Breakthrough triple therapy shows promise for Long-Term CF management

NCT ID NCT03525574

Summary

This study followed 507 people with cystic fibrosis to check the long-term safety and effectiveness of a three-drug combination therapy. It focused on patients with a specific genetic mutation (F508del) that causes most CF cases. Researchers monitored lung function, weight, quality of life, and side effects over an extended period to see if benefits were maintained.

This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes NO responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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Contacts and locations

Locations

  • Academic Medical Center

    Amsterdam, Netherlands

  • Akron Children's Hospital

    Akron, Ohio, 44308, United States

  • Ann & Robert Lurie Children's Hospital of Chicago

    Chicago, Illinois, 60611, United States

  • Augusta University

    Augusta, Georgia, 30912, United States

  • Azienda Ospedaliera Universitaria Policlinico G. Martino

    Messina, Italy

  • Azienda Ospedaliera di Verona-Ospedale Civile Maggiore

    Verona, Italy

  • Azienda Ospedaliero Universitaria Ospedale Pediatrico Meyer

    Florence, Italy

  • Azienda Ospedaliero Universitaria Ospendali Riuniti

    Ancona, Italy

  • Banner University of Arizona Medical Center

    Tucson, Arizona, 85724, United States

  • Belfast City Hospital

    Belfast, United Kingdom

  • Billings Clinic Hospital

    Billings, Montana, 59101, United States

  • British Columbia's Children's Hospital

    Vancouver, Canada

  • CHU Marseille - Hopital Nord

    Marseille, France

  • CHU de Nice - Hopital Pasteur

    Nice, France

  • CHU de Rouen - Hopital Charles Nicolle

    Rouen, France

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

    Milwaukee, Wisconsin, 53226, United States

  • Central Florida Pulmonary Group, PA

    Orlando, Florida, 32803, United States

  • Centre Hospitalier De L'Universite Laval

    Québec, Canada

  • Centre Hospitalier Lyon Sud

    Benite Cedex, France

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

    Potenza, Italy

  • Children's Hospital Los Angeles

    Los Angeles, California, 90027, United States

  • Children's Hospital of Philadelphia

    Philadelphia, Pennsylvania, 19104, United States

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

    Richmond, Virginia, 23298, United States

  • Children's Hospital of the King's Daughters

    Norfolk, Virginia, 23507, United States

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

    Minneapolis, Minnesota, 55404, United States

  • Children's Specialty Services at North Druid Hills

    Atlanta, Georgia, 30324, United States

  • Cliniques Universitaires de Bruxelles Hopital Erasme

    Brussels, Belgium

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

    Peoria, Illinois, 61637, United States

  • Dayton Children's Hospital

    Dayton, Ohio, 45404, United States

  • Dell Children's Medical Center of Central Texas

    Austin, Texas, 78723, United States

  • Dr. von Haunersches Kinderspital

    München, Germany

  • Erasmus Medical Center

    Rotterdam, Netherlands

  • Fakultni Nemocnice Brno

    Brno, Czechia

  • Fakultni nemocnice v Motole

    Prague, Czechia

  • Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico

    Milan, Italy

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

    Erlangen, Germany

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

    Marousi, Greece

  • Groupe Hospitaler Pellegrin, CHU De Bordeaux

    Bordeaux, France

  • HagaZiekenhuis van den Haag

    The Hague, Netherlands

  • Hannover Medical School

    Hanover, Germany

  • Harper University Hospital

    Detroit, Michigan, 48201, United States

  • Heart of England NHS Foundation Trust, Birmingham Heartlands Hospital

    Birmingham, United Kingdom

  • Hopital Cochin

    Paris, France

  • Hopital Foch (Suresnes), Hopital Foch, Adultes

    Suresnes, France

  • IRCCS Istituto Giannina Gaslini-Ospedale Pediatrico

    Genova, Italy

  • Joe DiMaggio Cystic Fibrosis & Pulmonary Center

    Hollywood, Florida, 33021, United States

  • Johannes Gutenberg-Universitaet

    Mainz, Germany

  • Justus-Leibig-Universitat Zentrum fur Kinderheilkunde und Jugendmedizin

    Giessen, Germany

  • Kaiser Permanente

    Oakland, California, 94611, United States

  • Karolinska Universitetssjukhuset, Huddinge

    Stockholm, Sweden

  • King's College Hospital

    London, United Kingdom

  • LKH - Universitätsklinikum der PMU Salzburg

    Salzburg, Austria

  • Leeds General Infirmary

