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

June 14, 2023 updated by: Vertex Pharmaceuticals Incorporated

A Phase 3, Open-label Study Evaluating the Long-term Safety of VX-445 Combination Therapy in Subjects With Cystic Fibrosis

This study evaluated the long-term safety and tolerability of elexacaftor (ELX), tezacaftor (TEZ), and ivacaftor (IVA) triple combination (TC) treatment in participants with cystic fibrosis (CF).

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

458

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
      • Adelaide, Australia
        • Royal Adelaide Hospital
      • Chermside, Australia
        • The Prince Charles Hospital
      • Melbourne, VIC, Australia
        • Alfred 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
      • South Brisbane, Australia
        • Queensland Children's Hospital
  • Canada
      • Edmonton, Canada
        • Stollery Children's Hospital
      • Halifax, Canada
        • Queen Elizabeth II Health Sciences Center
      • Toronto, Canada
        • St. Michael's Hospital
  • Denmark
      • Copenhagen, Denmark
        • Juliane Marie Center, Rigshospitalet
  • Germany
      • Berlin, Germany
        • Charite Paediatric Pulmonology Department
      • Essen, Germany
        • Ruhrlandklinik Westdeutsches Lungenzentrum am Klinikum Essen
      • Frankfurt, Germany
        • Johann Wolfgang Goethe University
      • Hannover, Germany
        • Hannover Medical School
      • Jena, Germany
        • Universitaetsklinikum Jena, Mukoviszidose-Zentrum
      • Koeln, Germany
        • Universitaetsklinkum Koeln, CF-Studienzentrum
      • Lübeck, Germany
        • Universitatsklinikum Schleswig-Holstein, Klinik für Kinder- und Jugendmedizin
      • Muenchen, Germany
        • Pneumologisches Studienzentrum Muenchen-West
      • München, Germany
        • Klinikum Innenstadt, University of Munich
  • Ireland
      • Cork, Ireland
        • Cork University Hospital
      • Dublin, Ireland
        • Beaumont Hospital
      • Dublin, Ireland
        • Children's Health Ireland at Crumlin
      • Dublin, Ireland
        • Children's Health Ireland at Temple Street
      • Dublin, Ireland
        • St. Vincent's University Hospital
      • Galway, Ireland
        • University Hospital Galway
      • Limerick, Ireland
        • University Hospital Limerick (Adults)
  • Israel
      • Haifa, Israel
        • Lady Davis Carmel Medical Center
      • Haifa, Israel
        • Pediatric Pulmonary Unit Rambam Medical Center
      • Jerusalem, Israel
        • Hadassah Medical Organization
      • Petah Tikva, Israel
        • Schneider Children's Medical Center
      • Tel Hashomer, Israel
        • Sheba Medical Center
  • Poland
      • Łomianki, Poland
        • Klinika Mukowiscydozy IMD Oddozial Chorob Pluc Szpzoz IM. Dzieci WarszaWY
  • Spain
      • Barcelona, Spain
        • Hospital Universitari Vall d Hebron
      • Barcelona, Spain
        • Hospital Universitari Vall d´Hebron Servicio de Broncoscopia
      • Madrid, Spain
        • Hospital Universitario 12 De Octubre
      • Madrid, Spain
        • Hospital Universitario Infantil la Paz
      • Sabadell, Spain
        • Parc Taulí Sabadell Hospital Universitari
      • Sevilla, Spain
        • Hospital Universitario Virgen del Rocío
      • Valencia, Spain
        • Hospital Universitario Y Politecnico La Fe
  • Switzerland
      • Bern, Switzerland
        • Lindenhofspital - Quartier Bleu
      • Zürich, Switzerland
        • Kinderspital Zuerich
  • United Kingdom
      • Cambridge, United Kingdom
        • Papworth Hospital NHS Foundation Trust, Papworth Everard
      • Glasgow, United Kingdom
        • NHS Greater Glasgow and Clyde, Gartnavel General Hospital
      • Leeds, United Kingdom
        • St. James University Hospital
      • Liverpool, United Kingdom
        • Liverpool Heart and Chest Hospital
      • London, United Kingdom
        • Royal Brompton & Harefield NHS Foundation Trust, Royal Brompton Hospital
      • Manchester, United Kingdom
        • Wythenshawe Hospital
      • 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
  • United States
    • Alabama
      • Birmingham, Alabama, United States, 35233
        • University of Alabama at Birmingham
    • California
      • Palo Alto, California, United States, 94304
        • Stanford University
    • Colorado
      • Aurora, Colorado, United States, 80045
        • Children's Hospital Colorado
    • Connecticut
      • Hartford, Connecticut, United States, 06102
        • Hartford Hospital
      • New Haven, Connecticut, United States, 06510
        • Yale New Haven Hospital
    • Florida
      • Miami, Florida, United States, 33136
        • University of Miami Miller School of Medicine
      • Miami, Florida, United States, 33155
        • Nicklaus Children's Hospital
      • Orlando, Florida, United States, 32806
        • Arnold Palmer Hospital Pulmonary and Sleep Medicine
      • Saint Petersburg, Florida, United States, 33701
        • Johns Hopkins All Children's Hospital Outpatient Care Center
    • Idaho
      • Boise, Idaho, United States, 83702
        • St. Luke's CF Center of Idaho
    • Illinois
      • Glenview, Illinois, United States, 60025
        • Cystic Fibrosis Institute
      • Niles, Illinois, United States, 60714
