A Study Evaluating the Long-term Safety and Efficacy of Elexacaftor/Tezacaftor/Ivacaftor in Cystic Fibrosis (CF) Subjects 6 Years and Older and F/MF Genotypes

April 21, 2023 updated by: Vertex Pharmaceuticals Incorporated

A Phase 3b Open-label Study Evaluating the Long-term Safety and Efficacy of Elexacaftor/Tezacaftor/Ivacaftor Combination Therapy in Cystic Fibrosis Subjects Ages 6 Years and Older Who Are Heterozygous for the F508del Mutation and a Minimal Function Mutation (F/MF)

This study will evaluate the long-term safety and efficacy of elexacaftor (ELX)/tezacaftor (TEZ)/ivacaftor (IVA) triple combination (TC) in subjects with CF who are 6 years of age and older with F/MF genotypes.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

120

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
      • Nedlands, Australia
        • Telethon Kids Institute
      • South Brisbane, Australia
        • Queensland Children's Hospital
      • Westmead, Australia
        • The Children's Hospital at Westmead
  • Canada
      • Montreal, Canada
        • McGill University Health Centre, Glen Site, Montreal Children's Hospital
      • Toronto, Canada
        • The Hospital for Sick Children
      • Vancouver, Canada
        • British Columbia Children's Hospital
  • Denmark
      • Copenhagen, Denmark
        • Juliane Marie Center, Rigshospitalet
  • France
      • Bordeaux cedex, France
        • Groupe Hospitaler Pellegrin, CHU De Bordeaux
      • Bron Cedex, France
        • CHU Lyon - Hopital Femme Mere-Enfant
      • Paris, France
        • Hôpital Robert Debré
      • Paris Cedex 15, France
        • Hopital Necker, Enfants Malades
      • Roscoff cedex, France
        • Centre de Perharidy
  • Germany
      • Berlin, Germany
        • Charite Paediatric Pulmonology Department
      • Essen, Germany
        • Universitatsklinikum Essen (AoR), Kinderklinik III, Abt. fur Pneumologie
      • Frankfurt, Germany
        • Johann Wolfgang Goethe University
      • Gießen, Germany
        • Justus-Liebig-Universität Gießen Zentrum für Kinderheilkunde und Jugendmedizin
      • Hannover, Germany
        • Medizinische Hochschule Hannover
      • Heidelberg, Germany
        • Universitaetsklinikum Heidelberg, Zenter fuer Kinder-und Jugendmedizin
      • Koeln, Germany
        • Universitaetsklinkum Koeln, CF-Studienzentrum
  • Israel
      • Jerusalem, Israel
        • Hadassah University Hospital Mount Scopus
      • Petach Tikvah, Israel
        • Schneider Children's Medical Center of Israel
  • Netherlands
      • Groningen, Netherlands
        • Universitair Medisch Centrum Groningen
      • Rotterdam, Netherlands
        • Erasmus Medical Center / Sophia Children's Hospital
  • Spain
      • Barcelona, Spain
        • Hospital Universitari Vall d Hebron
      • Murcia, Spain
        • Hospital Virgen de la Arrixaca
  • Switzerland
      • Bern, Switzerland
        • Inselspital - Universitaetsspital Bern
      • Zurich, Switzerland
        • Kinderspital Zuerich
  • United Kingdom
      • Bristol, United Kingdom
        • University Hospitals Bristol and Weston NHS Foundation Trust, Bristol Royal Hospital
      • Cardiff, United Kingdom
        • Children's Hospital of Wales
      • Edinburgh, United Kingdom
        • Royal Hospital for Sick Children
      • Liverpool, United Kingdom
        • Alder Hey Children's NHS Foundation Trust
      • London, United Kingdom
        • Royal Brompton & Harefield NHS Foundation Trust, Royal Brompton Hospital
      • London, United Kingdom
        • Great Ormond Street Hospital for Sick Children
      • 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

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

Accepts Healthy Volunteers

No

Description

Key Inclusion Criteria:

  • Completed study drug treatment in parent study (VX19-445-116, NCT04353817), 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 the 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
Participants will receive ELX/TEZ/IVA in the morning and IVA in the evening.
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
Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
Time Frame: Day 1 up to Week 100
Day 1 up to Week 100

Secondary Outcome Measures

Outcome Measure
Time Frame
Absolute Change in Sweat Chloride (SwCl)
Time Frame: From Baseline up to Week 96
From Baseline up to Week 96
Absolute Change in Lung Clearance Index 2.5 (LCI 2.5)
Time Frame: From Baseline up to Week 96
From Baseline up to Week 96

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)

January 11, 2021

Primary Completion (Actual)

March 24, 2023

Study Completion (Actual)

March 24, 2023

Study Registration Dates

First Submitted

September 3, 2020

First Submitted That Met QC Criteria

September 3, 2020

First Posted (Actual)

September 11, 2020

Study Record Updates

Last Update Posted (Actual)

April 24, 2023

Last Update Submitted That Met QC Criteria

April 21, 2023

Last Verified

April 1, 2023

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • VX20-445-119
  • 2020-001404-42 (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

product manufactured in and exported from the U.S.

Yes

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.

Clinical Trials on Cystic Fibrosis

Clinical Trials on IVA

Search Similar Trials

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Norway

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations

3
Subscribe