One-Shot gene therapy could transform treatment for fatal heart disease

NCT ID NCT06128629

Recruiting now Disease control Sponsor: Intellia Therapeutics Source: ClinicalTrials.gov ↗

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 and often fatal heart disease called ATTR amyloidosis with cardiomyopathy. About 1200 participants worldwide will receive either the therapy or a placebo to see if it reduces heart-related deaths and hospitalizations. The main goal is to see if this one-time treatment can better control the disease compared to a dummy treatment.

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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Study contacts

Contact

Phone: •••-•••-•••• Email: •••••@•••••

Locations

AP-HM - Hopital de la Timone
RECRUITING

Marseille, France
AP-HP Hopital Bichat - Claude Bernard
RECRUITING

Paris, France
AP-HP Hopital Henri Mondor
RECRUITING

Créteil, France
AZ Sint-Jan Brugge
RECRUITING

Bruges, Belgium
Aarhus University Hospital
RECRUITING

Aarhus, Denmark
Alberta Health Services - University of Calgary
RECRUITING

Calgary, Alberta, Canada
Allegheny General Hospital
RECRUITING

Pittsburgh, Pennsylvania, 15212, United States
Antwerp University Hospital
RECRUITING

Edegem, Belgium
Azienda Ospedaliero-Universitaria Careggi
RECRUITING

Florence, Italy
Azienda Ospedaliero-Universitaria di Ferrara-Arcispedale Sant'Anna
RECRUITING

Cona, Italy
Azienda sanitaria universitaria Giuliano Isontina (ASU GI) - Ospedale di Cattinara
RECRUITING

Trieste, Italy
Barbara Ann Karmanos Cancer Institute
WITHDRAWN

Detroit, Michigan, 48201, United States
Baylor College of Medicine
RECRUITING

Houston, Texas, 77030, United States
Baylor Scott & White Research Institute
RECRUITING

Dallas, Texas, 75201, United States
Baylor Scott and White - The Heart Hospital Baylor Plano
RECRUITING

Plano, Texas, 75093, United States
Boston University School of Medicine
RECRUITING

Boston, Massachusetts, 02118, United States
Brigham and Women's Hospital
RECRUITING

Boston, Massachusetts, 02115, United States
CAPED - Centro Avancado de Pesquisa
RECRUITING

Ribeirão Preto, Brazil
CHU Rennes - Hopital Pontchaillou
RECRUITING

Rennes, France
CHU de Toulouse - Hopital Rangueil
RECRUITING

Toulouse, France
Cairns Hospital
WITHDRAWN

Cairns, Queensland, Australia
Cardiopulmonar da Bahia S. A. (Hospital Cardio Pulmonar)
RECRUITING

Salvador, Brazil
Cedars-Sinai Medical Center
RECRUITING

Los Angeles, California, 90048, United States
Centre Hospitalier Regional de la Citadelle
RECRUITING

Liège, Belgium
Centro Hospitalar Universitario de Lisboa Norte
RECRUITING

Lisbon, Portugal
Centro Hospitalar Universitario do Porto
RECRUITING

Porto, Portugal
Centro de Pesquisa NUPEC - Instituto Orizonti em Belo Horizonte
RECRUITING

Belo Horizonte, Brazil
Charite Universitaetsmedizin Berlin
RECRUITING

Berlin, Germany
Chonnam National University Hospital
WITHDRAWN

Gwangju, South Korea
Cleveland Clinic
RECRUITING

Cleveland, Ohio, 44195, United States
Cleveland Clinic of Florida
RECRUITING

Weston, Florida, 33331, United States
Columbia University Medical Center
RECRUITING

New York, New York, 10032, United States
Complexo Hospitalar de Niterói (CHN-DASA)
RECRUITING

Niterói, Brazil
Duke University Medical Center
RECRUITING

Durham, North Carolina, 27710, United States
Eastern Health - Box Hill Hospital
RECRUITING

Box Hill, Victoria, Australia
Emory University School of Medicine
RECRUITING

Atlanta, Georgia, 30322, United States
Erasmus MC
RECRUITING

Rotterdam, Netherlands
Fakultni nemocnice u sv. Anny v Brne
RECRUITING

Brno, Czechia
Far Eastern Memorial Hospital
RECRUITING

New Taipei City, Taiwan
Fiona Stanley Hospital
RECRUITING

Perth, Western Australia, Australia
Fondazione IRCCS Policlinico San Matteo
RECRUITING

