Lumacaftor/Ivacaftor Combination Therapy in Subjects With CF Who Have an A455E CFTR Mutation

Phase 2 Completed 20 enrolled Vertex Pharmaceuticals Incorporated

RandomizedCrossoverQuadruple-blindTreatment

Summary

This is a Phase 2, randomized, double-blind, placebo-controlled, multicenter, crossover study that will evaluate the efficacy of LUM/IVA in subjects with CF 12 years of age and older who have at least one A455E mutation.

Timeline

Start: 2017-01-31
Primary completion: 2017-09-06
Completion: 2017-10-04

Outcome

Missed primary endpoint

registry analysis (superiority test); Placebo vs LUM/IVA; p = 0.9277; Least Squares Mean Difference 0.1 (95% CI -2.5 to 2.7); Mixed Model Repeated Measure (MMRM) NCT03061331 ↗

paper The primary endpoint (ppFEV1) did not show a statistically significant difference between LUM/IVA and placebo; PMID 33249003 ↗

Publications

Berkers G, van der Meer R, Heijerman H, Beekman JM, Boj SF, Vries RGJ, van Mourik P, Doyle JR, Audhya P, Yuan ZJ, Kinnman N, van der Ent CK. Lumacaftor/ivacaftor in people with cystic fibrosis with an A455E-CFTR mutation. J Cyst Fibros. 2021 Sep;20(5):761-767. doi: 10.1016/j.jcf.2020.11.007. Epub 2020 Nov 26.

Drugs

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