        • Advocate Children's Hospital - Park Ridge / North Suburban Pulmonary and Critical Care Consultants
    • Indiana
      • Indianapolis, Indiana, United States, 46202
        • Indiana University
    • Iowa
      • Iowa City, Iowa, United States, 52242
        • The University of Iowa Hospitals and Clinics
    • Kentucky
      • Lexington, Kentucky, United States, 40536
        • University of Kentucky
      • Louisville, Kentucky, United States, 40202
        • Kosair Charities Pediatric Clinical Research Unit
    • Maryland
      • Baltimore, Maryland, United States, 21287
        • Johns Hopkins Hospital
    • Massachusetts
      • Boston, Massachusetts, United States, 02115
        • Boston Children's Hospital
      • Worcester, Massachusetts, United States, 01655
        • UMass Memorial Medical Center
    • Michigan
      • Ann Arbor, Michigan, United States, 48109-5212
        • Michigan Medicine
    • Minnesota
      • Minneapolis, Minnesota, United States, 55455
        • University of Minnesota
    • Mississippi
      • Jackson, Mississippi, United States, 39216
        • University of Mississippi Medical Center
    • 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
    • New Hampshire
      • Lebanon, New Hampshire, United States, 03756
        • Dartmouth Hitchcock Medical Center
    • New Jersey
      • New Brunswick, New Jersey, United States, 08901
        • Rutgers Robert Wood Johnson Medical School
    • New York
      • Albany, New York, United States, 12208
        • Albany Medical College, Pulmonary and Critical Care Medicine
      • Buffalo, New York, United States, 14222
        • Lung and Cystic Fibrosis Center at Women and Children's Hospital of Buffalo
      • New Hyde Park, New York, United States, 11040
        • Northwell Health- Long Island Jewish Medical Center
      • New York, New York, United States, 10032
        • Columbia University Medical Center
      • Syracuse, New York, United States, 13210
        • SUNY Upstate Medical University
    • North Carolina
      • Charlotte, North Carolina, United States, 28277
        • Clinical Research of Charlotte
      • Durham, North Carolina, United States, 27710
        • Duke University Medical Center
    • Ohio
      • Cincinnati, Ohio, United States, 45229
        • Cincinnati Children's Hospital Medical Center
    • Oklahoma
      • Oklahoma City, Oklahoma, United States, 73112
        • Santiago Reyes, M.D.
    • Oregon
      • Portland, Oregon, United States, 97239
        • Oregon Health & Science University
    • Pennsylvania
      • Pittsburgh, Pennsylvania, United States, 15224
        • Children's Hospital of Pittsburgh of UPMC
    • South Dakota
      • Sioux Falls, South Dakota, United States, 57105
        • Sanford Children's Speciality Clinic
    • Tennessee
      • Knoxville, Tennessee, United States, 37920
        • University of Tennessee Medical Center
      • Memphis, Tennessee, United States, 38103
        • Children's Foundation Research Center / Le Bonheur Children's Hospital
      • Nashville, Tennessee, United States, 37232
        • Vanderbilt University Medical Center
    • Texas
      • Fort Worth, Texas, United States, 76104
        • Cook Children's Medical Center
      • Houston, Texas, United States, 77030
        • Texas Children's Hospital
    • Utah
      • Salt Lake City, Utah, United States, 84132
        • University of Utah / Primary Children's Medical Center
    • Washington
      • Seattle, Washington, United States, 98105
        • Seattle Children's Hospital
      • Spokane, Washington, United States, 99204
        • Providence Pediatric Pulmonary & Cystic Fibrosis Clinic

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 (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  • Currently participating in study VX17-659-105 (NCT03447262)

Exclusion Criteria:

  • History of drug intolerance in study VX17-659-105 that would pose an additional risk to the participant in the opinion of the investigator
  • Current participation in an investigational drug trial (other than study VX17-659-105)

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

Part A: Participants received ELX 200 milligram (mg) once daily (qd),TEZ 100 mg qd, and IVA 150 mg every 12 hours (q12h) in the treatment period for 96 weeks.

Part B: Participants from certain countries participated in Part B and continued to received ELX 200 mg qd /TEZ 100 mg qd/IVA 150 mg q12h in the treatment period for 48 weeks.
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
Part A: Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
Time Frame: From Baseline through Week 100
From Baseline through Week 100

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)

August 9, 2019

Primary Completion (Actual)

December 14, 2022

Study Completion (Actual)

December 14, 2022

Study Registration Dates

First Submitted

July 31, 2019

First Submitted That Met QC Criteria

July 31, 2019

First Posted (Actual)

August 2, 2019

Study Record Updates

Last Update Posted (Actual)

July 6, 2023

Last Update Submitted That Met QC Criteria

June 14, 2023

Last Verified

June 1, 2023

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • VX18-445-113
  • 2018-004652-38 (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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