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

Last updated: November 8, 2024
Sponsor: Ionis Pharmaceuticals, Inc.
Overall Status: Active - Recruiting

Phase

3

Condition

Circulation Disorders

Amyloidosis

Treatment

Eplontersen

Clinical Study ID

NCT05667493
ION-682884-CS12
2022-000826-74
  • Ages > 18
  • All Genders

Study Summary

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

Eligibility Criteria

Inclusion

Inclusion Criteria:

  1. Satisfactory completion of Treatment Period and the End of Treatment Visit of theIndex Study (ION-682884-CS2) OR diagnosis of ATTR-CM and satisfactory participationon 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

Exclusion Criteria:

  1. Permanently discontinued study drug administration while participating in the IndexStudy (ION 682884-CS2) or IST (ISIS 420915-CS101 Study).

  2. Have any new condition or worsening of an existing condition that in the opinion ofthe Investigator or Sponsor would make the participant unsuitable for enrolment, orwhich could interfere with the participant participating in or completing the study,including the need for treatment with medications disallowed in the Index Study.

Study Design

Total Participants: 1400
Treatment Group(s): 1
Primary Treatment: Eplontersen
Phase: 3
Study Start date:
November 30, 2022
Estimated Completion Date:
August 31, 2029

Study Description

This is a multicenter, open-label, Phase 3 study in up to approximately 1400 participants. Eligible participants will receive eplontersen once every 4 weeks for up to 36 months or 6 months 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. This study will consist of the following periods: less than or equal to (≤) 10-week screening assessment period, up to 36-month treatment period, and up to 6-month post-treatment evaluation period.

