Evaluation of Long-term Safety and Efficacy of ELX/TEZ/IVA TC Combination Therapy in Participants With Cystic Fibrosis Who Are 6 Years of Age and Older

May 1, 2025 updated by: Vertex Pharmaceuticals Incorporated

A Phase 3, Open-label Study Evaluating the Long-term Safety and Efficacy of ELX/TEZ/IVA Combination Therapy in Subjects With Cystic Fibrosis Who Are 6 Years of Age and Older

The study evaluates the long-term safety, tolerability, efficacy, and pharmacodynamics of elexacaftor (ELX, VX-445) in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in participants with cystic fibrosis (CF).

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

64

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
      • South Brisbane, Australia
        • Queensland Children's Hospital
      • Westmead, Australia
        • The Children's Hospital at Westmead
  • Canada
      • Toronto, Canada
        • The Hospital for Sick Children
      • Vancouver, Canada
        • British Columbia's Children's Hospital
  • Ireland
      • Dublin, Ireland
        • Children's Health Ireland at Crumlin
      • Dublin, Ireland
        • Children's Health Ireland at Temple Street
  • United Kingdom
      • Birmingham, United Kingdom
        • Birmingham Children's Hospital - NHS Foundation Trust
      • London, United Kingdom
        • Royal Brompton & Harefield NHS Foundation Trust, Royal Brompton Hospital
  • United States
    • California
      • Orange, California, United States, 92868
        • Children's Hospital of Orange County
    • Colorado
      • Aurora, Colorado, United States, 80045
        • Children's Hospital Colorado
    • Illinois
      • Chicago, Illinois, United States, 60611
        • Ann & Robert Lurie Children's Hospital of Chicago
    • 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
    • New York
      • New Hyde Park, New York, United States, 11040
        • Northwell Health- Long Island Jewish Medical Center
    • North Carolina
      • Charlotte, North Carolina, United States, 28277
        • Clinical Research of Charlotte
    • Ohio
      • Cleveland, Ohio, United States, 44106
        • Rainbow Babies and Children's Hospital/University Hospitals Cleveland Medical Center
      • Columbus, Ohio, United States, 43205
        • Nationwide Children's Hospital
    • Oregon
      • Portland, Oregon, United States, 97239
        • Oregon Health & Science University
    • Texas
      • Houston, Texas, United States, 77030
        • Texas Children's Hospital
    • 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

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

Accepts Healthy Volunteers

No

Description

Key Inclusion Criteria:

  • Completed study drug treatment in parent study (VX18-445-106 Part B, NCT03691779), 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
Participants greater than or equal to (≥) 6 years and less than (<) 12 years of age and weighing <30 kilograms (kg) received ELX (elexacaftor) 100 milligram (mg) once daily (qd) /TEZ (tezacaftor) 50 mg qd/IVA (ivacaftor) 75 mg every 12 hours (q12h) and those weighing (≥) 30 kg received ELX 200 mg qd/TEZ 100 mg qd/IVA 150 mg in the treatment period for up to 192 weeks. Participants ≥12 years of age received ELX 200 mg qd/ TEZ 100 mg qd/IVA 150 mg q12h in the treatment period for up to 192 weeks.
Drug: ELX/TEZ/IVA
Fixed-dose combination (FDC) tablet for oral administration
Other Names:
  • VX-445/VX-661/VX-770
  • elexacaftor/tezacaftor/ivacaftor
Drug: IVA
Mono tablet 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 Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
Time Frame: From Baseline up to Week 196
From Baseline up to Week 196

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1)
Time Frame: From Baseline up to Week 192
FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.
From Baseline up to Week 192
Absolute Change in Sweat Chloride (SwCl)
Time Frame: From Baseline up to Week 192
Sweat samples were collected using an approved collection device.
From Baseline up to Week 192
Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score
Time Frame: From Baseline up to Week 192
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 up to Week 192
Absolute Change in Body Mass Index (BMI)
Time Frame: From Baseline up to Week 192
BMI was defined as weight in kg divided by squared height in meters (m^2).
From Baseline up to Week 192
Absolute Change in BMI-for-age Z-score
Time Frame: From Baseline up to Week 192
BMI was defined as weight in kg divided by squared height in meters (m^2). The z-score is a statistical measure to describe whether a mean was above or below the standard. A z-score of 0 is equal to the mean and is considered normal. Lower numbers indicate values lower than the mean and higher numbers indicate values higher than the mean.
From Baseline up to Week 192
Number of Participants With Pulmonary Exacerbations (PEx) for 106/107
Time Frame: From Baseline up to Week 192
Pulmonary exacerbation was defined as the treatment with new or changed antibiotic therapy (intravenous, inhaled, or oral) for greater than or equal to 4 sinopulmonary signs/symptoms.
From Baseline up to Week 192
Number of CF-related Hospitalizations for 106/107
Time Frame: From Baseline up to Week 192
The total number of CF related hospitalization (Planned + Unplanned) events across all participants were reported.
From Baseline up to Week 192
Absolute Change in Lung Clearance Index 2.5 (LCI 2.5)
Time Frame: From Baseline up to Week 192
The LCI2.5 index is the number of lung turnovers required to reduce the end tidal inert gas concentration to 1/40th of its starting values and is calculated by dividing the sum of exhaled tidal breaths (cumulative exhaled volume (CEV)) by simultaneously measured functional residual capacity (FRC). An LCI of 7.5 and below is normal; values greater than 7.5 are abnormal. LCI is able to detect abnormalities in lung function earlier than more traditional modalities such as spirometry.
From Baseline up to Week 192
Absolute Change in Weight
Time Frame: From Baseline up to Week 192
From Baseline up to Week 192
Absolute Change in Weight-for-age Z-score
Time Frame: From Baseline up to Week 192
The z-score is a statistical measure to describe whether a mean was above or below the standard. A z-score of 0 is equal to the mean and is considered normal. Lower numbers indicate values lower than the mean and higher numbers indicate values higher than the mean.
From Baseline up to Week 192
Absolute Change in Height
Time Frame: From Baseline up to Week 192
From Baseline up to Week 192
Absolute Change in Height-for-age Z-score
Time Frame: From Baseline up to Week 192
The z-score is a statistical measure to describe whether a mean was above or below the standard. A z-score of 0 is equal to the mean and is considered normal. Lower numbers indicate values lower than the mean and higher numbers indicate values higher than the mean.
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

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)

February 17, 2020

Primary Completion (Actual)

February 24, 2024

Study Completion (Actual)

February 24, 2024

Study Registration Dates

First Submitted

November 28, 2019

First Submitted That Met QC Criteria

November 28, 2019

First Posted (Actual)

December 3, 2019

Study Record Updates

Last Update Posted (Actual)

May 18, 2025

Last Update Submitted That Met QC Criteria

May 1, 2025

Last Verified

May 1, 2025

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • VX19-445-107
  • 2019-001827-11 (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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