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

NCT ID: NCT04043806

Last Updated: 2023-07-06

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

458 participants

Study Classification

INTERVENTIONAL

Study Start Date

2019-08-09

Study Completion Date

2022-12-14

Brief Summary

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

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).

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

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.

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

* 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)
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.

Hartford Hospital

Hartford, Connecticut, United States

Site Status

Yale New Haven Hospital

New Haven, Connecticut, United States

Site Status

University of Alabama at Birmingham

Birmingham, Alabama, United States

Site Status

Stanford University

Palo Alto, California, United States

Site Status

Children's Hospital Colorado

Aurora, Colorado, United States

Site Status

University of Miami Miller School of Medicine

Miami, Florida, United States

Site Status

Nicklaus Children's Hospital

Miami, Florida, United States

Site Status

Arnold Palmer Hospital Pulmonary and Sleep Medicine

Orlando, Florida, United States

Site Status

Johns Hopkins All Children's Hospital Outpatient Care Center

St. Petersburg, Florida, United States

Site Status

St. Luke's CF Center of Idaho

Boise, Idaho, United States

Site Status

Cystic Fibrosis Institute

Glenview, Illinois, United States

Site Status

Advocate Children's Hospital - Park Ridge / North Suburban Pulmonary and Critical Care Consultants

Niles, Illinois, United States

Site Status

Indiana University

Indianapolis, Indiana, United States

Site Status

The University of Iowa Hospitals and Clinics

Iowa City, Iowa, United States

Site Status

University of Kentucky

Lexington, Kentucky, United States

Site Status

Kosair Charities Pediatric Clinical Research Unit

Louisville, Kentucky, United States

Site Status

Johns Hopkins Hospital

Baltimore, Maryland, United States

Site Status

Boston Children's Hospital

Boston, Massachusetts, United States

Site Status

UMass Memorial Medical Center

Worcester, Massachusetts, United States

Site Status

Michigan Medicine

Ann Arbor, Michigan, United States

Site Status

University of Minnesota

Minneapolis, Minnesota, United States

Site Status

University of Mississippi Medical Center

Jackson, Mississippi, United States

Site Status

The Children's Mercy Hospital

Kansas City, Missouri, United States

Site Status

Washington University School of Medicine / St. Louis Children's Hospital

St Louis, Missouri, United States

Site Status

Dartmouth Hitchcock Medical Center

Lebanon, New Hampshire, United States

Site Status

Rutgers Robert Wood Johnson Medical School

New Brunswick, New Jersey, United States

Site Status

Albany Medical College, Pulmonary and Critical Care Medicine

Albany, New York, United States

Site Status

Lung and Cystic Fibrosis Center at Women and Children's Hospital of Buffalo

Buffalo, New York, United States

Site Status

Northwell Health- Long Island Jewish Medical Center

New Hyde Park, New York, United States

Site Status

Columbia University Medical Center

New York, New York, United States

Site Status

SUNY Upstate Medical University

Syracuse, New York, United States

Site Status

Clinical Research of Charlotte

Charlotte, North Carolina, United States

Site Status

Duke University Medical Center

Durham, North Carolina, United States

Site Status

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, United States

Site Status

Santiago Reyes, M.D.

Oklahoma City, Oklahoma, United States

Site Status

Oregon Health & Science University

Portland, Oregon, United States

Site Status

Children's Hospital of Pittsburgh of UPMC

Pittsburgh, Pennsylvania, United States

Site Status

Sanford Children's Speciality Clinic

Sioux Falls, South Dakota, United States

Site Status

University of Tennessee Medical Center

Knoxville, Tennessee, United States

Site Status

Children's Foundation Research Center / Le Bonheur Children's Hospital

Memphis, Tennessee, United States

Site Status

Vanderbilt University Medical Center

Nashville, Tennessee, United States

Site Status

Cook Children's Medical Center

Fort Worth, Texas, United States

Site Status

Texas Children's Hospital

Houston, Texas, United States

Site Status

University of Utah / Primary Children's Medical Center

Salt Lake City, Utah, United States

Site Status

Seattle Children's Hospital

Seattle, Washington, United States

Site Status

Providence Pediatric Pulmonary & Cystic Fibrosis Clinic

Spokane, Washington, United States

Site Status

Royal Adelaide Hospital

Adelaide, , Australia

Site Status

The Prince Charles Hospital

Chermside, , Australia

Site Status

Alfred Hospital

Melbourne, VIC, , Australia

Site Status

Institute for Respiratory Health

Nedlands, , Australia

Site Status

Perth Children's Hospital

Nedlands, , Australia

Site Status

John Hunter Hospital & Hunter Medical Research Institute and John Hunter Children's Hospital