Pavia, Italy
Fondazione Toscana Gabriele Monasterio per la Ricerca Medica
RECRUITING

Pisa, Italy
Fundacion Favaloro
RECRUITING

Ciudad Autonoma de Buenos Aire, Argentina
Fundacion Favalro
RECRUITING

Buenos Aires, Argentina
HCFMRP - Hospital das Clinicas da Faculdade de Medicina de Ribeirao Preto
RECRUITING

Ribeirão Preto, Brazil
Hadassah University Hospital, Ein Karem
RECRUITING

Jerusalem, Israel
Hospital Israelita Albert Einstein
RECRUITING

São Paulo, Brazil
Hospital Italiano de Buenos Aires
RECRUITING

Buenos Aires, Argentina
Hospital Italiano de Buenos Aires
RECRUITING

Ciudad Autonoma de Buenos Aire, Argentina
Hospital Privado de Rosario
RECRUITING

Rosario, Argentina
Hospital Universitari de Bellvitge
RECRUITING

Barcelona, Spain
Hospital Universitario Puerta de Hierro
RECRUITING

Majadahonda, Spain
Hospital Universitario Son Llatzer
RECRUITING

Palma de Mallorca, Spain
Hospital Universitario Vall d'Hebron
RECRUITING

Barcelona, Spain
Hospital Universitario Virgen de la Victoria
RECRUITING

Málaga, Spain
Hospital Universitario de A Coruna
RECRUITING

A Coruña, Spain
Houston Methodist Hospital
RECRUITING

Houston, Texas, 77030, United States
ICBA
RECRUITING

Buenos Aires, Argentina
INOVA Fairfax Hospital
RECRUITING

Falls Church, Virginia, 22042, United States
IRCCS Azienda Ospedaliero-Universitaria di Bologna - Policlinico di Sant'Orsola
RECRUITING

Bologna, Italy
Indiana University Health
RECRUITING

Indianapolis, Indiana, 46202, United States
Instituto Dante Pazzanese de Cardiologia
RECRUITING

São Paulo, Brazil
Instituto Nacional de Ciencias Medicas y Nutricion Salvador Zubiran
RECRUITING

Mexico City, Mexico
Instituto do Coracao (InCor) do Hospital das Clinicas da FMUSP
RECRUITING

São Paulo, Brazil
Johns Hopkins University
RECRUITING

Baltimore, Maryland, 21287, United States
Karolinska University Hospital
RECRUITING

Stockholm, Sweden
Kumamoto University Hospital
RECRUITING

Kumamoto, Japan
Mayo Clinic
RECRUITING

Rochester, Minnesota, 55905, United States
Mayo Clinic - Jacksonville
RECRUITING

Jacksonville, Florida, 32224, United States
McGill University Health Centre
RECRUITING

Montreal, Quebec, Canada
MedStar Washington Hospital Center - MedStar Heart and Vascular Institute
RECRUITING

Washington D.C., District of Columbia, 20010, United States
Medical University of South Carolina
RECRUITING

Charleston, South Carolina, 29425, United States
Medizinische Universitaet Innsbruck
RECRUITING

Innsbruck, Austria
National Cerebral and Cardiovascular Center
RECRUITING

Osaka, Japan
National Heart Centre
RECRUITING

Singapore, Singapore
National Taiwan University Hospital
RECRUITING

Taipei, Taiwan
National University Hospital
RECRUITING

Singapore, Singapore
New York University (NYU) School of Medicine - Langone Medical Center
RECRUITING

New York, New York, 10016, United States
New Zealand Clinical Research
RECRUITING

Auckland, New Zealand
New Zealand Clinical Research
RECRUITING

Christchurch, New Zealand
Northwestern University
RECRUITING

Chicago, Illinois, 60611, United States
Ohio Health Research Institute
RECRUITING

Columbus, Ohio, 43214, United States
Oregon Health and Science University (OHSU)
RECRUITING

Portland, Oregon, 97239, United States
Oslo University Hospital - Rikshospitalet
RECRUITING

Oslo, Norway
Ospedale Policlinico San Martino IRCCS
RECRUITING

Genova, Italy
PSEG Centro de Pesquisa Clinica S.A.
RECRUITING

São Paulo, Brazil
Penn Presbyterian Medical Center
RECRUITING

Philadelphia, Pennsylvania, 19104, United States
Princess Alexandra Hospital
RECRUITING

