A Study Evaluating the Safety of Elexacaftor/Tezacaftor/Ivacaftor in Participants With Cystic Fibrosis (CF)

Phase 3

Completed

• Conditions: Cystic Fibrosis
• Interventions: Drug: ELX/TEZ/IVA; Drug: IVA

• Registration Number: NCT05111145
• Lead Sponsor: Vertex Pharmaceuticals Incorporated

Brief Summary

The purpose of this study is to evaluate the safety and tolerability of elexacaftor (ELX)/tezacaftor (TEZ)/ivacaftor (IVA) in participants with CF who are 12 years of age or older.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2021-10-27
• Study First Submitted QC: 2021-10-27
• Study First Posted: 2021-11-08
• Study Start: 2022-01-14
• Primary Completion: 2022-12-20
• Study Completion: 2022-12-20
• Status Verified: 2023-06
• Results First Submitted: 2023-06-19
• Results First Submitted QC: 2023-06-19
• Last Update Submitted: 2023-06-19
• Results First Posted: 2023-07-11
• Last Update Posted: 2023-07-11

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 86

Inclusion Criteria

• Completed study drug treatment in parent study (VX19-445-117 [NCT04599465] or VX20-445-126 [NCT04969224]), or had study drug interruption(s) in parent study but completed study visits up to the last scheduled visit of the treatment period in the parent study

Key

Exclusion Criteria

• History of drug intolerance in a parent study

Other protocol defined Inclusion/Exclusion criteria may apply.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
ELX/TEZ/IVA	ELX/TEZ/IVA	Participants received ELX 200 mg once daily (qd)/TEZ 100 mg qd/IVA 150 mg every 12 hours (q12h) in the treatment period for up to 36 weeks.
ELX/TEZ/IVA	IVA	Participants received ELX 200 mg once daily (qd)/TEZ 100 mg qd/IVA 150 mg every 12 hours (q12h) in the treatment period for up to 36 weeks.

Primary Outcome Measures

Name	Time	Method
Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)	Day 1 up to Week 36

Trial Locations

Locations (24)

The Prince Charles Hospital; 🇦🇺 Chermside, Australia

Alfred Hospital; 🇦🇺 Melbourne, Australia

Institute for Respiratory Health; 🇦🇺 Nedlands, Australia

Telethon Kids Institute; 🇦🇺 Nedlands, Australia

Mater Adult Hospital; 🇦🇺 South Brisbane, Australia

Queensland Children's Hospital; 🇦🇺 South Brisbane, Australia

Queen Elizabeth II Health Sciences Center; 🇨🇦 Halifax, Canada

University of Calgary Medical Clinic of the Foothills Medical Centre; 🇨🇦 Calgary, Canada

Stollery Children's Hospital; 🇨🇦 Edmonton, Canada

Klinika Nemoci Plicnich a Tuberkulozy; 🇨🇿 Brno, Czechia

Fakultni nemocnice v Motole; 🇨🇿 Praha 5, Czechia

Hospital Saint Joan de Deu; 🇪🇸 Barcelona, Spain

Hospital Universitari Vall d Hebron; 🇪🇸 Barcelona, Spain

Hospital Universitario 12 de Octubre; 🇪🇸 Madrid, Spain

Hospital Universitario Ramon y Cajal; 🇪🇸 Madrid, Spain

Hospital Virgen de la Arrixaca; 🇪🇸 Murcia, Spain

Corporacio Sanitaria Parc Tauli - Sabadell Hospital Universitari; 🇪🇸 Sabadell, Spain

Hospital Universitario Virgen del Rocio; 🇪🇸 Sevilla, Spain

Universitair Ziekenhuis Gent; 🇧🇪 Gent, Belgium

Cliniques Universitaires de Bruxelles Hopital Erasme; 🇧🇪 Brussels, Belgium

Universitair Ziekenhuis Brussel - Campus Jette; 🇧🇪 Brussels, Belgium

Universitaire Ziekenhuizen Leuven - Campus Gasthuisberg; 🇧🇪 Leuven, Belgium

The Royal Children's Hospital; 🇦🇺 Parkville, Australia

Hospital Universitari Vall d´Hebron Servicio de Broncoscopia; 🇪🇸 Barcelona, Spain