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
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Locations
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AP-HM - Hopital de la Timone
Marseille, France
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AP-HP Hopital Bichat - Claude Bernard
Paris, France
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AP-HP Hopital Henri Mondor
Créteil, France
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AZ Sint-Jan Brugge
Bruges, Belgium
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Aarhus University Hospital
Aarhus, Denmark
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Alberta Health Services - University of Calgary
Calgary, Alberta, Canada
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Allegheny General Hospital
Pittsburgh, Pennsylvania, 15212, United States
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Antwerp University Hospital
Edegem, Belgium
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Azienda Ospedaliero-Universitaria Careggi
Florence, Italy
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Azienda Ospedaliero-Universitaria di Ferrara-Arcispedale Sant'Anna
Cona, Italy
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Azienda sanitaria universitaria Giuliano Isontina (ASU GI) - Ospedale di Cattinara
Trieste, Italy
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Baylor College of Medicine
Houston, Texas, 77030, United States
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Baylor Scott & White Research Institute
Dallas, Texas, 75201, United States
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Baylor Scott and White - The Heart Hospital Baylor Plano
Plano, Texas, 75093, United States
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Boston University School of Medicine
Boston, Massachusetts, 02118, United States
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Brigham and Women's Hospital
Boston, Massachusetts, 02115, United States
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CAPED - Centro Avancado de Pesquisa
Ribeirão Preto, Brazil
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CHU Rennes - Hopital Pontchaillou
Rennes, France
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CHU de Toulouse - Hopital Rangueil
Toulouse, France
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Cardiopulmonar da Bahia S. A. (Hospital Cardio Pulmonar)
Salvador, Brazil
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Cedars-Sinai Medical Center
Los Angeles, California, 90048, United States
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Centre Hospitalier Regional de la Citadelle
Liège, Belgium
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Centro Hospitalar Universitario de Lisboa Norte
Lisbon, Portugal
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Centro Hospitalar Universitario do Porto
Porto, Portugal
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Centro de Pesquisa NUPEC - Instituto Orizonti em Belo Horizonte
Belo Horizonte, Brazil
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Charite Universitaetsmedizin Berlin
Berlin, Germany
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Cleveland Clinic
Cleveland, Ohio, 44195, United States
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Cleveland Clinic of Florida
Weston, Florida, 33331, United States
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Columbia University Medical Center
New York, New York, 10032, United States
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Complexo Hospitalar de Niterói (CHN-DASA)
Niterói, Brazil
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Duke University Medical Center
Durham, North Carolina, 27710, United States
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Eastern Health - Box Hill Hospital
Box Hill, Victoria, Australia
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Emory University School of Medicine
Atlanta, Georgia, 30322, United States
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Erasmus MC
Rotterdam, Netherlands
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Fakultni nemocnice u sv. Anny v Brne
Brno, Czechia
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Far Eastern Memorial Hospital
New Taipei City, Taiwan
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Fiona Stanley Hospital
Perth, Western Australia, Australia
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Fondazione IRCCS Policlinico San Matteo
Pavia, Italy
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Fondazione Toscana Gabriele Monasterio per la Ricerca Medica
Pisa, Italy
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Fundacion Favaloro
Ciudad Autonoma de Buenos Aire, Argentina
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Fundacion Favalro
Buenos Aires, Argentina
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HCFMRP - Hospital das Clinicas da Faculdade de Medicina de Ribeirao Preto
Ribeirão Preto, Brazil
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Hadassah University Hospital, Ein Karem
Jerusalem, Israel
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Hospital Israelita Albert Einstein
São Paulo, Brazil
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Hospital Italiano de Buenos Aires
Buenos Aires, Argentina
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Hospital Italiano de Buenos Aires
Ciudad Autonoma de Buenos Aire, Argentina
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Hospital Privado de Rosario
Rosario, Argentina
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Hospital Universitari de Bellvitge
Barcelona, Spain
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Hospital Universitario Puerta de Hierro
Majadahonda, Spain
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Hospital Universitario Son Llatzer
Palma de Mallorca, Spain
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Hospital Universitario Vall d'Hebron
Barcelona, Spain
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Hospital Universitario Virgen de la Victoria
Málaga, Spain
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Hospital Universitario de A Coruna
A Coruña, Spain
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Houston Methodist Hospital
Houston, Texas, 77030, United States
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ICBA
Buenos Aires, Argentina
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INOVA Fairfax Hospital
Falls Church, Virginia, 22042, United States
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IRCCS Azienda Ospedaliero-Universitaria di Bologna - Policlinico di Sant'Orsola
Bologna, Italy
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Indiana University Health
Indianapolis, Indiana, 46202, United States
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Instituto Dante Pazzanese de Cardiologia
São Paulo, Brazil
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Instituto Nacional de Ciencias Medicas y Nutricion Salvador Zubiran
Mexico City, Mexico
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Instituto do Coracao (InCor) do Hospital das Clinicas da FMUSP
São Paulo, Brazil
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Johns Hopkins University
Baltimore, Maryland, 21287, United States
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Karolinska University Hospital
Stockholm, Sweden
