One-Shot gene therapy aims to halt deadly heart disease

NCT ID NCT06128629

Summary

This large, late-stage study is testing whether a single dose of an experimental gene therapy, called NTLA-2001, can help people with a serious inherited heart condition called ATTR-CM. The main goal is to see if the treatment reduces heart-related deaths and hospital visits compared to a placebo. About 1,200 participants worldwide will receive either the therapy or a dummy infusion to compare the results.

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

  • AP-HM - Hopital de la Timone

    Marseille, France

  • AP-HP Hopital Bichat - Claude Bernard

    Paris, France

  • AP-HP Hopital Henri Mondor

    Créteil, France

  • AZ Sint-Jan Brugge

    Bruges, Belgium

  • Aarhus University Hospital

    Aarhus, Denmark

  • Alberta Health Services - University of Calgary

    Calgary, Alberta, Canada

  • Allegheny General Hospital

    Pittsburgh, Pennsylvania, 15212, United States

  • Antwerp University Hospital

    Edegem, Belgium

  • Azienda Ospedaliero-Universitaria Careggi

    Florence, Italy

  • Azienda Ospedaliero-Universitaria di Ferrara-Arcispedale Sant'Anna

    Cona, Italy

  • Azienda sanitaria universitaria Giuliano Isontina (ASU GI) - Ospedale di Cattinara

    Trieste, Italy

  • Baylor College of Medicine

    Houston, Texas, 77030, United States

  • Baylor Scott & White Research Institute

    Dallas, Texas, 75201, United States

  • Baylor Scott and White - The Heart Hospital Baylor Plano

    Plano, Texas, 75093, United States

  • Boston University School of Medicine

    Boston, Massachusetts, 02118, United States

  • Brigham and Women's Hospital

    Boston, Massachusetts, 02115, United States

  • CAPED - Centro Avancado de Pesquisa

    Ribeirão Preto, Brazil

  • CHU Rennes - Hopital Pontchaillou

    Rennes, France

  • CHU de Toulouse - Hopital Rangueil

    Toulouse, France

  • Cardiopulmonar da Bahia S. A. (Hospital Cardio Pulmonar)

    Salvador, Brazil

  • Cedars-Sinai Medical Center

    Los Angeles, California, 90048, United States

  • Centre Hospitalier Regional de la Citadelle

    Liège, Belgium

  • Centro Hospitalar Universitario de Lisboa Norte

    Lisbon, Portugal

  • Centro Hospitalar Universitario do Porto

    Porto, Portugal

  • Centro de Pesquisa NUPEC - Instituto Orizonti em Belo Horizonte

    Belo Horizonte, Brazil

  • Charite Universitaetsmedizin Berlin

    Berlin, Germany

  • Cleveland Clinic

    Cleveland, Ohio, 44195, United States

  • Cleveland Clinic of Florida

    Weston, Florida, 33331, United States

  • Columbia University Medical Center

    New York, New York, 10032, United States

  • Complexo Hospitalar de Niterói (CHN-DASA)

    Niterói, Brazil

  • Duke University Medical Center

    Durham, North Carolina, 27710, United States

  • Eastern Health - Box Hill Hospital

    Box Hill, Victoria, Australia

  • Emory University School of Medicine

    Atlanta, Georgia, 30322, United States

  • Erasmus MC

    Rotterdam, Netherlands

  • Fakultni nemocnice u sv. Anny v Brne

    Brno, Czechia

  • Far Eastern Memorial Hospital

    New Taipei City, Taiwan

  • Fiona Stanley Hospital

    Perth, Western Australia, Australia

  • Fondazione IRCCS Policlinico San Matteo

    Pavia, Italy

  • Fondazione Toscana Gabriele Monasterio per la Ricerca Medica

    Pisa, Italy

  • Fundacion Favaloro

    Ciudad Autonoma de Buenos Aire, Argentina

  • Fundacion Favalro

    Buenos Aires, Argentina

  • HCFMRP - Hospital das Clinicas da Faculdade de Medicina de Ribeirao Preto

    Ribeirão Preto, Brazil

  • Hadassah University Hospital, Ein Karem

    Jerusalem, Israel

  • Hospital Israelita Albert Einstein

    São Paulo, Brazil

  • Hospital Italiano de Buenos Aires

    Buenos Aires, Argentina

  • Hospital Italiano de Buenos Aires

    Ciudad Autonoma de Buenos Aire, Argentina

  • Hospital Privado de Rosario

    Rosario, Argentina

  • Hospital Universitari de Bellvitge

    Barcelona, Spain

  • Hospital Universitario Puerta de Hierro

    Majadahonda, Spain

  • Hospital Universitario Son Llatzer

    Palma de Mallorca, Spain

  • Hospital Universitario Vall d'Hebron

    Barcelona, Spain

  • Hospital Universitario Virgen de la Victoria

    Málaga, Spain

  • Hospital Universitario de A Coruna

    A Coruña, Spain

  • Houston Methodist Hospital

    Houston, Texas, 77030, United States

  • ICBA

    Buenos Aires, Argentina

  • INOVA Fairfax Hospital

    Falls Church, Virginia, 22042, United States

  • IRCCS Azienda Ospedaliero-Universitaria di Bologna - Policlinico di Sant'Orsola

