A Study Evaluating the Long-Term Safety of Elexacaftor Combination Therapy

Phase 3

Completed

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

• Registration Number: NCT04362761
• Lead Sponsor: Vertex Pharmaceuticals Incorporated

Brief Summary

This study evaluate the long-term safety and tolerability of elexacaftor (ELX)/tezacaftor (TEZ)/ ivacaftor (IVA) triple combination (TC) in participants with cystic fibrosis (CF) who are homozygous for F508del.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2020-04-22
• Study First Submitted QC: 2020-04-22
• Study First Posted: 2020-04-27
• Study Start: 2020-05-04
• Primary Completion: 2022-12-21
• Study Completion: 2022-12-21
• Results First Submitted: 2023-06-20
• Status Verified: 2023-07
• Results First Submitted QC: 2023-07-11
• Last Update Submitted: 2023-07-11
• Results First Posted: 2023-07-28
• Last Update Posted: 2023-07-28

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 172

Inclusion Criteria

• Completed study drug treatment in parent study (VX18-445-109); 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 study drug intolerance in 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	IVA	Part A: Participants received elexacaftor (ELX) 200 milligram (mg) once daily (qd)/tezacaftor (TEZ)100 mg qd/ivacaftor (IVA)150 mg every 12 hours (q12h) in the treatment period for 48 weeks. Part B: Participants received ELX 200 mg qd/TEZ 100 mg qd/IVA 150 mg q12h in the treatment period up to 86 weeks.
ELX/TEZ/IVA	ELX/TEZ/IVA	Part A: Participants received elexacaftor (ELX) 200 milligram (mg) once daily (qd)/tezacaftor (TEZ)100 mg qd/ivacaftor (IVA)150 mg every 12 hours (q12h) in the treatment period for 48 weeks. Part B: Participants received ELX 200 mg qd/TEZ 100 mg qd/IVA 150 mg q12h in the treatment period up to 86 weeks.

Primary Outcome Measures

Name	Time	Method
Part A: Safety and Tolerability as Assessed by Number of Participants With Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)	From Day 1 up to Week 52
Part B: Safety and Tolerability as Assessed by Number of Participants With Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)	From Day 1 up to Week 86

Trial Locations

Locations (29)

Telethon Kids Institute, Perth Children's Hospital; 🇦🇺 Nedlands, Australia

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

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

The Prince Charles Hospital; 🇦🇺 Chermside, Australia

Institute for Respiratory Health; 🇦🇺 Nedlands, Australia

Charite Paediatric Pulmonology Department; 🇩🇪 Berlin, Germany

Mukeviszidose-Zentrum am Universitatsklinikum Jena, Klinik fuer Kinder- und Jugendmedizin; 🇩🇪 Jena, Germany

Universitaetsklinkum Koeln, CF-Studienzentrum; 🇩🇪 Koeln, Germany

Klinikum Innenstadt, University of Munich; 🇩🇪 München, Germany

Belfast City Hospital; 🇬🇧 Belfast, United Kingdom

University Hospitals Birmingham NHS Foundation Trust; 🇬🇧 Birmingham, United Kingdom

Western General Hospital; 🇬🇧 Edinburgh, United Kingdom

University Hospitals Bristol and Weston NHS Foundation Trust, Bristol Royal Hospital; 🇬🇧 Bristol, United Kingdom

Clinical Research Facility, Queen Elizabeth University Hospital; 🇬🇧 Glasgow, United Kingdom

Royal Devon and Exeter NHS Foundation Trust, Royal Devon and Exeter Hospital; 🇬🇧 Exeter, United Kingdom

Leeds General Infirmary; 🇬🇧 Leeds, United Kingdom

Alder Hey Children's NHS Foundation Trust; 🇬🇧 Liverpool, United Kingdom

St. James University Hospital; 🇬🇧 Leeds, United Kingdom

Great Ormond Street Hospital for Sick Children; 🇬🇧 London, United Kingdom

London and St Bartholomew's Hospital; 🇬🇧 London, United Kingdom

The Newcastle upon Tyne Hospitals NHS Foundation Trust, The Royal Victoria Infirmary; 🇬🇧 Newcastle Upon Tyne, United Kingdom

Nottingham University Hospitals NHS Trust, Queens Medical Center; 🇬🇧 Nottingham, United Kingdom

All Wales Adult Cystic Fibrosis Centre, University Hospital Llandough; 🇬🇧 Penarth, United Kingdom

Southampton General Hospital; 🇬🇧 Southampton, United Kingdom

Royal Papworth Hospital NHS Foundation Trust; 🇬🇧 Cambridge, United Kingdom

Universitair Ziekenhuis Gent; 🇧🇪 Gent, Belgium

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

Ruhrlandklinik Westdeutsches Lungenzentrum am Klinikum Essen; 🇩🇪 Essen, Germany

Universitatsklinikum Essen (AoR), Kinderklinik III, Abt. fur Pneumologie; 🇩🇪 Essen, Germany