Clinical Trial
Study Details
Normal saline (0.9% NaCl) by IV infusion
Outcomes
Study Overview
To evaluate the efficacy and safety of a single dose of NTLA-2001 compared to placebo in participants with ATTR-CM.
This is a multinational, multicenter, double-blind, placebo-controlled study in approximately 1200 participants, who will be randomized to receive a single infusion of either NTLA-2001 or placebo.
Contacts and Locations
University of Arizona
Tucson, Arizona 85724
United States
Cedars-Sinai Medical Center
Los Angeles, California 90048
United States
Western Greater Los Angeles VA Medical Center
Los Angeles, California 90073
United States
Stanford University
Palo Alto, California 94305
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
Yale University School of Medicine
New Haven, Connecticut 06510
United States
MedStar Washington Hospital Center - MedStar Heart and Vascular Institute
Washington D.C., District of Columbia 20010
United States
Mayo Clinic - Jacksonville
Jacksonville, Florida 32224
United States
Cleveland Clinic of Florida
Weston, Florida 33331
United States
Emory University School of Medicine
Atlanta, Georgia 30322
United States
Northwestern University
Chicago, Illinois 60611
United States
Indiana University Health
Indianapolis, Indiana 46202
United States
University of Kentucky Gill Heart Institute
Lexington, Kentucky 40536
United States
Johns Hopkins University
Baltimore, Maryland 21287
United States
Brigham and Women's Hospital
Boston, Massachusetts 02115
United States
Boston University School of Medicine
Boston, Massachusetts 02118
United States
University of Michigan
Ann Arbor, Michigan 48109
United States
Barbara Ann Karmanos Cancer Institute
Detroit, Michigan 48201
United States
Mayo Clinic
Rochester, Minnesota 55905
United States
St. Luke's Hospital
Kansas City, Missouri 64111
United States
New York University (NYU) School of Medicine - Langone Medical Center
New York, New York 10016
United States
Columbia University Medical Center
New York, New York 10032
United States
Duke University Medical Center
Durham, North Carolina 27710
United States
Cleveland Clinic
Cleveland, Ohio 44195
United States
Ohio Health Research Institute
Columbus, Ohio 43214
United States
Oregon Health and Science University (OHSU)
Portland, Oregon 97239
United States
Penn Presbyterian Medical Center
Philadelphia, Pennsylvania 19104
United States
Allegheny General Hospital
Pittsburgh, Pennsylvania 15212
United States
University of Pittsburgh Medical Center
Pittsburgh, Pennsylvania 15219
United States
Medical University of South Carolina
Charleston, South Carolina 29425
United States
Baylor Scott & White Research Institute
Dallas, Texas 75201
United States
University of Texas Southwestern Medical Center
Dallas, Texas 75390
United States
Baylor College of Medicine
Houston, Texas 77030
United States
Houston Methodist Hospital
Houston, Texas 77030
United States
Baylor Scott and White - The Heart Hospital Baylor Plano
Plano, Texas 75093
United States
INOVA Fairfax Hospital
Falls Church, Virginia 22042
United States
Virginia Commonwealth University - Department of Internal Medicine
Richmond, Virginia 23298
United States
Fundacion Favalro
Buenos Aires
Argentina
Hospital Italiano de Buenos Aires
Buenos Aires
Argentina
ICBA
Buenos Aires
Argentina
Fundacion Favaloro
Ciudad Autonoma de Buenos Aire
Argentina
Hospital Italiano de Buenos Aires
Ciudad Autonoma de Buenos Aire
Argentina
Hospital Privado de Rosario
Rosario
Argentina
The Canberra Hospital
Garran Australian Capital Territory
Australia
Westmead Hospital
Westmead New South Wales
Australia
Cairns Hospital
Cairns Queensland
Australia
Eastern Health - Box Hill Hospital
Box Hill Victoria
Australia
Fiona Stanley Hospital
Perth Western Australia
Australia
Royal Adelaide Hospital
Adelaide
Australia
The Alfred Hospital
Melbourne
Australia
Princess Alexandra Hospital
Woolloongabba
Australia
Medizinische Universitaet Innsbruck
Innsbruck
Austria
Universitaesklinikum St. Poelten
Sankt Pölten
Austria
AZ Sint-Jan Brugge
Bruges
Belgium
Universite Libre de Bruxelles (ULB) - Hopital Erasme
Brussels
Belgium
Antwerp University Hospital
Edegem
Belgium
UZ Leuven
Leuven
Belgium
Centre Hospitalier Regional de la Citadelle
Liège
Belgium
Centro de Pesquisa NUPEC - Instituto Orizonti em Belo Horizonte
Belo Horizonte
Brazil
Complexo Hospitalar de Niterói (CHN-DASA)
Niterói
Brazil
CAPED - Centro Avancado de Pesquisa
Ribeirão Preto
Brazil
HCFMRP - Hospital das Clinicas da Faculdade de Medicina de Ribeirao Preto
Ribeirão Preto
Brazil
Cardiopulmonar da Bahia S. A. (Hospital Cardio Pulmonar)
Salvador
Brazil
Hospital Israelita Albert Einstein
São Paulo
Brazil
Instituto Dante Pazzanese de Cardiologia
São Paulo
Brazil
Instituto do Coracao (InCor) do Hospital das Clinicas da FMUSP
São Paulo
Brazil
PSEG Centro de Pesquisa Clinica S.A.
