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New drug eplontersen tested for Long-Term safety in heart disease patients

NCT ID NCT05667493

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 This study
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
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study is checking the long-term safety of a drug called eplontersen in people with ATTR-CM, a heart condition caused by abnormal protein buildup. About 1400 adults who completed earlier studies will receive eplontersen injections. Researchers will monitor side effects, kidney function, and blood counts over time.

What this could mean

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

Active substance
eplontersen
What this could lead to
If successful, this could confirm that eplontersen is safe for long-term use and may help control ATTR-CM, a serious heart condition.
What could go wrong
This is an extension study focused on safety, not on proving the drug works. Side effects like low platelets or kidney issues are possible. Results may not apply to all patients.

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

About 1,400 people

The number the study aims to enrol. It can still change while the study runs.

Started

Nov 2022

Expected to finish

Aug 2029

An estimate. End dates often move.

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 years and older

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: 1. Satisfactory completion of Treatment Period and the End of Treatment Visit of the Index Study (ION-682884-CS2) OR diagnosis of ATTR-CM and satisfactory participation on ISIS 420915- CS101 study as judged by the Investigator and Sponsor. 2. Investigator is willing to treat the participant with open-label eplontersen. 3. Willingness to adhere to vitamin A supplementation per protocol. Exclusion Criteria: 1. Permanently discontinued study drug administration while participating in the Index Study (ION 682884-CS2) or IST (ISIS 420915-CS101 Study). 2. Have any new condition or worsening of an existing condition that in the opinion of the Investigator or Sponsor would make the participant unsuitable for enrolment, or which could interfere with the participant participating in or completing the study, including the need for treatment with medications disallowed in the Index Study.

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Get notified about this study

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Conditions

The condition(s) this trial relates to.

amyloidosis wild type ATTR amyloidosis

As listed by the trial registrant

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

Transthyretin-mediated amyloid cardiomyopathy (attr-CM)

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The places running it

    134 sites in 20 countries. The list below names each one and where it is.

  2. The official record

    The full official record for this study. This one lists no contact details, but it is the first place any would appear.

    Open the record ↗

  3. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

  • A Beneficência Portuguesa de São Paulo - Unidade Mirante

    São Paulo, 01323-030, Brazil

  • AES - Synexus - Midlands

    Edgbaston, England, B15 2SQ, United Kingdom

  • AES - Synexus - Scotland Clinical Research Centre

    Bellshill, England, ML4 3NJ, United Kingdom

  • AES - Synexus - Wales

    Cardiff, Wales, CF15 9SS, United Kingdom

  • Advara HeartCare

    Joondalup, Western Australia, 6027, Australia

  • Advara HeartCare - Joondalup

    Joondalup, 6027, Australia

  • Advara HeartCare Murdoch

    Murdoch, Western Australia, 6150, Australia

  • Advara HeartCare Murdoch

    Murdoch, 6150, Australia

  • Advara Heartcare Leabrook

    Leabrook, 5068, Australia

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

    Bruges, 8000, Belgium

  • Allina Health Minneapolis Heart Institute - Abbott Northwestern's Heart Hospital

    Minneapolis, Minnesota, 55407, United States

  • Altman Clinical and Translational Research Institute - Center for Clinical Research

    La Jolla, California, 92037, United States

  • Azienda Ospedale Università di Padova

    Padova, 35128, Italy

  • Azienda Ospedaliera - Universitaria Sant' Andrea

    Roma, 00189, Italy

  • Azienda Ospedaliero - Universitaria Careggi

    Florence, 50134, Italy

  • Azienda Ospedaliero Universitaria Delle Marche

    Ancona, 60126, Italy

  • Barnes-Jewish Hospital

    St Louis, Missouri, 63110, United States

  • Boston University School of Medicine

    Boston, Massachusetts, 02118, United States

  • Box Hill Hospital

    Clayton, 3168, 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

    Beverly Hills, California, 90211, United States

  • Centre Hospitalier Départemental Vendée

    La Roche-sur-Yon, 85925, France

  • Centre Hospitalier Universitaire De Nantes - Hôpital Nord Laennec

    Nantes, 44093, France

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

    Montreal, Quebec, H2X 0C1, Canada

  • Centro Avançado de Pesquisa e Estudos para o Diagnóstico CAPED

    São Paulo, 14026-900, Brazil

  • 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 (CHUS)

    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 Brně

    Brno, 656 91, Czechia

  • Fondazione IRCCS Policlinico San Matteo

    Pavia, 27100, Italy

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

    Pisa, 56124, Italy

  • General Hospital of Athens "Alexandra"

    Athens, 115 28, Greece

  • Gordon and Leslie Diamond Health Care Centre

    Vancouver, V5Z 1M9, Canada

  • Hadassah University Hospital Ein Kerem

    Jerusalem, 9112001, Israel

  • Hamamatsu University Hospital

    Hamamatsu, 4313192, 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

    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, 28222, Spain

  • Hospital da Senhora da Oliveira

    Guimarães, 4835-044, Portugal

  • Hospital das Clínicas da Faculdade de Medicina de Ribeirão Preto

    Ribeirão Preto, 14040-030, Brazil

  • Houston Methodist Hospital

    Houston, Texas, 77030, United States

  • Hôpital Haut-Lévêque

    Pessac, 33604, France

  • Hôpital Rangueil

    Toulouse, 31059, France

  • Hôpital Regional de Rimouski

    Rimouski, G5L 5T1, Canada

  • Hôpital de la Timone

    Marseille, 13005, France

  • Hôpitaux Universitaires Henri Mondor

    Créteil, 94010, France

  • Indiana University Health University Hospital

    Indianapolis, Indiana, 46202, United States

  • Institut Klinické a Experimentální Medicíny

    Prague, 140 21, Czechia

  • Instituto Cardiovascular 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, 7661041, Israel

