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

NCT ID: NCT03525574

Last Updated: 2025-09-24

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

View full results

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

507 participants

Study Classification

INTERVENTIONAL

Study Start Date

2018-10-09

Study Completion Date

2023-01-09

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

The study evaluates 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

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Cystic Fibrosis

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

ELX/TEZ/IVA

Treatment Period: Participants received ELX 200 milligram (mg) once daily (qd)/TEZ 100 mg qd/IVA 150 mg every 12 hrs (q12h) in the treatment period for 192 weeks.

Extension Period: Participants from certain countries participated in extension period and continued to receive ELX 200 mg qd /TEZ 100 mg qd/IVA 150 mg q12h in the extension period for 48 weeks.

Group Type EXPERIMENTAL

ELX/TEZ/IVA

Intervention Type DRUG

Fixed-dose combination (FDC) tablet for oral administration.

IVA

Intervention Type DRUG

Tablet for oral administration.

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

ELX/TEZ/IVA

Fixed-dose combination (FDC) tablet for oral administration.

Intervention Type DRUG

IVA

Tablet for oral administration.

Intervention Type DRUG

Other Intervention Names

Discover alternative or legacy names that may be used to describe the listed interventions across different sources.

VX-445/VX-661/VX-770 elexacaftor/tezacaftor/ivacaftor VX-770 ivacaftor

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

Inclusion Criteria

* Completed study drug treatment in a parent study i.e.VX17-445-102 (NCT03525444) and VX17-445-103 (NCT03525548); 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)
Minimum Eligible Age

12 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

Meet the organizations funding or collaborating on the study and learn about their roles.

Vertex Pharmaceuticals Incorporated

INDUSTRY

Sponsor Role lead

Responsible Party

Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.

Responsibility Role SPONSOR

Locations

Explore where the study is taking place and check the recruitment status at each participating site.

Banner University of Arizona Medical Center

Tucson, Arizona, United States

Site Status

University of Arkansas for Medical Sciences

Little Rock, Arkansas, United States

Site Status

Miller Children's Hospital / Long Beach Memorial

Long Beach, California, United States

Site Status

Children's Hospital Los Angeles

Los Angeles, California, United States

Site Status

Valley Children's Hospital

Madera, California, United States

Site Status

Kaiser Permanente

Oakland, California, United States

Site Status

University of California Davis Medical Center

Sacramento, California, United States

Site Status

UCSF Gateway Medical Center

San Francisco, California, United States

Site Status

National Jewish Health

Denver, Colorado, United States

Site Status

University of Florida, Shands Hospital

Gainesville, Florida, United States

Site Status

Joe DiMaggio Cystic Fibrosis & Pulmonary Center

Hollywood, Florida, United States

Site Status

Nemours Children's Specialty Care

Jacksonville, Florida, United States

Site Status

Central Florida Pulmonary Group, PA

Orlando, Florida, United States

Site Status

Nemours Children's Hospital

Orlando, Florida, United States

Site Status

Tampa General Hospital Cardiac and Lung Transplant Clinic

Tampa, Florida, United States

Site Status

Children's Specialty Services at North Druid Hills

Atlanta, Georgia, United States

Site Status

Augusta University

Augusta, Georgia, United States

Site Status

Ann & Robert Lurie Children's Hospital of Chicago

Chicago, Illinois, United States

Site Status

Northwestern Memorial Hospital

Chicago, Illinois, United States

Site Status

Cystic Fibrosis Center, Children's Hospital of Illinois at OSF Saint Francis Medical Center

Peoria, Illinois, United States

Site Status

The University of Kansas Medical Center

Kansas City, Kansas, United States

Site Status

Tulane Medical Center

New Orleans, Louisiana, United States

Site Status

Maine Medical Partners

Portland, Maine, United States

Site Status

Massachusetts General Hospital Cystic Fibrosis Center Clinical Research Center

Boston, Massachusetts, United States

Site Status

Harper University Hospital

Detroit, Michigan, United States

Site Status

Children's Respiratory and Critical Care Specialists, P.A., Children's Hospitals and Clinics of Minnesota

Minneapolis, Minnesota, United States

Site Status

Billings Clinic Hospital

Billings, Montana, United States

Site Status

Nebraska Medical Center

Omaha, Nebraska, United States

Site Status

Morristown Medical Center

Morristown, New Jersey, United States

Site Status

UNM Clinical and Translational Science Center

Albuquerque, New Mexico, United States

Site Status

The Cystic Fibrosis Center, Mount Sinai Beth Israel

New York, New York, United States

Site Status

New York Medical College

Valhalla, New York, United States

Site Status

UNC Marsico Clinical Research Center

Chapel Hill, North Carolina, United States

Site Status

Akron Children's Hospital

Akron, Ohio, United States

Site Status

UC Health Holmes

Cincinnati, Ohio, United States

Site Status

Rainbow Babies and Children's Hospital/University Hospitals Cleveland Medical Center