São Paulo
Brazil
Alberta Health Services - University of Calgary
Calgary Alberta
Canada
Vancouver Coastal Health Research Institute
Vancouver British Columbia
Canada
University Health Network - Toronto General Hospital
Toronto Ontario
Canada
McGill University Health Centre
Montreal Quebec
Canada
Fakultni nemocnice u sv. Anny v Brne
Brno
Czechia
Aarhus University Hospital
Aarhus
Denmark
Rigshospitalet
Copenhagen
Denmark
AP-HP Hopital Henri Mondor
Créteil
France
AP-HM - Hopital de la Timone
Marseille
France
AP-HP Hopital Bichat - Claude Bernard
Paris
France
CHU Rennes - Hopital Pontchaillou
Rennes
France
CHU de Toulouse - Hopital Rangueil
Toulouse
France
Charite Universitaetsmedizin Berlin
Berlin
Germany
Universitaetsklinikum Essen
Essen
Germany
Universitaetsklinikum Heidelberg
Heidelberg
Germany
Universitaetsklinikum Wuerzburg
Würzburg
Germany
Semmelweis Egyetem Belgyogyaszati es Hematologiai Klinika
Budapest
Hungary
Rambam Health Care Campus
Haifa
Israel
Hadassah University Hospital, Ein Karem
Jerusalem
Israel
Sheba Medical Center
Tel Litwinsky
Israel
IRCCS Azienda Ospedaliero-Universitaria di Bologna - Policlinico di Sant'Orsola
Bologna
Italy
Azienda Ospedaliero-Universitaria di Ferrara-Arcispedale Sant'Anna
Cona
Italy
Azienda Ospedaliero-Universitaria Careggi
Florence
Italy
Ospedale Policlinico San Martino IRCCS
Genova
Italy
Fondazione IRCCS Policlinico San Matteo
Pavia
Italy
Fondazione Toscana Gabriele Monasterio per la Ricerca Medica
Pisa
Italy
Azienda sanitaria universitaria Giuliano Isontina (ASU GI) - Ospedale di Cattinara
Trieste
Italy
Kumamoto University Hospital
Kumamoto
Japan
National Cerebral and Cardiovascular Center
Osaka
Japan
The University of Tokyo Hospital
Tokyo
Japan
Toyama University Hospital
Toyama
Japan
Instituto Nacional de Ciencias Medicas y Nutricion Salvador Zubiran
Mexico City
Mexico
University Medical Center
Groningen
Netherlands
Erasmus MC
Rotterdam
Netherlands
Universitair Medisch Centrum Utrecht
Utrecht
Netherlands
New Zealand Clinical Research
Auckland
New Zealand
New Zealand Clinical Research
Christchurch
New Zealand
Oslo University Hospital - Rikshospitalet
Oslo
Norway
Centro Hospitalar Universitario de Lisboa Norte
Lisbon
Portugal
Centro Hospitalar Universitario do Porto
Porto
Portugal
National Heart Centre
Singapore
Singapore
National University Hospital
Singapore
Singapore
Chonnam National University Hospital
Gwangju
South Korea
Samsung Medical Center
Seoul
South Korea
Seoul National University Hospital
Seoul
South Korea
Severance Hospital, Yonsei University Health System
Seoul
South Korea
Hospital Universitario de A Coruna
A Coruña
Spain
Hospital Universitari de Bellvitge
Barcelona
Spain
Hospital Universitario Vall d'Hebron
Barcelona
Spain
Hospital Universitario Puerta de Hierro
Majadahonda
Spain
Hospital Universitario Virgen de la Victoria
Málaga
Spain
Hospital Universitario Son Llatzer
Palma de Mallorca
Spain
Karolinska University Hospital
Stockholm
Sweden
University Hospital of Umea
Umeå
Sweden
Far Eastern Memorial Hospital
New Taipei City
Taiwan
National Taiwan University Hospital
Taipei
Taiwan
Taipai Veterans General Hospital
Taipei
Taiwan
Synexus Midlands Clinical Research Centre
Birmingham
United Kingdom
Synexus Wales Clinical Research Centre
Cardiff
United Kingdom
Synexus Scotland Clinical Research Centre
Glasgow
United Kingdom
Synexus Hexham Clinical Research Centre
Hexham
United Kingdom
Synexus Merseyside Clinical Research Centre
Liverpool
United Kingdom
Richmond Pharmacology
London
United Kingdom
Synexus Manchester Clinical Research Centre
Manchester
United Kingdom
Participation Criteria
Inclusion Criteria:
* Documented diagnosis of ATTR amyloidosis with cardiomyopathy
* Medical history of heart failure (HF)
* Symptoms of HF are optimally managed and clinically stable within 28 days prior to administration of study intervention
* Screening NT-proBNP, a blood marker of HF severity, greater than or equal to 1000 pg/mL (or greater than or equal to 2000 pg/mL if participant has known atrial fibrillation)
Exclusion Criteria:
* New York Heart Association (NYHA) Class IV HF
* Polyneuropathy Disability score of IV (confined to wheelchair or bed)
* Has hepatitis B, hepatitis C or human immunodeficiency virus (HIV) infection
* History of active malignancy within 3 years prior to screening
* RNA silencer therapy (patisiran, inotersen and/or eplontersen) within 12 months prior to dosing. Any prior vutrisiran use is not allowed
* Initiation of tafamidis within 6 months prior to study dosing
* Estimated glomerular filtration rate (eGFR) \<30 mL/min/1.73m\^2
* Liver failure
* Uncontrolled blood pressure
* Unable or unwilling to take vitamin A supplementation for the duration of the study