  • Keio University Hospital

    Shinjuku-Ku, 160-8582, Japan

  • Krakowski Szpital Specjalistyczny im. Jana Pawła II

    Krakow, 31- 202, Poland

  • Kumamoto University Hospital

    Kumamoto, 860-8556, Japan

  • Kurume University Hospital

    Kurume-Shi, Fukuoka, 830-0011, Japan

  • Kurume University Hospital

    Kurume, 830-0011, Japan

  • Lancaster General Hospital

    Lancaster, Pennsylvania, 17602, United States

  • Liverpool Hospital

    Liverpool, 2170, Australia

  • Loyola University Medical Center

    Maywood, Illinois, 60153, United States

  • Massachusetts General Hospital

    Boston, Massachusetts, 02114, United States

  • Mayo Clinic

    Scottsdale, Arizona, 85259, United States

  • Mayo Clinic

    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, 8036, Austria

  • Medizinische Universität Innsbruck

    Innsbruck, 6020, Austria

  • Medizinische Universität Wien

    Vienna, 1090, Austria

  • New York University Langone Cardiology Associates

    New York, New York, 10016, United States

  • Northwestern University

    Chicago, Illinois, 60611, United States

  • Okayama University Hospital

    Okayama, 700-8558, Japan

  • Oregon Health and Science University

    Portland, Oregon, 97239, United States

  • Osaka Metropolitan University Hospital

    Osaka, 545-8586, Japan

  • Penn Presbyterian Medical Center

    Philadelphia, Pennsylvania, 19104, United States

  • Piedmont Atlanta Hospital

    Fayetteville, Georgia, 30214, United States

  • Piedmont Heart of Fayetteville

    Atlanta, Georgia, 30309, United States

  • Princess Alexandra Hospital

    Woolloongabba, 4102, Australia

  • Rambam Health Care Campus

    Haifa, 3109601, Israel

  • Richmond Pharmacology

    London, SE1 1YR, United Kingdom

  • Rigshospitalet

    Copenhagen, 2100, Denmark

  • Royal Free London NHS Foundation Trust

    Hampstead, NW3 2PF, United Kingdom

  • Royal Hobart Hospital

    Hobart, 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 [Fukuoka], 810-0001, Japan

  • Saiseikai Fukuoka General Hospital

    Fukuoka, 810-0001, Japan

  • Sapporo Medical University Hospital

    Sapporo, 060-8543, Japan

  • Shinshu University Hospital

    Matsumoto, 390-8621, Japan

  • Skellefteå lasarett

    Skellefteå, 931 86, Sweden

  • Stanford University (Leland Stanford Junior University)

    Stanford, California, 94305, United States

  • Synexus - Manchester Clinical Research Centre

    Manchester, England, M15 6SE, United Kingdom

  • Synexus - Merseyside Clinical Research Centre

    Liverpool, L22 0LG, United Kingdom

  • Synexus - Merseyside Clinical Research Centre

    Manchester, M15 6SE, 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

  • Toronto General Hospital

    Toronto, Ontario, M5G 2C4, Canada

  • Unidade Local de Saúde de Santo António, E. P. E - Hospital Geral de Santo Antonio

    Porto, 4099-001, Portugal

  • Unidade Local de Saúde de São José, E. P. E - Hospital de Santa Marta

    Lisbon, 1169-024, Portugal

  • Unidade Local de Saúde de São João, EPE

    Porto, 4200-319, Portugal

  • Unidade Local de Saúde do Alto Ave, E. P. E (Hospital da Senhora da Oliveira Guimarães)

    Guimarães, 4835-044, Portugal

  • Uniklinik Köln

    Cologne, 50937, Germany

  • Universidade Estadual de Campinas

    São Paulo, 13083881, Brazil

  • Universitaetsklinikum Heidelberg - Zentrum für Innere Medizin

    Heidelberg, 69120, Germany

  • University Hospital - London Health Sciences Centre

    London, Ontario, N6A 5A5, Canada

  • University Hospitals Cleveland Medical Center

    Cleveland, Ohio, 44106, United States

  • University Of California San Francisco Urology Practice

    San Francisco, California, 94143, United States

  • University of Calgary

    Calgary, Alberta, T2N 4Z6, Canada

  • 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 Kansas Medical Center (KUMC)

    Kansas City, Kansas, 66160, United States

  • University of Maryland Medical Center

    Baltimore, Maryland, 21201, United States

  • University of North Carolina at Chapel Hill

    Chapel Hill, North Carolina, 27599, 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, 66421, Germany

  • Vanderbilt Heart and Vascular Institute

    Nashville, Tennessee, 37232, United States

  • Virginia Commonwealth University

    Richmond, Virginia, 23298, United States

  • Vseobecna Fakultni Nemocnice v Praze

    Prague, 116 36, Czechia

  • Weill Cornell Medicine Cardiology

    New York, New York, 10021, United States

  • Westmead Hospital

    Westmead, 2145, Australia

  • Ziekenhuis Oost-Limburg - Campus Sint-Jan

    Limbourg, 3600, Belgium

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