CARDIO-TTRansform: A Study to Evaluate the Efficacy and Safety of Eplontersen (Formerly Known as ION-682884, IONIS-TTR-LRx and AKCEA-TTR-LRx) in Participants With Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM)
NCT ID: NCT04136171
Last Updated: 2025-08-08
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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ACTIVE_NOT_RECRUITING
PHASE3
1438 participants
INTERVENTIONAL
2020-03-13
2026-08-31
Brief Summary
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Detailed Description
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Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
DOUBLE
Study Groups
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Eplontersen
Eplontersen by subcutaneous injection once every 4 weeks
Eplontersen
Eplontersen by subcutaneous injection
Placebo
Eplontersen-matching placebo by subcutaneous injection once every 4 weeks
Placebo
Eplontersen-matching placebo by subcutaneous injection
Interventions
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Eplontersen
Eplontersen by subcutaneous injection
Placebo
Eplontersen-matching placebo by subcutaneous injection
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* Males must be surgically sterile or, abstinent or, if engaged in sexual relations with a woman of child-bearing potential, the participant or the participant's non-pregnant female partner must be using a highly effective contraceptive method
* Amyloid deposits in cardiac or non-cardiac tissue confirmed by Congo Red (or equivalent) staining OR technetium scintigraphy (99mTc -3,3-diphosphono-1,2- propanodicarboxylic acid \[DPD-Tc\], 99m Tc-pyrophosphate \[PYP-Tc\], or 99m Tc-hydroxymethylene-diphosphonate \[HMDP-Tc\]) with Grade 2 or 3 cardiac uptake in the absence of abnormal light chains ratio, centrally confirmed
* End-diastolic interventricular septum thickness of \> 12 millimeters (mm) on Screening echocardiogram
* New York Heart Association (NYHA) class I-III
Exclusion Criteria
* Cardiomyopathy not primarily caused by ATTR-CM, for example, cardiomyopathy due to hypertension, valvular heart disease, or ischemic heart disease
* Monoclonal gammopathy of undetermined significance (MGUS) and/or alterations in immunoglobulin free light chain (FLC) ratio unless fat, bone marrow, or heart biopsy confirming the absence of light chain and the presence of TTR protein by mass spectrometry or immunoelectron microscopy. For participants with chronic kidney disease (CKD) and without presence of monoclonal protein in blood and urine, the acceptable FLC ratio is 0.26-2.25. Results different from that may be discussed with local hematologist, Investigator and Medical Monitor if the risks associated with the biopsy outweigh the benefits
* Prior liver or heart transplant, and/or Left Ventricular Assist Device (LVAD) or anticipated liver transplant or LVAD within 1 year after randomization
* Current or previous treatment with Tegsedi™ (inotersen) or Onpattro™ (patisiran) or other oligonucleotide or ribonucleic acid (RNA) therapeutic (including small interfering ribonucleic acid \[siRNA\]; does not apply to COVID-19 mitochondrial \[mRNA\] vaccinations)
* Current treatment with diflunisal, doxycycline, with or without ursodeoxycholic acid, and/or non-dihydropyridine calcium-channel blocker (e.g., verapamil, diltiazem). Participants receiving any of these agents must respect a wash-out period of 14 days before randomization.
18 Years
90 Years
ALL
No
Sponsors
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AstraZeneca
INDUSTRY
Ionis Pharmaceuticals, Inc.
INDUSTRY
Responsible Party
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Locations
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Mayo Clinic - Arizona
Phoenix, Arizona, United States
Altman Clinical and Translational Research Institute Center for Clinical Research
La Jolla, California, United States
Cedars-Sinai Medical Center
Los Angeles, California, United States
University of California, San Francisco (UCSF) - Medical Center
San Francisco, California, United States
Stanford Hospital
Stanford, California, United States
University of Colorado Hospital - Anschutz Medical Campus
Aurora, Colorado, United States
MedStar Washington Hospital Center
Washington D.C., District of Columbia, United States
The George Washington Medical Faculty Associates - Foggy Bottom North Pavilion
Washington D.C., District of Columbia, United States
Cleveland Clinic Florida
Weston, Florida, United States
Emory Heart and Vascular Center - Emory Clifton Campus