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Kumamoto University Hospital
Kumamoto, Japan
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Mayo Clinic
Rochester, Minnesota, 55905, United States
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Mayo Clinic - Jacksonville
Jacksonville, Florida, 32224, United States
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McGill University Health Centre
Montreal, Quebec, Canada
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MedStar Washington Hospital Center - MedStar Heart and Vascular Institute
Washington D.C., District of Columbia, 20010, United States
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Medical University of South Carolina
Charleston, South Carolina, 29425, United States
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Medizinische Universitaet Innsbruck
Innsbruck, Austria
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National Cerebral and Cardiovascular Center
Osaka, Japan
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National Heart Centre
Singapore, Singapore
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National Taiwan University Hospital
Taipei, Taiwan
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National University Hospital
Singapore, Singapore
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New York University (NYU) School of Medicine - Langone Medical Center
New York, New York, 10016, United States
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New Zealand Clinical Research
Auckland, New Zealand
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New Zealand Clinical Research
Christchurch, New Zealand
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Northwestern University
Chicago, Illinois, 60611, United States
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Ohio Health Research Institute
Columbus, Ohio, 43214, United States
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Oregon Health and Science University (OHSU)
Portland, Oregon, 97239, United States
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Oslo University Hospital - Rikshospitalet
Oslo, Norway
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Ospedale Policlinico San Martino IRCCS
Genova, Italy
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PSEG Centro de Pesquisa Clinica S.A.
São Paulo, Brazil
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Penn Presbyterian Medical Center
Philadelphia, Pennsylvania, 19104, United States
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Princess Alexandra Hospital
Woolloongabba, Australia
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Rambam Health Care Campus
Haifa, Israel
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Richmond Pharmacology
London, United Kingdom
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Rigshospitalet
Copenhagen, Denmark
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Royal Adelaide Hospital
Adelaide, Australia
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Semmelweis Egyetem Belgyogyaszati es Hematologiai Klinika
Budapest, Hungary
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Sheba Medical Center
Tel Litwinsky, Israel
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St. Luke's Hospital
Kansas City, Missouri, 64111, United States
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Stanford University
Palo Alto, California, 94305, United States
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Synexus Hexham Clinical Research Centre
Hexham, United Kingdom
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Synexus Manchester Clinical Research Centre
Manchester, United Kingdom
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Synexus Merseyside Clinical Research Centre
Liverpool, United Kingdom
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Synexus Midlands Clinical Research Centre
Birmingham, United Kingdom
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Synexus Scotland Clinical Research Centre
Glasgow, United Kingdom
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Synexus Wales Clinical Research Centre
Cardiff, United Kingdom
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Taipai Veterans General Hospital
Taipei, Taiwan
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The Alfred Hospital
Melbourne, Australia
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The Canberra Hospital
Garran, Australian Capital Territory, Australia
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The University of Tokyo Hospital
Tokyo, Japan
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Toyama University Hospital
Toyama, Japan
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UZ Leuven
Leuven, Belgium
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Universitaesklinikum St. Poelten
Sankt Pölten, Austria
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Universitaetsklinikum Essen
Essen, Germany
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Universitaetsklinikum Heidelberg
Heidelberg, Germany
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Universitaetsklinikum Wuerzburg
Würzburg, Germany
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Universitair Medisch Centrum Utrecht
Utrecht, Netherlands
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Universite Libre de Bruxelles (ULB) - Hopital Erasme
Brussels, Belgium
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University Health Network - Toronto General Hospital
Toronto, Ontario, Canada
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University Hospital of Umea
Umeå, Sweden
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University Medical Center
Groningen, Netherlands
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University of Arizona
Tucson, Arizona, 85724, United States
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University of California, San Diego (UCSD) - Medical Center
San Diego, California, 92103, United States
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University of Colorado Denver
Denver, Colorado, 80045, United States
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University of Kentucky Gill Heart Institute
Lexington, Kentucky, 40536, United States
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University of Michigan
Ann Arbor, Michigan, 48109, United States
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University of Pittsburgh Medical Center
Pittsburgh, Pennsylvania, 15219, United States
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University of Texas Southwestern Medical Center
Dallas, Texas, 75390, United States
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Vancouver Coastal Health Research Institute
Vancouver, British Columbia, Canada
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Virginia Commonwealth University - Department of Internal Medicine
Richmond, Virginia, 23298, United States
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Western Greater Los Angeles VA Medical Center
Los Angeles, California, 90073, United States
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Westmead Hospital
Westmead, New South Wales, Australia
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Yale University School of Medicine
New Haven, Connecticut, 06510, United States
Conditions
Explore the condition pages connected to this study.