A Study Evaluating the Efficacy and Safety of VX-445/Tezacaftor/Ivacaftor in Cystic Fibrosis Subjects, Homozygous for F508del

July 23, 2021 updated by: Vertex Pharmaceuticals Incorporated

A Phase 3b, Randomized, Double-blind, Controlled Study Evaluating the Efficacy and Safety of VX-445/Tezacaftor/Ivacaftor in Cystic Fibrosis Subjects, Homozygous for F508del

This study will evaluate the efficacy, safety, and pharmacodynamics of elexacaftor (ELX, VX-445) in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in subjects with cystic fibrosis (CF) who are homozygous for F508del.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

176

Phase

  • Phase 3

Expanded Access

Approved for sale to the public. See expanded access record.

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
        • Perth Children's Hospital
      • New Lambton, Australia
        • John Hunter Hospital & Hunter Medical Research Institute and John Hunter Children's Hospital
      • Parkville, VIC, Australia
        • The Royal Children's Hospital
      • South Brisbane, Australia
        • Queensland Children's Hospital
  • Belgium
      • Brussels, Belgium
        • Universitair Ziekenhuis Brussel - Campus Jette
      • Edegem, Belgium
        • UZ Antwerpen
      • Gent, Belgium
        • Universitair Ziekenhuis Gent
      • Leuven, Belgium
        • Universitaire 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
      • Frankfurt, Germany
        • Johann Wolfgang Goethe University
      • Jena, Germany
        • Mukeviszidose-Zentrum am Universitatsklinikum Jena, Klinik fuer Kinder- und Jugendmedizin
      • Koeln, Germany
        • Universitaetsklinkum Koeln, CF-Studienzentrum
      • Muenchen, Germany
        • Pneumologisches Studienzentrum Muenchen-West
      • 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 NHS Foundation Trust, Bristol Royal Hospital
      • Cambridge, United Kingdom
        • Papworth Hospital NHS Foundation Trust, Papworth Everard
      • 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
        • St. James University Hospital
      • Leeds, West Yorkshire, United Kingdom
        • Leeds General Infirmary
      • Liverpool, United Kingdom
        • Alder Hey Children's 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
      • Manchester, United Kingdom
        • The University Hospital of South Manchester
      • 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

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

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Key Inclusion Criteria:

  • Homozygous for the F508del mutation (F/F)
  • Forced expiratory volume in 1 second (FEV1) value ≥40% and ≤90% of predicted mean for age, sex, and height

Key Exclusion Criteria:

  • Clinically significant cirrhosis with or without portal hypertension
  • Lung infection with organisms associated with a more rapid decline in pulmonary status
  • Solid organ or hematological transplantation

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: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Active Comparator: TEZ/IVA
Following TEZ/IVA run-in period of 4 weeks, participants received TEZ 100 milligrams (mg) once daily (qd)/IVA 150 mg every 12 hours (q12h) in the treatment period for 24 weeks.
Drug: TEZ/IVA
Fixed-dose combination (FDC) tablet for oral administration.
Other Names:
  • VX-661/VX-770
  • tezacaftor/ivacaftor
Drug: IVA
Mono tablet for oral administration.
Other Names:
  • VX-770
  • ivacaftor
Experimental: ELX/TEZ/IVA
Following TEZ/IVA run-in period of 4 weeks, participants received ELX 200 mg qd/TEZ 100 mg qd/IVA 150 mg q12h in the treatment period for 24 weeks.
Drug: IVA
Mono tablet for oral administration.
Other Names:
  • VX-770
  • ivacaftor
Drug: ELX/TEZ/IVA
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
Measure Description
Time Frame
Absolute Change in CF Questionnaire-Revised (CFQ-R) Respiratory Domain Score
Time Frame: From Baseline Through Week 24
The CFQ-R is a validated participant-reported outcome measuring health-related quality of life for participants with cystic fibrosis. Respiratory domain assessed respiratory symptoms, score range: 0-100; higher scores indicating fewer symptoms and better health-related quality of life.
From Baseline Through Week 24

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Absolute Change in Sweat Chloride (SwCl)
Time Frame: From Baseline Through Week 24
Sweat samples were collected using an approved collection device.
From Baseline Through Week 24
Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1)
Time Frame: From Baseline Through Week 24
FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.
From Baseline Through Week 24
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 in the Treatment Period up to 28 Days After Last Dose of Study Drug or to the Completion of Study Participation Date, Whichever Occurs First (up to Week 28)
From Day 1 in the Treatment Period up to 28 Days After Last Dose of Study Drug or to the Completion of Study Participation Date, Whichever Occurs First (up to Week 28)

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)

October 3, 2019

Primary Completion (Actual)

July 24, 2020

Study Completion (Actual)

July 24, 2020

Study Registration Dates

First Submitted

September 24, 2019

First Submitted That Met QC Criteria

September 24, 2019

First Posted (Actual)

September 26, 2019

Study Record Updates

Last Update Posted (Actual)

August 18, 2021

Last Update Submitted That Met QC Criteria

July 23, 2021

Last Verified

July 1, 2021

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • VX18-445-109
  • 2019-001735-31 (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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