Atlanta, Georgia, United States
Piedmont Heart of Fayetteville
Fayetteville, Georgia, United States
Northwestern University
Chicago, Illinois, United States
The University of Chicago Medical Center
Chicago, Illinois, United States
Loyola University Medical Center
Maywood, Illinois, United States
Indiana University Health
Indianapolis, Indiana, United States
University of Kansas Medical Center
Kansas City, Kansas, United States
Louisiana State University Health Sciences Center
New Orleans, Louisiana, United States
Tulane University Heart and Vascular Institute
New Orleans, Louisiana, United States
Ochsner Health System
New Orleans, Louisiana, United States
University of Maryland Medical Center
Baltimore, Maryland, United States
Massachusetts General Hospital
Boston, Massachusetts, United States
Brigham and Women's Hospital
Boston, Massachusetts, United States
Boston University School of Medicine
Boston, Massachusetts, United States
Henry Ford Health System
Detroit, Michigan, United States
Minneapolis Heart Institute
Minneapolis, Minnesota, United States
Mayo Clinic - Rochester
Rochester, Minnesota, United States
Saint Luke's Hospital of Kansas City
Kansas City, Missouri, United States
Barnes-Jewish Hospital
St Louis, Missouri, United States
New York University Langone Cardiology Associates
New York, New York, United States
Columbia University Irving Medical Center
New York, New York, United States
Weill Cornell Medicine Cardiology
New York, New York, United States
Laurelton Heart Specialists
Rosedale, New York, United States
The University of North Carolina at Chapel Hill
Chapel Hill, North Carolina, United States
Duke University Medical Center
Durham, North Carolina, United States
Carl and Edyth Lindner Research Center at The Christ Hospital
Cincinnati, Ohio, United States
University Hospitals Cleveland Medical Center
Cleveland, Ohio, United States
Cleveland Clinic Main Campus
Cleveland, Ohio, United States
The Ohio State University College of Medicine
Columbus, Ohio, United States
Oregon Health and Science University
Portland, Oregon, United States
Lancaster General Hospital
Lancaster, Pennsylvania, United States
Penn Presbyterian Medical Center
Philadelphia, Pennsylvania, United States
University of Pittsburgh Medical School
Pittsburgh, Pennsylvania, United States
Medical University of South Carolina
Charleston, South Carolina, United States
Vanderbilt University Medical Center
Nashville, Tennessee, United States
University of Texas Southwestern Medical Center
Dallas, Texas, United States
Houston Methodist Hospital
Houston, Texas, United States
University of Utah
Salt Lake City, Utah, United States
Virginia Commonwealth University (VCU)
Richmond, Virginia, United States
University of Washington
Seattle, Washington, United States
University of Wisconsin - Madison
Madison, Wisconsin, United States
Froedtert & Medical College of Wisconsin
Milwaukee, Wisconsin, United States
Instituto Cardiovascular de Buenos Aires
Ciudad Autonoma de Buenos Aires, Buenos Aires, Argentina
Hospital Italiano de Buenos Aires
Ciudad Autónoma de Buenos Aires, Buenos Aires, Argentina
Liverpool Hospital
Liverpool, New South Wales, Australia
The Westmead Institute for Medical Research
Westmead, New South Wales, Australia
Princess Alexandra Hospital
Woolloongabba, Queensland, Australia
Advara Heartcare
Leabrook, South Australia, Australia
Royal Hobart Hospital
Hobart, Tasmania, Australia
Box Hill Hospital
Box Hill, Victoria, Australia
Advara HeartCare
Joondalup, Western Australia, Australia
Advara HeartCare Murdoch
Murdoch, Western Australia, Australia
Medizinische Universität Graz
Graz, Styria, Austria
Medizinische Universität Innsbruck
Innsbruck, Tyrol, Austria
Medizinische Universität Wien
Vienna, , Austria
Ziekenhuis Oost-Limburg - Campus Sint-Jan
Genk, Limburg, Belgium
Algemeen Ziekenhuis Sint-Jan Brugge-Oostende - Campus Sint-Jan
Bruges, West-Vlaanderen, Belgium
Universidade Estadual de Campinas
Campinas, , Brazil
Hospital Moinhos de Vento
Porto Alegre, , Brazil
Centro Avançado de Pesquisa e Estudos para o Diagnóstico CAPED
Ribeirão Preto, , Brazil
Hospital das Clínicas da Faculdade de Medicina de Ribeirão Preto
Ribeirão Preto, , Brazil
A Beneficência Portuguesa de São Paulo - Unidade Mirante
São Paulo, , Brazil
Instituto Dante Pazzanese de Cardiologia
São Paulo, , Brazil
Instituto do Coração de São Paulo
São Paulo, , Brazil
University of Calgary
Calgary, Alberta, Canada
Gordon and Leslie Diamond Health Care Centre
Vancouver, British Columbia, Canada
