Evaluation of VX-121/Tezacaftor/Deutivacaftor in Cystic Fibrosis (CF) Participants 1 Through 11 Years of Age

Phase 3 Active not recruiting 210 enrolled Vertex Pharmaceuticals Incorporated

Non-randomizedSequentialOpen-labelTreatment

Summary

The purpose of this study is to evaluate the pharmacokinetics, safety, tolerability and efficacy of VX-121/tezacaftor/deutivacaftor (VX-121/TEZ/D-IVA) in CF participants with at least 1 triple combination responsive (TCR) mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.

Timeline

Start: 2022-06-21
Primary completion: 2030-06-30
Completion: 2030-06-30

Publications

Hoppe JE, Kasi AS, Pittman JE, Jensen R, Thia LP, Robinson P, Tirakitsoontorn P, Ramsey B, Mall MA, Taylor-Cousar JL, McKone EF, Tullis E, Salinas DB, Zhu J, Chen YC, Rodriguez-Romero V, Sosnay PR, Davies G; VX21-121-105 Study Group. Vanzacaftor-tezacaftor-deutivacaftor for children aged 6-11 years with cystic fibrosis (RIDGELINE Trial VX21-121-105): an analysis from a single-arm, phase 3 trial. Lancet Respir Med. 2025 Mar;13(3):244-255. doi: 10.1016/S2213-2600(24)00407-7. Epub 2025 Jan 2.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Vanzacaftor	Small molecule	12 mg	Oral
Subject	Vanzacaftor	Small molecule	20 mg	Oral
Subject	deutivacaftor	Unknown	150 mg	Oral
Subject	deutivacaftor	Unknown	250 mg	Oral
Subject	tezacaftor	Other / unclassified	60 mg	Oral
Subject	tezacaftor	Other / unclassified	100 mg	Oral

Evaluation of VX-121/Tezacaftor/Deutivacaftor in Cystic Fibrosis (CF) Participants 1 Through 11 Years of Age

Summary

Timeline

Publications

Drugs

Indications