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Can a new injection slow a rare heart disease?

NCT ID NCT04136171

Disease control Sponsor: Ionis Pharmaceuticals, Inc. Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed This study
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Aug 25, 2026 · Last updated Sep 04, 2026 · Updated 3 times

Summary

This trial is testing an experimental drug called eplontersen in people with transthyretin-mediated amyloid cardiomyopathy (ATTR CM), a condition where abnormal protein builds up in the heart and leads to heart failure. The study aims to see if the drug, given as a regular injection, can reduce the risk of heart-related death and serious heart events compared to a placebo, while people also receive standard care. About 1,400 participants will be followed for up to 140 weeks to measure outcomes like walking ability and overall heart health.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
Eplontersen, an experimental drug given by injection under the skin
What this could lead to
If successful, this could provide a new treatment option to help people with ATTR amyloid cardiomyopathy live longer and have fewer heart-related hospitalizations.
What could go wrong
This is a large, late-stage trial, but the drug may still fail to show a clear benefit or could cause unexpected side effects. Results will take time to confirm.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

1,438 people

The number who actually took part.

Started

Mar 2020

Finished

Aug 2026

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 to 90 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * Females must be non-pregnant and non-lactating, and either surgically sterile or post-menopausal or abstinent. If engaged in sexual relations of child-bearing potential, agree to use 1 highly effective contraceptive method * 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: * Acute coronary syndrome, unstable angina, stroke, transient ischemic attack (TIA), coronary revascularization, cardiac device implantation, cardiac valve repair, or major surgery within 3 months of Screening * 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.

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Conditions

The condition(s) this trial relates to.

amyloidosis cardiomyopathy

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Transthyretin-mediated amyloid cardiomyopathy (attr CM)

Contacts and locations

Locations

  • A Beneficência Portuguesa de São Paulo - Unidade Mirante

    São Paulo, 01323-030, Brazil

  • Advara HeartCare

    Joondalup, Western Australia, 6027, Australia

  • Advara HeartCare Murdoch

    Murdoch, Western Australia, 6150, Australia

  • Advara Heartcare

    Leabrook, South Australia, 5068, Australia

  • Alexandra General Hospital

    Athens, 11528, Greece

  • Algemeen Ziekenhuis Sint-Jan Brugge-Oostende - Campus Sint-Jan

    Bruges, West-Vlaanderen, 8000, Belgium

  • Altman Clinical and Translational Research Institute Center for Clinical Research

    La Jolla, California, 92037, United States

  • Azienda Ospedale - Università di Padova

    Padua, 35128, Italy

  • Azienda Ospedaliera - Universitaria Sant' Andrea

    Roma, 00189, Italy

  • Azienda Ospedaliero - Universitaria Careggi

    Florence, 50139, Italy

  • Azienda Ospedaliero Universitaria Delle Marche

    Ancona, 60126, Italy

  • Azienda Ospedaliero-Universitaria di Bologna Policlinico Sant Orsola-Malpighi

    Bologna, 40138, Italy

  • Barnes-Jewish Hospital

    St Louis, Missouri, 63110, United States

  • Boston University School of Medicine

    Boston, Massachusetts, 02118, United States

  • Box Hill Hospital

    Box Hill, Victoria, 3128, Australia

  • Brigham and Women's Hospital

    Boston, Massachusetts, 02115, United States

  • Carl and Edyth Lindner Research Center at The Christ Hospital

    Cincinnati, Ohio, 45219, United States

  • Cedars-Sinai Medical Center

    Los Angeles, California, 90048, United States

  • Centre Hospitalier Départemental Vendée

    La Roche-sur-Yon, 85925, France

  • Centre Hospitalier Universitaire De Nantes - Hôpital Nord Laennec

    Saint-Herblain, 44805, France

  • Centre Hospitalier de l'Université de Montréal (CHUM)

    Montreal, Quebec, H2X 0C1, Canada

  • Centro Avançado de Pesquisa e Estudos para o Diagnóstico CAPED

    Ribeirão Preto, 14026-900, Brazil

  • Centro Hospitalar Universitário de Lisboa Central - Hospital de Santa Marta

    Lisbon, 1169-024, Portugal

  • Centro Hospitalar Universitário de São João

    Porto, 4200-319, Portugal

  • Centro Hospitalar Universitário do Porto - Hospital Geral de Santo Antonio

    Porto, 4099-001, Portugal

  • Cleveland Clinic Florida

    Weston, Florida, 33331, United States

  • Cleveland Clinic Main Campus

    Cleveland, Ohio, 44195, United States

  • Columbia University Irving Medical Center

    New York, New York, 10034, United States

  • Complejo Hospitalario Universitario de Santiago

    Santiago de Compostela, 15706, Spain

  • Duke University Medical Center

    Durham, North Carolina, 27710, United States

  • Emory Heart and Vascular Center - Emory Clifton Campus

    Atlanta, Georgia, 30322, United States

  • Fakultní Nemocnice u sv. Anny v Brn

    Brno, South Moravian, 656 91, Czechia

  • Fondazione IRCCS Policlinico San Matteo

    Pavia, 27100, Italy

  • Fondazione Toscana Gabriele Monasterio per la Ricerca Medica e di Sanita Pubblica

