Evaluation of Efficacy and Safety of Elexacaftor/Tezacaftor/Ivacaftor (ELX/TEZ/IVA) in Cystic Fibrosis Subjects Without an F508del Mutation

Summary

This study evaluated the efficacy, pharmacodynamics (PD) and safety of ELX/TEZ/IVA in participants 6 years of age and older with a non-F508del ELX/TEZ/IVA-responsive cystic fibrosis transmembrane conductance regulator gene (CFTR) mutation.

Timeline

Start

2022-05-09

Primary completion

2023-07-05

Completion

2023-07-05

Outcome

Met primary endpoint

registry analysis (superiority test); Placebo; p < 0.0001; LS Mean difference 9.2 (95% CI 7.2 to 11.3) NCT05274269 ↗

release “Phase 3, randomized, placebo-controlled clinical study in people with rare non-F508del KAFTRIO®-responsive CFTR mutations. This study met its primary endpoint” vrtx.com ↗

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Ivacaftor	Other / unclassified	75 mg	Oral
Subject	Ivacaftor	Other / unclassified	150 mg	Oral
Subject	elexacaftor	Small molecule	100 mg	Oral
Subject	elexacaftor	Small molecule	200 mg	Oral
Subject	tezacaftor	Other / unclassified	50 mg	Oral
Subject	tezacaftor	Other / unclassified	100 mg	Oral

Indications