Woolloongabba, Australia
Rambam Health Care Campus
RECRUITING

Haifa, Israel
Richmond Pharmacology
RECRUITING

London, United Kingdom
Rigshospitalet
RECRUITING

Copenhagen, Denmark
Royal Adelaide Hospital
RECRUITING

Adelaide, Australia
Samsung Medical Center
WITHDRAWN

Seoul, South Korea
Semmelweis Egyetem Belgyogyaszati es Hematologiai Klinika
RECRUITING

Budapest, Hungary
Seoul National University Hospital
WITHDRAWN

Seoul, South Korea
Severance Hospital, Yonsei University Health System
WITHDRAWN

Seoul, South Korea
Sheba Medical Center
RECRUITING

Tel Litwinsky, Israel
St. Luke's Hospital
RECRUITING

Kansas City, Missouri, 64111, United States
Stanford University
RECRUITING

Palo Alto, California, 94305, United States
Synexus Hexham Clinical Research Centre
ACTIVE_NOT_RECRUITING

Hexham, United Kingdom
Synexus Manchester Clinical Research Centre
ACTIVE_NOT_RECRUITING

Manchester, United Kingdom
Synexus Merseyside Clinical Research Centre
ACTIVE_NOT_RECRUITING

Liverpool, United Kingdom
Synexus Midlands Clinical Research Centre
ACTIVE_NOT_RECRUITING

Birmingham, United Kingdom
Synexus Scotland Clinical Research Centre
ACTIVE_NOT_RECRUITING

Glasgow, United Kingdom
Synexus Wales Clinical Research Centre
ACTIVE_NOT_RECRUITING

Cardiff, United Kingdom
Taipai Veterans General Hospital
RECRUITING

Taipei, Taiwan
The Alfred Hospital
RECRUITING

Melbourne, Australia
The Canberra Hospital
RECRUITING

Garran, Australian Capital Territory, Australia
The University of Tokyo Hospital
RECRUITING

Tokyo, Japan
Toyama University Hospital
RECRUITING

Toyama, Japan
UZ Leuven
RECRUITING

Leuven, Belgium
Universitaesklinikum St. Poelten
RECRUITING

Sankt Pölten, Austria
Universitaetsklinikum Essen
RECRUITING

Essen, Germany
Universitaetsklinikum Heidelberg
RECRUITING

Heidelberg, Germany
Universitaetsklinikum Wuerzburg
RECRUITING

Würzburg, Germany
Universitair Medisch Centrum Utrecht
RECRUITING

Utrecht, Netherlands
Universite Libre de Bruxelles (ULB) - Hopital Erasme
RECRUITING

Brussels, Belgium
University Health Network - Toronto General Hospital
RECRUITING

Toronto, Ontario, Canada
University Hospital of Umea
RECRUITING

Umeå, Sweden
University Medical Center
RECRUITING

Groningen, Netherlands
University of Arizona
RECRUITING

Tucson, Arizona, 85724, United States
University of California, San Diego (UCSD) - Medical Center
RECRUITING

San Diego, California, 92103, United States
University of Colorado Denver
RECRUITING

Denver, Colorado, 80045, United States
University of Kentucky Gill Heart Institute
RECRUITING

Lexington, Kentucky, 40536, United States
University of Michigan
RECRUITING

Ann Arbor, Michigan, 48109, United States
University of Pittsburgh Medical Center
RECRUITING

Pittsburgh, Pennsylvania, 15219, United States
University of Texas Southwestern Medical Center
RECRUITING

Dallas, Texas, 75390, United States
Vancouver Coastal Health Research Institute
RECRUITING

Vancouver, British Columbia, Canada
Virginia Commonwealth University - Department of Internal Medicine
RECRUITING

Richmond, Virginia, 23298, United States
Western Greater Los Angeles VA Medical Center
RECRUITING

Los Angeles, California, 90073, United States
Westmead Hospital
RECRUITING

Westmead, New South Wales, Australia
Yale University School of Medicine
RECRUITING

New Haven, Connecticut, 06510, United States

Conditions

Explore the condition pages connected to this study.

TRANSTHYRETIN AMYLOIDOSIS (ATTR) WITH CARDIOMYOPATHY