Cleveland, Ohio, United States

Site Status

Nationwide Children's Hospital

Columbus, Ohio, United States

Site Status

Dayton Children's Hospital

Dayton, Ohio, United States

Site Status

ProMedica Toledo Hospital/Toledo Children's Hospital/Pediatric Pulmonary & Cystic Fibrosis Center

Toledo, Ohio, United States

Site Status

University of Oklahoma Health Sciences Center

Oklahoma City, Oklahoma, United States

Site Status

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, United States

Site Status

Medical University of South Carolina

Charleston, South Carolina, United States

Site Status

Dell Children's Medical Center of Central Texas

Austin, Texas, United States

Site Status

The University of Texas Southwestern Medical Center

Dallas, Texas, United States

Site Status

Vermont Lung Center

Colchester, Vermont, United States

Site Status

University of Virginia Primary Care Center

Charlottesville, Virginia, United States

Site Status

Children's Hospital of the King's Daughters

Norfolk, Virginia, United States

Site Status

Children's Hospital of Richmond at VCU, Children's Pavilion

Richmond, Virginia, United States

Site Status

University of Washington Medical Center

Seattle, Washington, United States

Site Status

West Virginia University

Morgantown, West Virginia, United States

Site Status

University of Wisconsin Hospitals and Clinics

Madison, Wisconsin, United States

Site Status

CTSI Adult Translational Research Unit/Medical College of Wisconsin/Froedtert Hospital

Milwaukee, Wisconsin, United States

Site Status

Women & Children's Hospital

North Adelaide, , Australia

Site Status

The Royal Children's Hospital

Parkville, , Australia

Site Status

Mater Misericordiae Ltd

South Brisbane, , Australia

Site Status

The Children's Hospital at Westmead

Westmead, , Australia

Site Status

Westmead Hospital

Westmead, , Australia

Site Status

University of Graz

Graz, , Austria

Site Status

Medizinische Universität Innsbruck

Innsbruck, , Austria

Site Status

LKH - Universitätsklinikum der PMU Salzburg

Salzburg, , Austria

Site Status

Medizinische Universitat Wien

Vienna, , Austria

Site Status

Cliniques Universitaires de Bruxelles Hopital Erasme

Brussels, , Belgium

Site Status

Universitair Ziekenhuis Brussel - Campus Jette

Brussels, , Belgium

Site Status

UZ Antwerpen

Edegem, , Belgium

Site Status

Universitair Ziekenhuis Gent

Ghent, , Belgium

Site Status

Universitaire Ziekenhuizen Leuven - Campus Gasthuisberg

Leuven, , Belgium

Site Status

University of Calgary Medical Clinic of the Foothills Medical Centre

Calgary, , Canada

Site Status

McGill University Health Centre, Glen Site, Montreal Children's Hospital

Montreal, , Canada

Site Status

Centre Hospitalier De L'Universite Laval

Québec, , Canada

Site Status

The Hospital for Sick Children

Toronto, , Canada

Site Status

British Columbia's Children's Hospital

Vancouver, , Canada

Site Status

St. Paul's Hospital

Vancouver, , Canada

Site Status

Fakultni Nemocnice Brno

Brno, , Czechia

Site Status

Fakultni nemocnice v Motole

Prague, , Czechia

Site Status

Centre Hospitalier Lyon Sud

Benite Cedex, , France

Site Status

Groupe Hospitaler Pellegrin, CHU De Bordeaux

Bordeaux, , France

Site Status

CHU Marseille - Hopital Nord

Marseille, , France

Site Status

CHU de Nice - Hopital Pasteur

Nice, , France

Site Status

Hopital Cochin

Paris, , France

Site Status

CHU de Rouen - Hopital Charles Nicolle

Rouen, , France

Site Status

Hopital Foch (Suresnes), Hopital Foch, Adultes

Suresnes, , France

Site Status

Friedrich-Alexander University of Erlangen-Nuremberg, University Children's Hospital