    Pisa, 56126, Italy

  • Froedtert & Medical College of Wisconsin

    Milwaukee, Wisconsin, 53226, United States

  • Gordon and Leslie Diamond Health Care Centre

    Vancouver, British Columbia, V5Z 1M9, Canada

  • Hadassah University Hospital Ein Kerem

    Jerusalem, 9112001, Israel

  • Hamamatsu University Hospital

    Hamamatsu, Shizuoka, 431-3192, Japan

  • Henry Ford Health System

    Detroit, Michigan, 48202, United States

  • Hospital Clínico Universitario Virgen de la Arrixaca

    Murcia, 30120, Spain

  • Hospital Italiano de Buenos Aires

    Ciudad Autónoma de Buenos Aires, Buenos Aires, C1199ABB, Argentina

  • Hospital Moinhos de Vento

    Porto Alegre, 90560-030, Brazil

  • Hospital Universitari Vall d'Hebrón

    Barcelona, 08035, Spain

  • Hospital Universitario Puerta de Hierro - Majadahonda

    Majadahonda, 28222, Spain

  • Hospital da Senhora da Oliveira - Guimarães

    Guimarães, 4835-044, Portugal

  • Hospital das Clínicas da Faculdade de Medicina de Ribeirão Preto

    Ribeirão Preto, 14040-030, Brazil

  • Hospital of the University of Occupational and Environmental Health

    Kitakyushu-shi, Fukuoka, 807-8555, Japan

  • Houston Methodist Hospital

    Houston, Texas, 77030, United States

  • Hôpital Haut-Lévêque

    Pessac, 33604, France

  • Hôpital Rangueil

    Toulouse, Occitanie, 31059, France

  • Hôpital Regional de Rimouski

    Rimouski, Quebec, G5L 5T1, Canada

  • Hôpital de la Timone

    Marseille, 13005, France

  • Hôpitaux Universitaires Henri Mondor

    Créteil, 94010, France

  • Indiana University Health

    Indianapolis, Indiana, 46202, United States

  • Institut Klinické a Experimentální Medicíny

    Prague, 140 21, Czechia

  • Instituto Cardiovascular de Buenos Aires

    Ciudad Autonoma de Buenos Aires, Buenos Aires, C1428ART, Argentina

  • Instituto Dante Pazzanese de Cardiologia

    São Paulo, 04012-909, Brazil

  • Instituto do Coração de São Paulo

    São Paulo, 05403-000, Brazil

  • Kaplan Medical Center

    Rehovot, Israel

  • Keio University Hospital

    Shinjuku-Ku, Tokyo, 160-8582, Japan

  • Krakowski Szpital Specjalistyczny im. Jana Pawa II

    Krakow, 31-202, Poland

  • Kumamoto University Hospital

    Kumamoto, 860-8556, Japan

  • Kurume University Hospital

    Kurume-shi, Fukuoka, 830-0011, Japan

  • Lancaster General Hospital

    Lancaster, Pennsylvania, 17602, United States

  • Laurelton Heart Specialists

    Rosedale, New York, 11422, United States

  • Liverpool Hospital

    Liverpool, New South Wales, 2170, Australia

  • Loyola University Medical Center

    Maywood, Illinois, 60153, United States

  • Massachusetts General Hospital

    Boston, Massachusetts, 02114, United States

  • Mayo Clinic - Arizona

    Phoenix, Arizona, 85054, United States

  • Mayo Clinic - Rochester

    Rochester, Minnesota, 55905, United States

  • MedStar Washington Hospital Center

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

  • Medical University of South Carolina

    Charleston, South Carolina, 29425, United States

  • Medizinische Universität Graz

    Graz, Styria, 8036, Austria

  • Medizinische Universität Innsbruck

    Innsbruck, Tyrol, 6020, Austria

  • Medizinische Universität Wien

    Vienna, 1090, Austria

  • Minneapolis Heart Institute

    Minneapolis, Minnesota, 55407, United States

  • Narodowy Instytut Kardiologii Stefana kardyna Wyszyskiego - Pastwowy Instytut Badawczy