University Hospital - London Health Sciences Centre
London, Ontario, Canada
Toronto General Hospital
Toronto, Ontario, Canada
Centre Hospitalier de l'Université de Montréal (CHUM)
Montreal, Quebec, Canada
Hôpital Regional de Rimouski
Rimouski, Quebec, Canada
Fakultní Nemocnice u sv. Anny v Brn
Brno, South Moravian, Czechia
Vseobecna Fakultni Nemocnice v Praze
Prague, , Czechia
Institut Klinické a Experimentální Medicíny
Prague, , Czechia
Rigshospitalet
Copenhagen, Capital Region, Denmark
Odense Universitetshospital
Odense, Region Syddanmark, Denmark
Hôpital Rangueil
Toulouse, Occitanie, France
Hôpitaux Universitaires Henri Mondor
Créteil, , France
Centre Hospitalier Départemental Vendée
La Roche-sur-Yon, , France
Hôpital de la Timone
Marseille, , France
Hôpital Haut-Lévêque
Pessac, , France
Centre Hospitalier Universitaire De Nantes - Hôpital Nord Laennec
Saint-Herblain, , France
Universitätsklinikum des Saarlandes
Homburg, Saarland, Germany
Charité Campus Mitte
Berlin, , Germany
Charité Universitätsmedizin Berlin
Berlin, , Germany
Uniklinik Koln
Cologne, , Germany
Universitaetsklinikum Heidelberg - Zentrum fur Innere Medizin
Heidelberg, , Germany
Universitätsklinikum Münster
Münster, , Germany
Universitätsklinikum Würzburg
Würzburg, , Germany
Alexandra General Hospital
Athens, , Greece
Rambam Health Care Campus
Haifa, , Israel
Hadassah University Hospital Ein Kerem
Jerusalem, , Israel
Kaplan Medical Center
Rehovot, , Israel
Azienda Ospedaliero Universitaria Delle Marche
Ancona, , Italy
Azienda Ospedaliero-Universitaria di Bologna Policlinico Sant Orsola-Malpighi
Bologna, , Italy
Azienda Ospedaliero - Universitaria Careggi
Florence, , Italy
Azienda Ospedale - Università di Padova
Padua, , Italy
Fondazione IRCCS Policlinico San Matteo
Pavia, , Italy
Fondazione Toscana Gabriele Monasterio per la Ricerca Medica e di Sanita Pubblica
Pisa, , Italy
Azienda Ospedaliera - Universitaria Sant' Andrea
Roma, , Italy
Uwajima City Hospital
Uwajima-shi, Ehime, Japan
Hospital of the University of Occupational and Environmental Health
Kitakyushu-shi, Fukuoka, Japan
Kurume University Hospital
Kurume-shi, Fukuoka, Japan
Sapporo Medical University Hospital
Sapporo, Hokkaido, Japan
Saiseikai Fukuoka General Hospital
Fukuoka, Hukuoka, Japan
Kokura Memorial Hospital
Kitakyushu, Hukuoka, Japan
University Hospital Kyoto Prefectural University of Medicine
Kyoto, Kyoto, Japan
Shinshu University Hospital
Matsumoto, Nagano, Japan
Okayama University Hospital
Okayama, Okayama-ken, Japan
Osaka Metropolitan University Hospital
Osaka, Osaka, Japan
Hamamatsu University Hospital
Hamamatsu, Shizuoka, Japan
Keio University Hospital
Shinjuku-Ku, Tokyo, Japan
Kumamoto University Hospital
Kumamoto, , Japan
Krakowski Szpital Specjalistyczny im. Jana Pawa II
Krakow, , Poland
Narodowy Instytut Kardiologii Stefana kardyna Wyszyskiego - Pastwowy Instytut Badawczy
Warsaw, , Poland
Hospital da Senhora da Oliveira - Guimarães
Guimarães, , Portugal
Centro Hospitalar Universitário de Lisboa Central - Hospital de Santa Marta
Lisbon, , Portugal
Centro Hospitalar Universitário do Porto - Hospital Geral de Santo Antonio
Porto, , Portugal
Centro Hospitalar Universitário de São João
Porto, , Portugal
Veterans Affairs Caribbean Healthcare System
San Juan, , Puerto Rico
Hospital Universitari Vall d'Hebrón
Barcelona, , Spain
Hospital Universitario Puerta de Hierro - Majadahonda
Majadahonda, , Spain
Hospital Clínico Universitario Virgen de la Arrixaca
Murcia, , Spain
Complejo Hospitalario Universitario de Santiago
Santiago de Compostela, , Spain
Sahlgrenska Universitetssjukhuset
Gothenburg, , Sweden
Skellefteå lasarett
Skellefteå, , Sweden
Synexus - Scotland Clinical Research Centre
Glasgow, Scotland, United Kingdom
Synexus - Wales
Cardiff, Wales, United Kingdom
Synexus Midlands Clinical Research Centre
Edgbaston, , United Kingdom
Synexus - North Tees Clinical Research Centre
Hexham, , United Kingdom
Synexus - Manchester Clinical Research Centre
Liverpool, , United Kingdom
Synexus - Merseyside Clinical Research Centre
Liverpool, , United Kingdom
Royal Free London NHS Foundation Trust
London, , United Kingdom
Richmond Pharmacology
London, , United Kingdom
Countries
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Other Identifiers
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2019-002835-27
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
2024-514434-20-00
Identifier Type: CTIS
Identifier Source: secondary_id
ION-682884-CS2
Identifier Type: -
Identifier Source: org_study_id
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