Erlangen, , Germany

Site Status

Justus-Leibig-Universitat Zentrum fur Kinderheilkunde und Jugendmedizin

Giessen, , Germany

Site Status

Hannover Medical School

Hanover, , Germany

Site Status

Johannes Gutenberg-Universitaet

Mainz, , Germany

Site Status

Dr. von Haunersches Kinderspital

München, , Germany

Site Status

Universitaetsklinikum Tuebingen Klinik fuer Kinder- und Jugendmedizin

Tübingen, , Germany

Site Status

University Hospital Wuerzburg

Würzburg, , Germany

Site Status

General Hospital of Attika "Sismanoglio"(Adult CF center, NHS)

Marousi, , Greece

Site Status

Azienda Ospedaliero Universitaria Ospendali Riuniti

Ancona, , Italy

Site Status

Azienda Ospedaliero Universitaria Ospedale Pediatrico Meyer

Florence, , Italy

Site Status

IRCCS Istituto Giannina Gaslini-Ospedale Pediatrico

Genova, , Italy

Site Status

Azienda Ospedaliera Universitaria Policlinico G. Martino

Messina, , Italy

Site Status

Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico

Milan, , Italy

Site Status

Centro Regionale Fibrosi Cistica, A.O. Ospedale San Carlo

Potenza, , Italy

Site Status

Azienda Ospedaliera di Verona-Ospedale Civile Maggiore

Verona, , Italy

Site Status

Academic Medical Center

Amsterdam, , Netherlands

Site Status

University Medical Center, Utrecht, Department of Pulmonology and Tuberculosis

Heidelberglaan, , Netherlands

Site Status

UMC St. Radboud

Nijmegen, , Netherlands

Site Status

Erasmus Medical Center

Rotterdam, , Netherlands

Site Status

HagaZiekenhuis van den Haag

The Hague, , Netherlands

Site Status

Karolinska Universitetssjukhuset, Huddinge

Stockholm, , Sweden

Site Status

Belfast City Hospital

Belfast, , United Kingdom

Site Status

Heart of England NHS Foundation Trust, Birmingham Heartlands Hospital

Birmingham, , United Kingdom

Site Status

Western General Hospital

Edinburgh, , United Kingdom

Site Status

Royal Devon and Exeter NHS Foundation Trust, Royal Devon and Exeter Hospital

Exeter, , United Kingdom

Site Status

Leeds General Infirmary

Leeds, , United Kingdom

Site Status

King's College Hospital

London, , United Kingdom

Site Status

Royal Manchester Children's Hospital

Manchester, , United Kingdom

Site Status

Southampton General Hospital

Southampton, , United Kingdom

Site Status

Countries

Review the countries where the study has at least one active or historical site.

United States Australia Austria Belgium Canada Czechia France Germany Greece Italy Netherlands Sweden United Kingdom

References

Explore related publications, articles, or registry entries linked to this study.

Daines CL, Polineni D, Tullis E, Costa S, Linnemann RW, Mall MA, McKone EF, Quon BS, Ringshausen FC, Selvadurai H, Taylor-Cousar JL, Withers NJ, Sawicki GS, Lee T, Ahluwalia N, Morlando Geiger J, Jennings M, Tan YV, Waltz D, Ramsey B, Griese M; VX17-445-105 Study Group. Long-Term Safety and Efficacy of Elexacaftor/Tezacaftor/Ivacaftor in Adults and Adolescents with Cystic Fibrosis and at Least One F508del Allele: A Phase 3 Open-Label Extension Study. Am J Respir Crit Care Med. 2025 Oct;211(10):1901-1914. doi: 10.1164/rccm.202411-2231OC.

Reference Type DERIVED
PMID: 40209082 (View on PubMed)

Heneghan M, Southern KW, Murphy J, Sinha IP, Nevitt SJ. Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants (most commonly F508del). Cochrane Database Syst Rev. 2023 Nov 20;11(11):CD010966. doi: 10.1002/14651858.CD010966.pub4.

Reference Type DERIVED
PMID: 37983082 (View on PubMed)

Southern KW, Murphy J, Sinha IP, Nevitt SJ. Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants (most commonly F508del). Cochrane Database Syst Rev. 2020 Dec 17;12(12):CD010966. doi: 10.1002/14651858.CD010966.pub3.

Reference Type DERIVED
PMID: 33331662 (View on PubMed)

Provided Documents

Download supplemental materials such as informed consent forms, study protocols, or participant manuals.

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

Review additional registry numbers or institutional identifiers associated with this trial.

2018-000185-11

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

VX17-445-105

Identifier Type: -

Identifier Source: org_study_id

More Related Trials

Additional clinical trials that may be relevant based on similarity analysis.