NCT05274269

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

A Phase 3 Double-blind, Randomized, Placebo-controlled Study Evaluating the Efficacy and Safety of ELX/TEZ/IVA in Cystic Fibrosis Subjects 6 Years of Age and Older With a Non-F508del ELX/TEZ/IVA-responsive CFTR Mutation

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 307 (actual)
• Sponsor: Vertex Pharmaceuticals Incorporated · Industry
• Sex: All
• Age: 6 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Cystic Fibrosis

Interventions

Type	Name	Description
DRUG	ELX/TEZ/IVA	Fixed-dose combination (FDC) tablets for oral administration.
DRUG	IVA	Tablet for oral administration.
OTHER	Placebo (matched to ELX/TEZ/IVA)	Placebo matched to ELX/TEZ/IVA for oral administration.
OTHER	Placebo (matched to IVA)	Placebo matched to IVA for oral administration.

Timeline

• Start date: 2022-05-09
• Primary completion: 2023-07-05
• Completion: 2023-07-05
• First posted: 2022-03-10
• Last updated: 2024-08-01
• Results posted: 2024-08-01

Locations

90 sites across 15 countries: Austria, Belgium, Canada, Czechia, France, Germany, Hungary, Italy, Netherlands, Norway, Poland, Portugal, Spain, Sweden, Switzerland

Regulatory

• FDA-regulated drug study