Rollover Study to Evaluate the Safety and Efficacy of Long-term Treatment With Lumacaftor in Combination With Ivacaftor

Phase 3 Completed 246 enrolled Vertex Pharmaceuticals Incorporated

Non-randomizedParallel-groupOpen-labelTreatment

Summary

Study 110 is a Phase 3, multicenter study in subjects aged 6 years and older with cystic fibrosis (CF) who are homozygous for the F508del-CF transmembrane conductance regulator (CFTR) mutation and who participated in Study 109 (NCT02514473) or Study 011B (NCT01897233). Study 110 is designed to evaluate the safety and efficacy of long term treatment of lumacaftor in combination with ivacaftor.

Timeline

Start: 2015-08
Primary completion: 2018-08
Completion: 2020-04

Publications

Chilvers MA, Davies JC, Milla C, Tian S, Han Z, Cornell AG, Owen CA, Ratjen F. Long-term safety and efficacy of lumacaftor-ivacaftor therapy in children aged 6-11 years with cystic fibrosis homozygous for the F508del-CFTR mutation: a phase 3, open-label, extension study. Lancet Respir Med. 2021 Jul;9(7):721-732. doi: 10.1016/S2213-2600(20)30517-8. Epub 2021 Jan 28.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Ivacaftor	Other / unclassified	250 mg	Oral
Subject	Lumacaftor	Small molecule	200 mg	Oral
Subject	Lumacaftor	Small molecule	400 mg	Oral

Rollover Study to Evaluate the Safety and Efficacy of Long-term Treatment With Lumacaftor in Combination With Ivacaftor

Summary

Timeline

Publications

Drugs

Indications