New Lambton, , Australia

Site Status

Queensland Children's Hospital

South Brisbane, , Australia

Site Status

Stollery Children's Hospital

Edmonton, , Canada

Site Status

Queen Elizabeth II Health Sciences Center

Halifax, , Canada

Site Status

St. Michael's Hospital

Toronto, , Canada

Site Status

Juliane Marie Center, Rigshospitalet

Copenhagen, , Denmark

Site Status

Charite Paediatric Pulmonology Department

Berlin, , Germany

Site Status

Universitaetsklinkum Koeln, CF-Studienzentrum

Cologne, , Germany

Site Status

Ruhrlandklinik Westdeutsches Lungenzentrum am Klinikum Essen

Essen, , Germany

Site Status

Johann Wolfgang Goethe University

Frankfurt, , Germany

Site Status

Hannover Medical School

Hanover, , Germany

Site Status

Universitaetsklinikum Jena, Mukoviszidose-Zentrum

Jena, , Germany

Site Status

Universitatsklinikum Schleswig-Holstein, Klinik für Kinder- und Jugendmedizin

Lübeck, , Germany

Site Status

Klinikum Innenstadt, University of Munich

München, , Germany

Site Status

Pneumologisches Studienzentrum Muenchen-West

München, , Germany

Site Status

Cork University Hospital

Cork, , Ireland

Site Status

Beaumont Hospital

Dublin, , Ireland

Site Status

Children's Health Ireland at Crumlin

Dublin, , Ireland

Site Status

Children's Health Ireland at Temple Street

Dublin, , Ireland

Site Status

St. Vincent's University Hospital

Dublin, , Ireland

Site Status

University Hospital Galway

Galway, , Ireland

Site Status

University Hospital Limerick (Adults)

Limerick, , Ireland

Site Status

Lady Davis Carmel Medical Center

Haifa, , Israel

Site Status

Pediatric Pulmonary Unit Rambam Medical Center

Haifa, , Israel

Site Status

Hadassah Medical Organization

Jerusalem, , Israel

Site Status

Schneider Children's Medical Center

Petah Tikva, , Israel

Site Status

Sheba Medical Center

Tel Litwinsky, , Israel

Site Status

Klinika Mukowiscydozy IMD Oddozial Chorob Pluc Szpzoz IM. Dzieci WarszaWY

Łomianki, , Poland

Site Status

Hospital Universitari Vall d Hebron

Barcelona, , Spain

Site Status

Hospital Universitari Vall d´Hebron Servicio de Broncoscopia

Barcelona, , Spain

Site Status

Hospital Universitario 12 de Octubre

Madrid, , Spain

Site Status

Hospital Universitario Infantil La Paz

Madrid, , Spain

Site Status

Parc Tauli Sabadell Hospital Universitari

Sabadell, , Spain

Site Status

Hospital Universitario Virgen del Rocio

Seville, , Spain

Site Status

Hospital Universitario y Politecnico La Fe

Valencia, , Spain

Site Status

Lindenhofspital - Quartier Bleu

Bern, , Switzerland

Site Status

Kinderspital Zuerich

Zurich, , Switzerland

Site Status

Papworth Hospital NHS Foundation Trust, Papworth Everard

Cambridge, , United Kingdom

Site Status

NHS Greater Glasgow and Clyde, Gartnavel General Hospital

Glasgow, , United Kingdom

Site Status

St. James University Hospital

Leeds, , United Kingdom

Site Status

Liverpool Heart and Chest Hospital

Liverpool, , United Kingdom

Site Status

Royal Brompton & Harefield NHS Foundation Trust, Royal Brompton Hospital

London, , United Kingdom

Site Status

Wythenshawe Hospital

Manchester, , United Kingdom

Site Status

The Newcastle upon Tyne Hospitals NHS Foundation Trust, The Royal Victoria Infirmary

Newcastle upon Tyne, , United Kingdom

Site Status

Nottingham University Hospitals NHS Trust, Queens Medical Center

Nottingham, , United Kingdom

Site Status

All Wales Adult Cystic Fibrosis Centre, University Hospital Llandough

Penarth, , United Kingdom

Site Status

Countries

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

United States Australia Canada Denmark Germany Ireland Israel Poland Spain Switzerland United Kingdom

References

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

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.

2018-004652-38

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

VX18-445-113

Identifier Type: -

Identifier Source: org_study_id

More Related Trials

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