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

July 11, 2023 updated by: Vertex Pharmaceuticals Incorporated

A Phase 3b Open-label Extension Study Evaluating the Safety of Elexacaftor/Tezacaftor/Ivacaftor Combination Therapy in Cystic Fibrosis Subjects

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

172

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Australia
      • Chermside, Australia
        • The Prince Charles Hospital
      • Nedlands, Australia
        • Institute for Respiratory Health
      • Nedlands, Australia
        • Telethon Kids Institute, Perth Children's Hospital
      • Parkville, VIC, Australia
        • The Royal Children's Hospital
      • South Brisbane, Australia
        • Queensland CHILDREN'S HOSPITAL
  • Belgium
      • Gent, Belgium
        • Universitair Ziekenhuis Gent
      • Leuven, Belgium
        • Universitair Ziekenhuizen Leuven - Campus Gasthuisberg
  • Germany
      • Berlin, Germany
        • Charite Paediatric Pulmonology Department
      • Essen, Germany
        • Ruhrlandklinik Westdeutsches Lungenzentrum am Klinikum Essen
      • Essen, Germany
        • Universitatsklinikum Essen (AoR), Kinderklinik III, Abt. fur Pneumologie
      • Jena, Germany
        • Mukeviszidose-Zentrum am Universitatsklinikum Jena, Klinik fuer Kinder- und Jugendmedizin
      • Koeln, Germany
        • Universitaetsklinkum Koeln, CF-Studienzentrum
      • München, Germany
        • Klinikum Innenstadt, University of Munich
  • United Kingdom
      • Belfast, United Kingdom
        • Belfast City Hospital
      • Birmingham, United Kingdom
        • University Hospitals Birmingham NHS Foundation Trust
      • Bristol, United Kingdom
        • University Hospitals Bristol and Weston NHS Foundation Trust, Bristol Royal Hospital
      • Cambridge, United Kingdom
        • Royal Papworth Hospital NHS Foundation Trust
      • Edinburgh, United Kingdom
        • Western General Hospital
      • Exeter, United Kingdom
        • Royal Devon and Exeter NHS Foundation Trust, Royal Devon and Exeter Hospital
      • Glasgow, United Kingdom
        • Clinical Research Facility, Queen Elizabeth University Hospital
      • Leeds, United Kingdom
        • Leeds General Infirmary
      • Leeds, United Kingdom
        • St. James University Hospital
      • Liverpool, United Kingdom
        • Alder Hey Children's NHS Foundation Trust
      • London, United Kingdom
        • Great Ormond Street Hospital for Sick Children
      • London, United Kingdom
        • London and St Bartholomew's Hospital
      • Newcastle Upon Tyne, United Kingdom
        • The Newcastle upon Tyne Hospitals NHS Foundation Trust, The Royal Victoria Infirmary
      • Nottingham, United Kingdom
        • Nottingham University Hospitals NHS Trust, Queens Medical Center
      • Penarth, United Kingdom
        • All Wales Adult Cystic Fibrosis Centre, University Hospital Llandough
      • Southampton, United Kingdom
        • Southampton General Hospital

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

12 years and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

Key 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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: 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.
Drug: IVA
Tablet for oral administration.
Other Names:
  • VX-770
  • ivacaftor
Drug: ELX/TEZ/IVA
Fixed-dose combination (FDC) tablet for oral administration.
Other Names:
  • VX-445/VX-661/VX-770
  • elexacaftor/tezacaftor/ivacaftor

What is the study measuring?

Primary Outcome Measures

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

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Vertex Pharmaceuticals Incorporated

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

May 4, 2020

Primary Completion (Actual)

December 21, 2022

Study Completion (Actual)

December 21, 2022

Study Registration Dates

First Submitted

April 22, 2020

First Submitted That Met QC Criteria

April 22, 2020

First Posted (Actual)

April 27, 2020

Study Record Updates

Last Update Posted (Actual)

July 28, 2023

Last Update Submitted That Met QC Criteria

July 11, 2023

Last Verified

July 1, 2023

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • VX19-445-115
  • 2019-003455-11 (EudraCT Number)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

IPD Plan Description

Details on Vertex data sharing criteria and process for requesting access can be found at: https://www.vrtx.com/independent research/clinical-trial-data-sharing.

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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