Evaluation of Long-term Safety and Efficacy of ELX/TEZ/IVA in Cystic Fibrosis (CF) Participants 2 Years and Older

April 25, 2024 updated by: Vertex Pharmaceuticals Incorporated

A Phase 3 Open-label Study Evaluating the Long-term Safety and Efficacy of Elexacaftor/Tezacaftor/Ivacaftor Triple Combination Therapy in Cystic Fibrosis Subjects 2 Years and Older

The purpose of this study is to evaluate the long term safety, tolerability, efficacy and pharmacodynamics of elexacaftor (ELX)/tezacaftor (TEZ)/ivacaftor (IVA) in CF participants 2 years of age and older.

Study Overview

Status

Active, not recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

71

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
      • Parkville, Australia
        • The Royal Children's Hospital
      • South Brisbane, Australia
        • Queensland Children's Hospital
  • Canada
      • Toronto, Canada
        • The Hospital for Sick Children
      • Vancouver, Canada
        • British Columbia Children's Hospital
  • Germany
      • Berlin, Germany
        • Charite Paediatric Pulmonology Department
      • Essen, Germany
        • Universitatsklinikum Essen (AoR), Kinderklinik III, Abt. fur Pneumologie
  • United Kingdom
      • Liverpool, United Kingdom
        • Alder Hey Children's NHS Foundation Trust
      • London, United Kingdom
        • Royal Brompton Hospital
  • United States
    • Arizona
      • Tucson, Arizona, United States, 85724
        • Banner University of Arizona Medical Center
    • California
      • Palo Alto, California, United States, 94304
        • Stanford University
    • Colorado
      • Aurora, Colorado, United States, 80045
        • Children's Hospital of Colorado
    • Illinois
      • Chicago, Illinois, United States, 60611
        • Ann & Robert H. Lurie Children's Hospital of Chicago
    • Indiana
      • Indianapolis, Indiana, United States, 46202
        • Riley Hospital for Children at Indiana University Health
    • Massachusetts
      • Boston, Massachusetts, United States, 02115
        • Boston Children's Hospital
    • Minnesota
      • Minneapolis, Minnesota, United States, 55404
        • Children's Respiratory and Critical Care Specialists, P.A., Children's Hospitals and Clinics of Minnesota
    • Missouri
      • Kansas City, Missouri, United States, 64108
        • The Children's Mercy Hospital
      • Saint Louis, Missouri, United States, 63110
        • Washington University School of Medicine / St. Louis Children's Hospital
    • North Carolina
      • Chapel Hill, North Carolina, United States, 27599
        • NC TraCS Institute - CTRC University of North Carolina at Chapel Hill
    • Oregon
      • Portland, Oregon, United States, 97239
        • Oregon Health & Science University
    • Pennsylvania
      • Pittsburgh, Pennsylvania, United States, 15224
        • UPMC Children's Hospital of Pittsburgh
    • Washington
      • Seattle, Washington, United States, 98105
        • Seattle Children's 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

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

Accepts Healthy Volunteers

No

Description

key Inclusion Criteria:

  • Completed study drug treatment in the parent study (VX20-445-111 Part B, NCT04537793), or had study drug interruption(s) in the parent study, but did not permanently discontinue study drug, and completed study visits up to the last scheduled visit of the Treatment Period of the parent study

Key Exclusion Criteria:

  • History of study drug intolerance in the parent study
  • History of poor compliance with ELX/TEZ/IVA and/or procedures 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: ELX/TEZ/IVA
Fixed-dose combination granules or tablets for oral administration.
Other Names:
  • VX-445/VX-661/VX-770
  • elexacaftor/tezacaftor/ivacaftor
Drug: IVA
Granules or tablets for oral administration.
Other Names:
  • VX-770
  • 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: From Day 1 up to Week 196
From Day 1 up to Week 196

Secondary Outcome Measures

Outcome Measure
Time Frame
Absolute Change in Sweat Chloride (SwCl)
Time Frame: From Baseline up to Week 192
From Baseline up to Week 192
Absolute Change in Lung Clearance Index (LCI) 2.5
Time Frame: From Baseline up to Week 192
From Baseline up to Week 192

Collaborators and Investigators

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

Sponsor

Vertex Pharmaceuticals Incorporated

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 17, 2022

Primary Completion (Estimated)

April 1, 2026

Study Completion (Estimated)

April 1, 2026

Study Registration Dates

First Submitted

November 29, 2021

First Submitted That Met QC Criteria

November 29, 2021

First Posted (Actual)

December 10, 2021

Study Record Updates

Last Update Posted (Actual)

April 26, 2024

Last Update Submitted That Met QC Criteria

April 25, 2024

Last Verified

March 1, 2024

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • VX20-445-112
  • 2020-002239-31 (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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