A Study Evaluating the Long-term Safety and Efficacy of VX-445 Combination Therapy

February 7, 2023 updated by: Vertex Pharmaceuticals Incorporated

A Phase 3, Open-label Study Evaluating the Long-term Safety and Efficacy of VX-445 Combination Therapy in Subjects With Cystic Fibrosis Who Are Homozygous or Heterozygous for the F508del Mutation

This study will evaluate the long-term safety and tolerability of VX-445 in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in subjects with cystic fibrosis (CF) who are homozygous or heterozygous for the F508del mutation

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

507

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
      • North Adelaide, Australia
        • Women & Children's Hospital
      • Parkville, Australia
        • The Royal Children's Hospital
      • South Brisbane, Australia
        • Mater Misericordiae Ltd
      • Westmead, Australia
        • The Children's Hospital at Westmead
      • Westmead, Australia
        • Westmead Hospital
  • Austria
      • Graz, Austria
        • University of Graz
      • Innsbruck, Austria
        • Medizinische Universität Innsbruck
      • Salzburg, Austria
        • LKH - Universitätsklinikum der PMU Salzburg
      • Vienna, Austria
        • Medizinische Universität Wien
  • Belgium
      • Brussels, Belgium
        • Universitair Ziekenhuis Brussel - Campus Jette
      • Brussels, Belgium
        • Cliniques Universitaires de Bruxelles Hopital Erasme
      • Edegem, Belgium
        • UZ Antwerpen
      • Gent, Belgium
        • Universitair Ziekenhuis Gent
      • Leuven, Belgium
        • Universitaire Ziekenhuizen Leuven - Campus Gasthuisberg
  • Canada
      • Calgary, Canada
        • University of Calgary Medical Clinic of the Foothills Medical Centre
      • Montreal, Canada
        • McGill University Health Centre, Glen Site, Montreal Children's Hospital
      • Quebec City, Canada
        • Centre Hospitalier de l'Universite Laval
      • Toronto, Canada
        • The Hospital for Sick Children
      • Vancouver, Canada
        • British Columbia's Children's Hospital
      • Vancouver, Canada
        • St. Paul's Hospital
  • Czechia
      • Brno, Czechia
        • Fakultní Nemocnice Brno
      • Praha 5, Czechia
        • Fakultni nemocnice v Motole
  • France
      • Benite Cedex, France
        • Centre Hospitalier Lyon Sud
      • Bordeaux cedex, France
        • Groupe Hospitaler Pellegrin, CHU De Bordeaux
      • Marseille, France
        • CHU Marseille - Hopital Nord
      • Nice, France
        • CHU de Nice - Hopital Pasteur
      • Paris, France
        • Hôpital Cochin
      • Rouen, France
        • CHU de Rouen - Hôpital Charles Nicolle
      • Suresnes, France
        • Hopital Foch (Suresnes), Hopital Foch, Adultes
  • Germany
      • Erlangen, Germany
        • Friedrich-Alexander University of Erlangen-Nuremberg, University Children's Hospital
      • Giessen, Germany
        • Justus-Leibig-Universitat Zentrum fur Kinderheilkunde und Jugendmedizin
      • Hannover, Germany
        • Hannover Medical School
      • Mainz, Germany
        • Johannes Gutenberg-Universitaet
      • München, Germany
        • Dr. Von Haunersches Kinderspital
      • Tuebingen, Germany
        • Universitaetsklinikum Tuebingen Klinik fuer Kinder- und Jugendmedizin
      • Wurzburg, Germany
        • University Hospital Wuerzburg
  • Greece
      • Maroussi, Greece
        • General Hospital of Attika "Sismanoglio"(Adult CF center, NHS)
  • Italy
      • Ancona, Italy
        • Azienda Ospedaliero Universitaria Ospendali Riuniti
      • Firenze, Italy
        • Azienda Ospedaliero Universitaria Ospedale Pediatrico Meyer
      • Genova, Italy
        • IRCCS Istituto Giannina Gaslini-Ospedale Pediatrico
      • Messina, Italy
        • Azienda Ospedaliera Universitaria Policlinico G. Martino
      • Milano, Italy
        • Fondazione Irccs Ca' Granda Ospedale Maggiore Policlinico
      • Potenza, Italy
        • Centro Regionale Fibrosi Cistica, A.O. Ospedale San Carlo
      • Verona, Italy
        • Azienda Ospedaliera di Verona-Ospedale Civile Maggiore
  • Netherlands
      • Amsterdam, Netherlands
        • Academic Medical Center
      • Den Haag, Netherlands
        • HagaZiekenhuis van Den Haag
      • Heidelberglaan, Netherlands
        • University Medical Center, Utrecht, Department of Pulmonology and Tuberculosis
      • Nijmegen, Netherlands
        • UMC St. Radboud
      • Rotterdam, Netherlands
        • Erasmus Medical Center
  • Sweden
      • Stockholm, Sweden
        • Karolinska Universitetssjukhuset, Huddinge
  • United Kingdom
      • Belfast, United Kingdom
        • Belfast City Hospital
      • Birmingham, United Kingdom
        • Heart of England NHS Foundation Trust, Birmingham Heartlands Hospital
      • Edinburgh, United Kingdom
        • Western General Hospital
      • Exeter, United Kingdom
        • Royal Devon and Exeter NHS Foundation Trust, Royal Devon and Exeter Hospital
      • Leeds, United Kingdom
        • Leeds General Infirmary
      • London, United Kingdom
        • King's College Hospital
      • Manchester, United Kingdom
        • Royal Manchester Children's Hospital
      • Southampton, United Kingdom
        • Southampton General Hospital
  • United States
    • Arizona
      • Tucson, Arizona, United States, 85724
        • Banner University of Arizona Medical Center
    • Arkansas
      • Little Rock, Arkansas, United States, 72205
