NCT05033080

A Phase 3 Study of VX-121 Combination Therapy in Participants With Cystic Fibrosis (CF) Heterozygous for F508del and a Minimal Function Mutation (F/MF)

A Phase 3, Randomized, Double-blind, Controlled Study Evaluating the Efficacy and Safety of VX-121 Combination Therapy in Subjects With Cystic Fibrosis (CF) Who Are Heterozygous for F508del and a Minimal Function Mutation (F/MF)

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 435 (actual)
• Sponsor: Vertex Pharmaceuticals Incorporated · Industry
• Sex: All
• Age: 12 Years
• Healthy volunteers: Not accepted

Summary

The purpose of this study evaluates the efficacy and safety of VX-121/tezacaftor/deutivacaftor (VX-121/TEZ/D-IVA) in CF participants who were heterozygous for F508del and a minimal function mutation (F/MF participants).

Conditions

• Cystic Fibrosis

Interventions

Type	Name	Description
DRUG	VX-121/TEZ/D-IVA	Fixed-dose combination tablets for oral administration.
DRUG	ELX/TEZ/IVA	Fixed-dose combination tablets for oral administration.
DRUG	IVA	Tablet for oral administration.
DRUG	Placebo (matched to VX-121/TEZ/D-IVA)	Placebo matched to VX-121/TEZ/D-IVA for oral administration.
DRUG	Placebo (matched to ELX/TEZ/IVA)	Placebo matched to ELX/TEZ/IVA for oral administration.
DRUG	Placebo (matched to IVA)	Placebo matched to IVA for oral administration.

Timeline

• Start date: 2021-09-14
• Primary completion: 2023-05-12
• Completion: 2023-11-21
• First posted: 2021-09-02
• Last updated: 2024-10-03
• Results posted: 2024-06-10

Locations

139 sites across 12 countries: United States, Australia, Czechia, Germany, Hungary, Ireland, Israel, New Zealand, Portugal, Spain, Sweden, United Kingdom

Regulatory

• FDA-regulated drug study