 mg	Oral
Subject	tezacaftor	Other / unclassified	100 mg	Oral

Indications