        • University of Arkansas for Medical Sciences
    • California
      • Long Beach, California, United States, 90806
        • Miller Children's Hospital / Long Beach Memorial
      • Los Angeles, California, United States, 90027
        • Children's Hospital Los Angeles
      • Madera, California, United States, 93636
        • Valley Children's Hospital
      • Oakland, California, United States, 94611
        • Kaiser Permanente
      • Sacramento, California, United States, 95817
        • University of California Davis Medical Center
      • San Francisco, California, United States, 94143
        • UCSF Gateway Medical Center
    • Colorado
      • Denver, Colorado, United States, 80206
        • National Jewish Health
    • Florida
      • Gainesville, Florida, United States, 32610
        • University of Florida, Shands Hospital
      • Hollywood, Florida, United States, 33021
        • Joe DiMaggio Cystic Fibrosis & Pulmonary Center
      • Jacksonville, Florida, United States, 32207
        • Nemours Children's Specialty Care
      • Orlando, Florida, United States, 32827
        • Nemours Children's Hospital
      • Orlando, Florida, United States, 32803
        • Central Florida Pulmonary Group, PA
      • Tampa, Florida, United States, 33606
        • Tampa General Hospital Cardiac and Lung Transplant Clinic
    • Georgia
      • Atlanta, Georgia, United States, 30324
        • Children's Specialty Services at North Druid Hills
      • Augusta, Georgia, United States, 30912
        • Augusta University
    • Illinois
      • Chicago, Illinois, United States, 60611
        • Northwestern Memorial Hospital
      • Chicago, Illinois, United States, 60611
        • Ann & Robert Lurie Children's Hospital of Chicago
      • Peoria, Illinois, United States, 61637
        • Cystic Fibrosis Center, Children's Hospital of Illinois at OSF Saint Francis Medical Center
    • Kansas
      • Kansas City, Kansas, United States, 66160
        • The University of Kansas Medical Center
    • Louisiana
      • New Orleans, Louisiana, United States, 70112
        • Tulane Medical Center
    • Maine
      • Portland, Maine, United States, 04102
        • Maine Medical Partners
    • Massachusetts
      • Boston, Massachusetts, United States, 02114
        • Massachusetts General Hospital Cystic Fibrosis Center Clinical Research Center
    • Michigan
      • Detroit, Michigan, United States, 48201
        • Harper University Hospital
    • Minnesota
      • Minneapolis, Minnesota, United States, 55404
        • Children's Respiratory and Critical Care Specialists, P.A., Children's Hospitals and Clinics of Minnesota
    • Montana
      • Billings, Montana, United States, 59101
        • Billings Clinic Hospital
    • Nebraska
      • Omaha, Nebraska, United States, 68198
        • Nebraska Medical Center
    • New Jersey
      • Morristown, New Jersey, United States, 07960
        • Morristown Medical Center
    • New Mexico
      • Albuquerque, New Mexico, United States, 87131
        • UNM Clinical and Translational Science Center
    • New York
      • New York, New York, United States, 10003
        • The Cystic Fibrosis Center, Mount Sinai Beth Israel
      • Valhalla, New York, United States, 10595
        • New York Medical College
    • North Carolina
      • Chapel Hill, North Carolina, United States, 27517
        • UNC Marsico Clinical Research Center
    • Ohio
      • Akron, Ohio, United States, 44308
        • Akron Children's Hospital
      • Cincinnati, Ohio, United States, 45220
        • UC Health Holmes
      • 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
      • Dayton, Ohio, United States, 45404
        • Dayton Children's Hospital
      • Toledo, Ohio, United States, 43606
        • ProMedica Toledo Hospital/Toledo Children's Hospital/Pediatric Pulmonary & Cystic Fibrosis Center
    • Oklahoma
      • Oklahoma City, Oklahoma, United States, 73104
        • University of Oklahoma Health Sciences Center
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19104
        • Children's Hospital of Philadelphia
    • South Carolina
      • Charleston, South Carolina, United States, 29425
        • Medical University of South Carolina
    • Texas
      • Austin, Texas, United States, 78723
        • Dell Children's Medical Center of Central Texas
      • Dallas, Texas, United States, 75390
        • The University of Texas Southwestern Medical Center
    • Vermont
      • Colchester, Vermont, United States, 05446
        • Vermont Lung Center
    • Virginia
      • Charlottesville, Virginia, United States, 22908
        • University of Virginia Primary Care Center
      • Norfolk, Virginia, United States, 23507
        • Children's Hospital of The King's Daughters
      • Richmond, Virginia, United States, 23298
        • Children's Hospital of Richmond at VCU, Children's Pavilion
    • Washington
      • Seattle, Washington, United States, 98195
        • University of Washington Medical Center
    • West Virginia
      • Morgantown, West Virginia, United States, 26506
        • West Virginia University
    • Wisconsin
      • Madison, Wisconsin, United States, 53792
        • University of Wisconsin Hospitals and Clinics
      • Milwaukee, Wisconsin, United States, 53226
        • CTSI Adult Translational Research Unit/Medical College of Wisconsin/Froedtert 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 (ADULT, OLDER_ADULT, CHILD)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Inclusion Criteria:

  • Completed study drug treatment in a parent study; or had study drug interruption(s) in a parent study but completed study visits up to the last scheduled visit of the Treatment Period in the parent study.

Exclusion Criteria:

  • History of drug intolerance in a parent study that would pose an additional risk to the subject in the opinion of the investigator.
  • Current participation in an investigational drug trial (other than a 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: NA
  • Interventional Model: SINGLE_GROUP
  • Masking: NONE

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
EXPERIMENTAL: Open-label Triple Combination

Subjects will receive 200 mg VX-445/ 100 mg TEZ/ 150 mg IVA as FDC tablets in the morning and 150 mg IVA as mono tablet in the evening.

Parent studies are Phase 3 Vertex studies investigating VX-445 in combination with TEZ and IVA. This includes Studies VX17-445-102 and VX17-445-103.
Drug: VX-445
Fixed-dose combination (FDC) tablets (VX-445/TEZ/IVA)
Drug: TEZ
FDC tablets (VX-445/TEZ/IVA)
Other Names:
  • tezacaftor; VX-661
Drug: IVA
FDC tablets (VX-445/TEZ/IVA)
Other Names:
  • ivacaftor; VX-770
Drug: IVA
IVA tablet
Other Names:
  • ivacaftor; VX-770

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Safety and tolerability as assessed by number of subjects with adverse events (AEs) and serious adverse events (SAEs)
Time Frame: from baseline through safety follow-up (up to 196 weeks)
from baseline through safety follow-up (up to 196 weeks)

Secondary Outcome Measures

Outcome Measure
Time Frame
Absolute change from baseline in percent predicted forced expiratory volume in 1 second (ppFEV1)
Time Frame: from baseline through last dose of study drug (up to 192 weeks)
from baseline through last dose of study drug (up to 192 weeks)
Absolute change in sweat chloride (SwCl)
Time Frame: from baseline through last dose of study drug (up to 192 weeks)
from baseline through last dose of study drug (up to 192 weeks)
Number of pulmonary exacerbations (PEx)
Time Frame: from baseline through last dose of study drug (up to 192 weeks)
from baseline through last dose of study drug (up to 192 weeks)
Time to first PEx
Time Frame: from baseline through last dose of study drug (up to 192 weeks)
from baseline through last dose of study drug (up to 192 weeks)
Absolute change in body mass index (BMI)
Time Frame: from baseline through last dose of study drug (up to 192 weeks)
from baseline through last dose of study drug (up to 192 weeks)
Absolute change in BMI z-score
Time Frame: from baseline through last dose of study drug (up to 192 weeks)
from baseline through last dose of study drug (up to 192 weeks)
Absolute change in body weight
Time Frame: from baseline through last dose of study drug (up to 192 weeks)
from baseline through last dose of study drug (up to 192 weeks)
Absolute change from baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) respiratory domain score
Time Frame: from baseline through last dose of study drug (up to 192 weeks)
from baseline through last dose of study drug (up to 192 weeks)

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 9, 2018

Primary Completion (ACTUAL)

January 9, 2023

Study Completion (ACTUAL)

January 9, 2023

Study Registration Dates

First Submitted

May 2, 2018

First Submitted That Met QC Criteria

May 2, 2018

First Posted (ACTUAL)

May 15, 2018

Study Record Updates

Last Update Posted (ACTUAL)

February 8, 2023

Last Update Submitted That Met QC Criteria

February 7, 2023

Last Verified

February 1, 2023

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • VX17-445-105
  • 2018-000185-11 (EUDRACT_NUMBER)

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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