    Warsaw, 04-628, Poland

  • New York University Langone Cardiology Associates

    New York, New York, 10016, United States

  • Northwestern University

    Chicago, Illinois, 60611, United States

  • Ochsner Health System

    New Orleans, Louisiana, 70121, United States

  • Odense Universitetshospital

    Odense, Region Syddanmark, 5000, Denmark

  • Okayama University Hospital

    Okayama, Okayama-ken, 700-8558, Japan

  • Oregon Health and Science University

    Portland, Oregon, 97239, United States

  • Osaka Metropolitan University Hospital

    Osaka, Osaka, 545-8586, Japan

  • Penn Presbyterian Medical Center

    Philadelphia, Pennsylvania, 19104, United States

  • Piedmont Heart of Fayetteville

    Atlanta, Georgia, 30309, United States

  • Piedmont Heart of Fayetteville

    Fayetteville, Georgia, 30214, United States

  • Princess Alexandra Hospital

    Woolloongabba, Queensland, 4102, Australia

  • Rambam Health Care Campus

    Haifa, 3109601, Israel

  • Richmond Pharmacology

    London, SE1 1YR, United Kingdom

  • Rigshospitalet

    Copenhagen, Capital Region, 2100, Denmark

  • Royal Free London NHS Foundation Trust

    London, HR3M+6R, United Kingdom

  • Royal Hobart Hospital

    Hobart, Tasmania, 7000, Australia

  • Sahlgrenska Universitetssjukhuset

    Gothenburg, 413 45, Sweden

  • Saint Luke's Hospital of Kansas City

    Kansas City, Missouri, 64111, United States

  • Saiseikai Fukuoka General Hospital

    Fukuoka, Hukuoka, 810-0001, Japan

  • Sapporo Medical University Hospital

    Sapporo, Hokkaido, 060-8543, Japan

  • Shinshu University Hospital

    Matsumoto, Nagano, 390-8621, Japan

  • Skellefteå lasarett

    Skellefteå, 931 86, Sweden

  • Stanford Hospital

    Stanford, California, 94305, United States

  • Synexus - Manchester Clinical Research Centre

    Liverpool, L22 0LG, United Kingdom

  • Synexus - Merseyside Clinical Research Centre

    Liverpool, L22 0LG, United Kingdom

  • Synexus - North Tees Clinical Research Centre

    Hexham, TS19 8PE, United Kingdom

  • Synexus - Scotland Clinical Research Centre

    Glasgow, Scotland, G20 0SP, United Kingdom

  • Synexus - Wales

    Cardiff, Wales, CF15 9SS, United Kingdom

  • Synexus Midlands Clinical Research Centre

    Edgbaston, B15 2SQ, United Kingdom

  • The George Washington Medical Faculty Associates - Foggy Bottom North Pavilion

    Washington D.C., District of Columbia, 20037, United States

  • The Ohio State University College of Medicine

    Columbus, Ohio, 43210, United States

  • The University of Chicago Medical Center

    Chicago, Illinois, 60637, United States

  • The University of North Carolina at Chapel Hill

    Chapel Hill, North Carolina, 27599, United States

  • The Westmead Institute for Medical Research

    Westmead, New South Wales, NSW 2145, Australia

  • Toronto General Hospital

    Toronto, Ontario, M5G 2M9, Canada

  • Tulane University Heart and Vascular Institute

    New Orleans, Louisiana, 70112, United States

  • Uniklinik Koln

    Cologne, 50937, Germany

  • Universidade Estadual de Campinas

    Campinas, 13083881, Brazil

  • Universitaetsklinikum Heidelberg - Zentrum fur Innere Medizin

    Heidelberg, 69120, Germany

  • University Hospital - London Health Sciences Centre

    London, Ontario, N6A 5A5, Canada

  • University Hospital Kyoto Prefectural University of Medicine

    Kyoto, Kyoto, 602-8566, Japan

  • University Hospitals Cleveland Medical Center

    Cleveland, Ohio, 44106, United States

  • University of Calgary

    Calgary, Alberta, T2N 4Z6, Canada

  • University of California, San Francisco (UCSF) - Medical Center

    San Francisco, California, 94143, United States

  • University of Colorado Hospital - Anschutz Medical Campus

    Aurora, Colorado, 80045, United States

  • University of Kansas Medical Center

    Kansas City, Kansas, 66160, United States

  • University of Maryland Medical Center

    Baltimore, Maryland, 21201, United States

  • University of Pittsburgh Medical School

    Pittsburgh, Pennsylvania, 15261, United States

  • University of Texas Southwestern Medical Center

    Dallas, Texas, 75390, United States

  • University of Utah

    Salt Lake City, Utah, 84112, United States

  • University of Washington

    Seattle, Washington, 98195, United States

  • University of Wisconsin - Madison

    Madison, Wisconsin, 53792, United States

  • Universitätsklinikum Münster

    Münster, 48149, Germany

  • Universitätsklinikum Würzburg

    Würzburg, 97078, Germany

  • Universitätsklinikum des Saarlandes

    Homburg, Saarland, 66421, Germany

  • Uwajima City Hospital

    Uwajima-shi, Ehime, 798-8510, Japan

  • Vanderbilt University Medical Center

    Nashville, Tennessee, 37232, United States

  • Veterans Affairs Caribbean Healthcare System

    San Juan, 00921, Puerto Rico

  • Virginia Commonwealth University (VCU)

    Richmond, Virginia, 23298, United States

  • Vseobecna Fakultni Nemocnice v Praze

    Prague, 128 08, Czechia

  • Weill Cornell Medicine Cardiology

    New York, New York, 10065, United States

  • Ziekenhuis Oost-Limburg - Campus Sint-Jan

    Genk, Limburg, 3600, Belgium

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