    Bologna, Italy

  • Indiana University Health

    Indianapolis, Indiana, 46202, United States

  • Instituto Dante Pazzanese de Cardiologia

    São Paulo, Brazil

  • Instituto Nacional de Ciencias Medicas y Nutricion Salvador Zubiran

    Mexico City, Mexico

  • Instituto do Coracao (InCor) do Hospital das Clinicas da FMUSP

    São Paulo, Brazil

  • Johns Hopkins University

    Baltimore, Maryland, 21287, United States

  • Karolinska University Hospital

    Stockholm, Sweden

  • Kumamoto University Hospital

    Kumamoto, Japan

  • Mayo Clinic

    Rochester, Minnesota, 55905, United States

  • Mayo Clinic - Jacksonville

    Jacksonville, Florida, 32224, United States

  • McGill University Health Centre

    Montreal, Quebec, Canada

  • MedStar Washington Hospital Center - MedStar Heart and Vascular Institute

    Washington D.C., District of Columbia, 20010, United States

  • Medical University of South Carolina

    Charleston, South Carolina, 29425, United States

  • Medizinische Universitaet Innsbruck

    Innsbruck, Austria

  • National Cerebral and Cardiovascular Center

    Osaka, Japan

  • National Heart Centre

    Singapore, Singapore

  • National Taiwan University Hospital

    Taipei, Taiwan

  • National University Hospital

    Singapore, Singapore

  • New York University (NYU) School of Medicine - Langone Medical Center

    New York, New York, 10016, United States

  • New Zealand Clinical Research

    Auckland, New Zealand

  • New Zealand Clinical Research

    Christchurch, New Zealand

  • Northwestern University

    Chicago, Illinois, 60611, United States

  • Ohio Health Research Institute

    Columbus, Ohio, 43214, United States

  • Oregon Health and Science University (OHSU)

    Portland, Oregon, 97239, United States

  • Oslo University Hospital - Rikshospitalet

    Oslo, Norway

  • Ospedale Policlinico San Martino IRCCS

    Genova, Italy

  • PSEG Centro de Pesquisa Clinica S.A.

    São Paulo, Brazil

  • Penn Presbyterian Medical Center

    Philadelphia, Pennsylvania, 19104, United States

  • Princess Alexandra Hospital

    Woolloongabba, Australia

  • Rambam Health Care Campus

    Haifa, Israel

  • Richmond Pharmacology

    London, United Kingdom

  • Rigshospitalet

    Copenhagen, Denmark

  • Royal Adelaide Hospital

    Adelaide, Australia

  • Semmelweis Egyetem Belgyogyaszati es Hematologiai Klinika

    Budapest, Hungary

  • Sheba Medical Center

    Tel Litwinsky, Israel

  • St. Luke's Hospital

    Kansas City, Missouri, 64111, United States

  • Stanford University

    Palo Alto, California, 94305, United States

  • Synexus Hexham Clinical Research Centre

    Hexham, United Kingdom

  • Synexus Manchester Clinical Research Centre

    Manchester, United Kingdom

  • Synexus Merseyside Clinical Research Centre

    Liverpool, United Kingdom

  • Synexus Midlands Clinical Research Centre

    Birmingham, United Kingdom

  • Synexus Scotland Clinical Research Centre

    Glasgow, United Kingdom

  • Synexus Wales Clinical Research Centre

    Cardiff, United Kingdom

  • Taipai Veterans General Hospital

    Taipei, Taiwan

  • The Alfred Hospital

    Melbourne, Australia

  • The Canberra Hospital

    Garran, Australian Capital Territory, Australia

  • The University of Tokyo Hospital

    Tokyo, Japan

  • Toyama University Hospital

    Toyama, Japan

  • UZ Leuven

    Leuven, Belgium

  • Universitaesklinikum St. Poelten

    Sankt Pölten, Austria

  • Universitaetsklinikum Essen

    Essen, Germany

  • Universitaetsklinikum Heidelberg

    Heidelberg, Germany

  • Universitaetsklinikum Wuerzburg

    Würzburg, Germany

  • Universitair Medisch Centrum Utrecht

    Utrecht, Netherlands

  • Universite Libre de Bruxelles (ULB) - Hopital Erasme

    Brussels, Belgium

  • University Health Network - Toronto General Hospital

    Toronto, Ontario, Canada

  • University Hospital of Umea

    Umeå, Sweden

  • University Medical Center

    Groningen, Netherlands

  • University of Arizona

    Tucson, Arizona, 85724, United States

  • University of California, San Diego (UCSD) - Medical Center

    San Diego, California, 92103, United States

  • University of Colorado Denver

    Denver, Colorado, 80045, United States

  • University of Kentucky Gill Heart Institute

    Lexington, Kentucky, 40536, United States

  • University of Michigan

    Ann Arbor, Michigan, 48109, United States

  • University of Pittsburgh Medical Center

    Pittsburgh, Pennsylvania, 15219, United States

  • University of Texas Southwestern Medical Center

    Dallas, Texas, 75390, United States

  • Vancouver Coastal Health Research Institute

    Vancouver, British Columbia, Canada

  • Virginia Commonwealth University - Department of Internal Medicine

    Richmond, Virginia, 23298, United States

  • Western Greater Los Angeles VA Medical Center

    Los Angeles, California, 90073, United States

  • Westmead Hospital

    Westmead, New South Wales, Australia

  • Yale University School of Medicine

    New Haven, Connecticut, 06510, United States

Conditions

Explore the condition pages connected to this study.