    Leeds, United Kingdom

  • Maine Medical Partners

    Portland, Maine, 04102, United States

  • Massachusetts General Hospital Cystic Fibrosis Center Clinical Research Center

    Boston, Massachusetts, 02114, United States

  • Mater Misericordiae Ltd

    South Brisbane, Australia

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

    Montreal, Canada

  • Medical University of South Carolina

    Charleston, South Carolina, 29425, United States

  • Medizinische Universitat Wien

    Vienna, Austria

  • Medizinische Universität Innsbruck

    Innsbruck, Austria

  • Miller Children's Hospital / Long Beach Memorial

    Long Beach, California, 90806, United States

  • Morristown Medical Center

    Morristown, New Jersey, 07960, United States

  • National Jewish Health

    Denver, Colorado, 80206, United States

  • Nationwide Children's Hospital

    Columbus, Ohio, 43205, United States

  • Nebraska Medical Center

    Omaha, Nebraska, 68198, United States

  • Nemours Children's Hospital

    Orlando, Florida, 32827, United States

  • Nemours Children's Specialty Care

    Jacksonville, Florida, 32207, United States

  • New York Medical College

    Valhalla, New York, 10595, United States

  • Northwestern Memorial Hospital

    Chicago, Illinois, 60611, United States

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

    Toledo, Ohio, 43606, United States

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

    Cleveland, Ohio, 44106, United States

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

    Exeter, United Kingdom

  • Royal Manchester Children's Hospital

    Manchester, United Kingdom

  • Southampton General Hospital

    Southampton, United Kingdom

  • St. Paul's Hospital

    Vancouver, Canada

  • Tampa General Hospital Cardiac and Lung Transplant Clinic

    Tampa, Florida, 33606, United States

  • The Children's Hospital at Westmead

    Westmead, Australia

  • The Cystic Fibrosis Center, Mount Sinai Beth Israel

    New York, New York, 10003, United States

  • The Hospital for Sick Children

    Toronto, Canada

  • The Royal Children's Hospital

    Parkville, Australia

  • The University of Kansas Medical Center

    Kansas City, Kansas, 66160, United States

  • The University of Texas Southwestern Medical Center

    Dallas, Texas, 75390, United States

  • Tulane Medical Center

    New Orleans, Louisiana, 70112, United States

  • UC Health Holmes

    Cincinnati, Ohio, 45220, United States

  • UCSF Gateway Medical Center

    San Francisco, California, 94143, United States

  • UMC St. Radboud

    Nijmegen, Netherlands

  • UNC Marsico Clinical Research Center

    Chapel Hill, North Carolina, 27517, United States

  • UNM Clinical and Translational Science Center

    Albuquerque, New Mexico, 87131, United States

  • UZ Antwerpen

    Edegem, Belgium

  • Universitaetsklinikum Tuebingen Klinik fuer Kinder- und Jugendmedizin

    Tübingen, Germany

  • Universitair Ziekenhuis Brussel - Campus Jette

    Brussels, Belgium

  • Universitair Ziekenhuis Gent

    Ghent, Belgium

  • Universitaire Ziekenhuizen Leuven - Campus Gasthuisberg

    Leuven, Belgium

  • University Hospital Wuerzburg

    Würzburg, Germany

  • University Medical Center, Utrecht, Department of Pulmonology and Tuberculosis

    Heidelberglaan, Netherlands

  • University of Arkansas for Medical Sciences

    Little Rock, Arkansas, 72205, United States

  • University of Calgary Medical Clinic of the Foothills Medical Centre

    Calgary, Canada

  • University of California Davis Medical Center

    Sacramento, California, 95817, United States

  • University of Florida, Shands Hospital

    Gainesville, Florida, 32610, United States

  • University of Graz

    Graz, Austria

  • University of Oklahoma Health Sciences Center

    Oklahoma City, Oklahoma, 73104, United States

  • University of Virginia Primary Care Center

    Charlottesville, Virginia, 22908, United States

  • University of Washington Medical Center

    Seattle, Washington, 98195, United States

  • University of Wisconsin Hospitals and Clinics

    Madison, Wisconsin, 53792, United States

  • Valley Children's Hospital

    Madera, California, 93636, United States

  • Vermont Lung Center

    Colchester, Vermont, 05446, United States

  • West Virginia University

    Morgantown, West Virginia, 26506, United States

  • Western General Hospital

    Edinburgh, United Kingdom

  • Westmead Hospital

    Westmead, Australia

  • Women & Children's Hospital

    North Adelaide, Australia

Conditions

Explore the condition pages connected to this study.