NCT04353817

A Study Evaluating Efficacy and Safety of Elexacaftor/Tezacaftor/Ivacaftor in Subjects 6 Through 11 Years of Age With Cystic Fibrosis and F/MF Genotypes

A Phase 3b, Randomized, Placebo-controlled Study Evaluating the Efficacy and Safety of Elexacaftor/Tezacaftor/Ivacaftor in Cystic Fibrosis Subjects 6 Through 11 Years of Age Who Are Heterozygous for the F508del Mutation and a Minimal Function Mutation (F/MF)

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 121 (actual)
• Sponsor: Vertex Pharmaceuticals Incorporated · Industry
• Sex: All
• Age: 6 Years – 11 Years
• Healthy volunteers: Not accepted

Summary

This study will evaluate the efficacy and safety of elexacaftor (ELX) / tezacaftor (TEZ) / ivacaftor (IVA) triple combination (TC) in subjects 6 through 11 years of age with cystic fibrosis (CF) who are heterozygous for F508del and a minimal function (MF) mutation (F/MF genotypes).

Conditions

• Cystic Fibrosis

Interventions

Type	Name	Description
DRUG	ELX/TEZ/IVA	Fixed-dose combination tablet for oral administration qd in the morning.
DRUG	IVA	Tablet for oral administration qd in the evening.
OTHER	Placebo (matched to ELX/TEZ/IVA)	Placebo matched to ELX/TEZ/IVA for oral administration once daily (qd) in the morning.
OTHER	Placebo (matched to IVA)	Placebo matched to IVA for oral administration qd in the evening.

Timeline

• Start date: 2020-06-19
• Primary completion: 2021-05-17
• Completion: 2021-05-17
• First posted: 2020-04-20
• Last updated: 2022-07-26
• Results posted: 2022-07-26

Locations

34 sites across 10 countries: Australia, Canada, Denmark, France, Germany, Israel, Netherlands, Spain, Switzerland, United Kingdom

Regulatory

• FDA-regulated drug study