An Extension Study to Assess Long-Term Safety of Eplontersen in Adults With Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR-CM)

NCT ID: NCT05667493

Last Updated: 2025-12-12

Study Results

Results pending

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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Recruitment Status

ENROLLING_BY_INVITATION

Clinical Phase

PHASE3

Total Enrollment

1400 participants

Study Classification

INTERVENTIONAL

Study Start Date

2022-11-30

Study Completion Date

2029-08-31

Brief Summary

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The purpose of this study is to evaluate the safety and tolerability of extended dosing with eplontersen in participants with ATTR-CM.

Detailed Description

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This is a multicenter, open-label extension, Phase 3 study in up to approximately 1400 participants from the 682884-CS2 study. Eligible participants will receive eplontersen once every 4 weeks for up to 36 months or until after eplontersen is approved and available in the site's country, whichever occurs first. Participants will also receive daily supplemental doses of the recommended daily allowance (RDA) of vitamin A.

Conditions

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Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM)

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Eplontersen

Eplontersen will be administered once every month by sub-cutaneous (SC) injection for up to 36 months or 6 months after eplontersen is approved and available in the site's country, whichever occurs first.

Group Type EXPERIMENTAL

Eplontersen

Intervention Type DRUG

Eplontersen will be administered by SC injection.

Interventions

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Eplontersen

Eplontersen will be administered by SC injection.

Intervention Type DRUG

Other Intervention Names

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ION-682884 IONIS-TTR-LRx

Eligibility Criteria

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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.
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Ionis Pharmaceuticals, Inc.

INDUSTRY

Sponsor Role lead

AstraZeneca

INDUSTRY

Sponsor Role collaborator

Responsible Party

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Responsibility Role SPONSOR

Locations

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Mayo Clinic

Scottsdale, Arizona, United States

Site Status

Cedars-Sinai Medical Center

Beverly Hills, California, United States

Site Status

Altman Clinical and Translational Research Institute - Center for Clinical Research

La Jolla, California, United States

Site Status

University Of California San Francisco Urology Practice

San Francisco, California, United States

Site Status

Stanford University (Leland Stanford Junior University)

Stanford, California, United States

Site Status

University of Colorado Hospital - Anschutz Medical Campus

Aurora, Colorado, United States

Site Status

MedStar Washington Hospital Center

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

Site Status

The George Washington Medical Faculty Associates - Foggy Bottom North Pavilion

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

Site Status

Cleveland Clinic Florida

Weston, Florida, United States

Site Status

Piedmont Heart of Fayetteville

Atlanta, Georgia, United States

Site Status

Emory Heart and Vascular Center - Emory Clifton Campus

Atlanta, Georgia, United States

Site Status

Piedmont Atlanta Hospital

Fayetteville, Georgia, United States

Site Status

Northwestern University

Chicago, Illinois, United States

Site Status

Loyola University Medical Center

Maywood, Illinois, United States

Site Status

Indiana University Health University Hospital

Indianapolis, Indiana, United States

Site Status

University of Kansas Medical Center (KUMC)

Kansas City, Kansas, United States

Site Status

University of Kansas Medical Center

Kansas City, Kansas, United States

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University of Maryland Medical Center

Baltimore, Maryland, United States

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Massachusetts General Hospital

Boston, Massachusetts, United States

Site Status

Brigham and Women's Hospital

Boston, Massachusetts, United States

Site Status

Boston University School of Medicine

Boston, Massachusetts, United States

Site Status

Henry Ford Health System

Detroit, Michigan, United States

Site Status

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

Minneapolis, Minnesota, United States

Site Status

Mayo Clinic

Rochester, Minnesota, United States

Site Status

Saint Luke's Hospital of Kansas City

Kansas City, Missouri, United States

Site Status

Barnes-Jewish Hospital

St Louis, Missouri, United States

Site Status

New York University Langone Cardiology Associates

New York, New York, United States

Site Status

Weill Cornell Medicine Cardiology

New York, New York, United States

Site Status

Columbia University Irving Medical Center

New York, New York, United States

Site Status

University of North Carolina at Chapel Hill

Chapel Hill, North Carolina, United States

Site Status

Duke University Medical Center

Durham, North Carolina, United States

Site Status

Carl and Edyth Lindner Research Center at The Christ Hospital

Cincinnati, Ohio, United States

Site Status

University Hospitals Cleveland Medical Center

Cleveland, Ohio, United States

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Cleveland Clinic Main Campus

Cleveland, Ohio, United States

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The Ohio State University College of Medicine

Columbus, Ohio, United States

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Oregon Health and Science University

Portland, Oregon, United States

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Lancaster General Hospital

Lancaster, Pennsylvania, United States

Site Status

Penn Presbyterian Medical Center

Philadelphia, Pennsylvania, United States

Site Status

Medical University of South Carolina

Charleston, South Carolina, United States

Site Status

Vanderbilt Heart and Vascular Institute

Nashville, Tennessee, United States

Site Status

University of Texas Southwestern Medical Center

Dallas, Texas, United States

Site Status

Houston Methodist Hospital

Houston, Texas, United States

Site Status

University of Utah

Salt Lake City, Utah, United States

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Virginia Commonwealth University

Richmond, Virginia, United States

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University of Washington

Seattle, Washington, United States

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University of Wisconsin - Madison

Madison, Wisconsin, United States

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Hospital Italiano de Buenos Aires

Buenos Aires, , Argentina

Site Status

Instituto Cardiovascular de Buenos Aires

Buenos Aires, , Argentina

Site Status

Advara HeartCare

Joondalup, Western Australia, Australia

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Advara HeartCare Murdoch

Murdoch, Western Australia, Australia

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Box Hill Hospital

Clayton, , Australia

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Royal Hobart Hospital

Hobart, , Australia

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Advara HeartCare - Joondalup

