NCT03150719

A Study to Evaluate Safety, Efficacy, and Tolerability of TEZ/IVA in Orkambi® (Lumacaftor/Ivacaftor) -Experienced Subjects With Cystic Fibrosis (CF)

Phase 3b, Randomized, Double-blind, Placebo-controlled, Parallel Group Study to Assess the Safety, Efficacy, and Tolerability of Tezacaftor/Ivacaftor (TEZ/IVA) in an Orkambi-experienced Population Who Are Homozygous for the F508del CFTR Mutation

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 98 (actual)
• Sponsor: Vertex Pharmaceuticals Incorporated · Industry
• Sex: All
• Age: 12 Years
• Healthy volunteers: Not accepted

Summary

Study VX16-661-114 (Study 114) is a Phase 3b, randomized, double-blind, placebo-controlled, parallel-group, multicenter study in subjects aged 12 years and older with CF who are homozygous for the F508del mutation on the cystic fibrosis transmembrane conductance regulator gene (CFTR) gene and who discontinued treatment with Orkambi due to respiratory symptoms considered related to treatment. This study is designed to evaluate the safety and efficacy of Tezacaftor/Ivacaftor (TEZ/IVA).

Conditions

• Cystic Fibrosis

Interventions

Type	Name	Description
DRUG	Tezacaftor/Ivacaftor	TEZ 100 mg/IVA 150 mg fixed-dose combination tablet.
DRUG	Ivacaftor	IVA 150 mg tablet.
DRUG	Placebo	Placebo matched to TEZ/IVA fixed-dose combination tablet.
DRUG	Placebo	Placebo matched to IVA tablet.

Timeline

• Start date: 2017-05-24
• Primary completion: 2018-08-09
• Completion: 2018-08-09
• First posted: 2017-05-12
• Last updated: 2019-09-12
• Results posted: 2019-09-12

Locations

53 sites across 3 countries: United States, France, Germany

Regulatory

• FDA-regulated drug study