Joondalup, , Australia

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Liverpool Hospital

Liverpool, , Australia

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Advara HeartCare Murdoch

Murdoch, , Australia

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Westmead Hospital

Westmead, , Australia

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Princess Alexandra Hospital

Woolloongabba, , Australia

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Medizinische Universität Graz

Graz, , Austria

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Medizinische Universität Innsbruck

Innsbruck, , Austria

Site Status

Medizinische Universität Wien

Vienna, , Austria

Site Status

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

Bruges, , Belgium

Site Status

Ziekenhuis Oost-Limburg - Campus Sint-Jan

Limbourg, , Belgium

Site Status

Hospital Moinhos de Vento

Porto Alegre, , Brazil

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Hospital das Clínicas da Faculdade de Medicina de Ribeirão Preto

Ribeirão Preto, , Brazil

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A Beneficência Portuguesa de São Paulo - Unidade Mirante

São Paulo, , Brazil

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Instituto Dante Pazzanese de Cardiologia

São Paulo, , Brazil

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Instituto do Coração de São Paulo

São Paulo, , Brazil

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Universidade Estadual de Campinas

São Paulo, , Brazil

Site Status

Centro Avançado de Pesquisa e Estudos para o Diagnóstico CAPED

São Paulo, , Brazil

Site Status

University of Calgary

Calgary, Alberta, Canada

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University Hospital - London Health Sciences Centre

London, Ontario, Canada

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Centre Hospitalier de l'Université de Montréal (CHUM)

Montreal, Quebec, Canada

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Hôpital Regional de Rimouski

Rimouski, , Canada

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Gordon and Leslie Diamond Health Care Centre

Vancouver, , Canada

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Fakultní Nemocnice u sv. Anny v Brně

Brno, , Czechia

Site Status

Vseobecna Fakultni Nemocnice v Praze

Prague, , Czechia

Site Status

Institut Klinické a Experimentální Medicíny

Prague, , Czechia

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Rigshospitalet

Copenhagen, , Denmark

Site Status

Hôpitaux Universitaires Henri Mondor

Créteil, , France

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Centre Hospitalier Départemental Vendée

La Roche-sur-Yon, , France

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Hôpital de la Timone

Marseille, , France

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Centre Hospitalier Universitaire De Nantes - Hôpital Nord Laennec

Nantes, , France

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Hôpital Haut-Lévêque

Pessac, , France

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Hôpital Rangueil

Toulouse, , France

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Uniklinik Köln

Cologne, , Germany

Site Status

Universitaetsklinikum Heidelberg - Zentrum für Innere Medizin

Heidelberg, , Germany

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Universitätsklinikum des Saarlandes

Homburg, , Germany

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Universitätsklinikum Münster

Münster, , Germany

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Universitätsklinikum Würzburg

Würzburg, , Germany

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General Hospital of Athens "Alexandra"

Athens, , Greece

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Rambam Health Care Campus

Haifa, , Israel

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Hadassah University Hospital Ein Kerem

Jerusalem, , Israel

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Kaplan Medical Center

Rehovot, , Israel

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Azienda Ospedaliero Universitaria Delle Marche

Ancona, , Italy

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Azienda Ospedaliero - Universitaria Careggi

Florence, , Italy

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Azienda Ospedale Università di Padova

Padua, , Italy

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Fondazione IRCCS Policlinico San Matteo

Pavia, , Italy

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

Pisa, , Italy

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Azienda Ospedaliera - Universitaria Sant' Andrea

Roma, , Italy

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Kurume University Hospital

Kurume-Shi, Fukuoka, Japan

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Saiseikai Fukuoka General Hospital

Fukuoka, Hukuoka [Fukuoka], Japan

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Kumamoto University Hospital

Kumamoto, , Japan

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Krakowski Szpital Specjalistyczny im. Jana Pawła II

Krakow, , Poland

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Hospital da Senhora da Oliveira

Guimarães, , Portugal

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

Guimarães, , Portugal

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

Lisbon, , Portugal

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Centro Hospitalar Universitário do Porto - Hospital Geral de Santo Antonio

Porto, , Portugal

Site Status

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

Porto, , Portugal

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Centro Hospitalar Universitário de São João

Porto, , Portugal

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Unidade Local de Saúde de São João, EPE

Porto, , Portugal

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Hospital Universitari Vall d'Hebrón

Barcelona, , Spain

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Hospital Universitario Puerta de Hierro

Majadahonda, , Spain

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Hospital Clínico Universitario Virgen de la Arrixaca

Murcia, , Spain

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Complejo Hospitalario Universitario de Santiago (CHUS)

Santiago de Compostela, , Spain

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Sahlgrenska Universitetssjukhuset

Gothenburg, , Sweden

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Skellefteå lasarett

Skellefteå, , Sweden

Site Status

AES - Synexus - Scotland Clinical Research Centre

Bellshill, England, United Kingdom

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AES - Synexus - Midlands

Edgbaston, England, United Kingdom

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Synexus - Manchester Clinical Research Centre

Manchester, England, United Kingdom

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AES - Synexus - Wales

Cardiff, Wales, United Kingdom

Site Status

Royal Free London NHS Foundation Trust

Hampstead, , United Kingdom

Site Status

Richmond Pharmacology

London, , United Kingdom

Site Status

Countries

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United States Argentina Australia Austria Belgium Brazil Canada Czechia Denmark France Germany Greece Israel Italy Japan Poland Portugal Spain Sweden United Kingdom

Other Identifiers

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2022-000826-74

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

ION-682884-CS12

Identifier Type: -

Identifier Source: org_study_id

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