Connect with a study center

  • Instituto Cardiovascular de Buenos Aires

    Ciudad Autonoma de Buenos Aires, C1428ART
    Argentina

    Active - Recruiting

  • Hospital Italiano de Buenos Aires

    Ciudad Autónoma De Buenos Aires, C1199ABB
    Argentina

    Active - Recruiting

  • Advara HeartCare Murdoch

    Murdoch, West Australia 6150
    Australia

    Active - Recruiting

  • Advara HeartCare

    Joondalup, Western Australia 6027
    Australia

    Active - Recruiting

  • Box Hill Hospital

    Clayton, 3168
    Australia

    Active - Recruiting

  • Royal Hobart Hospital

    Hobart, 7000
    Australia

    Active - Recruiting

  • Advara HeartCare - Joondalup

    Joondalup, 6027
    Australia

    Active - Recruiting

  • Liverpool Hospital

    Liverpool, 2170
    Australia

    Active - Recruiting

  • Westmead Hospital

    Westmead, 2145
    Australia

    Active - Recruiting

  • Princess Alexandra Hospital

    Woolloongabba, 4102
    Australia

    Active - Recruiting

  • Medizinische Universität Graz

    Graz, 8036
    Austria

    Active - Recruiting

  • Medizinische Universität Innsbruck

    Innsbruck, 6020
    Austria

    Active - Recruiting

  • Medizinische Universität Wien

    Wien, 1090
    Austria

    Active - Recruiting

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

    Brugge, 8000
    Belgium

    Active - Recruiting

  • Hospital das Clínicas da Faculdade de Medicina de Ribeirão Preto

    Ribeirão Preto, 14040-030
    Brazil

    Active - Recruiting

  • Instituto do Coração de São Paulo

    São Paulo, 05403-000
    Brazil

    Active - Recruiting

  • University of Calgary

    Calgary, Alberta T2N 4Z6
    Canada

    Active - Recruiting

  • University Hospital - London Health Sciences Centre

    London, Ontario N6A 5A5
    Canada

    Active - Recruiting

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

    Montréal, Quebec H2X 0C1
    Canada

    Active - Recruiting

  • Hôpital Regional de Rimouski

    Rimouski, G5L 5T1
    Canada

    Active - Recruiting

  • Gordon and Leslie Diamond Health Care Centre

    Vancouver, V5Z 1M9
    Canada

    Active - Recruiting

  • Institut Klinické a Experimentální Medicíny

    Praha, 140 21
    Czechia

    Active - Recruiting

  • Vseobecna Fakultni Nemocnice v Praze

    Praha, 116 36
    Czechia

    Active - Recruiting

  • Rigshospitalet

    Copenhagen, 2100
    Denmark

    Active - Recruiting

  • Hôpitaux Universitaires Henri Mondor

    Créteil, 94010
    France

    Active - Recruiting

  • Centre Hospitalier Départemental Vendée

    La Roche-sur-Yon, 85925
    France

    Active - Recruiting

  • Hôpital de la Timone

    Marseille, 13005
    France

    Active - Recruiting

  • Centre Hospitalier Universitaire De Nantes - Hôpital Nord Laennec

    Nantes, 44093
    France

    Active - Recruiting

  • Hôpital Haut-Lévêque

    Pessac, 33604
    France

    Active - Recruiting

  • Hôpital Rangueil

    Toulouse, 31059
    France

    Active - Recruiting

  • Universitaetsklinikum Heidelberg - Zentrum für Innere Medizin

    Heidelberg, 69120
    Germany

    Active - Recruiting

  • Universitätsklinikum des Saarlandes

    Homburg, 66421
    Germany

    Active - Recruiting

  • Uniklinik Köln

    Köln, 50937
    Germany

    Active - Recruiting

  • Universitätsklinikum Münster

    Münster, 48149
    Germany

    Active - Recruiting

  • Universitätsklinikum Würzburg

    Würzburg, 97078
    Germany

    Active - Recruiting

  • General Hospital of Athens "Alexandra"

    Athens, 115 28
    Greece

    Active - Recruiting

  • Rambam Health Care Campus

    Haifa, 3109601
    Israel

    Active - Recruiting

  • Kaplan Medical Center

    Rehovot, 7661041
    Israel

    Active - Recruiting

  • Azienda Ospedaliero - Universitaria Careggi

    Firenze, 50134
    Italy

    Active - Recruiting

  • Fondazione IRCCS Policlinico San Matteo

    Pavia, 27100
    Italy

    Active - Recruiting

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

    Pisa, 56124
    Italy

    Active - Recruiting

  • Azienda Ospedaliera - Universitaria Sant' Andrea

    Roma, 00189
    Italy

    Active - Recruiting

  • Hospital da Senhora da Oliveira

    Guimarães, 4835-044
    Portugal

    Active - Recruiting

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

    Lisbon, 1169-024
    Portugal

    Active - Recruiting

  • Centro Hospitalar Universitário de São João

    Porto, 4200-319
    Portugal

    Active - Recruiting

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

    Porto, 4099-001
    Portugal

    Active - Recruiting

  • Hospital Universitari Vall d'Hebrón

    Barcelona, 08035
    Spain

    Active - Recruiting

  • Hospital Universitario Puerta de Hierro

    Majadahonda, 28222
    Spain

    Active - Recruiting

  • Hospital Clínico Universitario Virgen de la Arrixaca

    Murcia, 30120
    Spain

    Active - Recruiting

  • Complejo Hospitalario Universitario de Santiago (CHUS)

    Santiago De Compostela, 15706
    Spain

    Active - Recruiting

  • Sahlgrenska Universitetssjukhuset

    Göteborg, 413 45
    Sweden

    Active - Recruiting

  • Skellefteå lasarett

    Skellefteå, 931 86
    Sweden

    Active - Recruiting

  • Royal Free London NHS Foundation Trust

    Hampstead, NW3 2PF
    United Kingdom

    Active - Recruiting

  • Richmond Pharmacology

    London, SE1 1YR
    United Kingdom

    Active - Recruiting

  • Mayo Clinic

    Scottsdale, Arizona 85259
    United States

    Active - Recruiting

  • Cedars-Sinai Medical Center

    Beverly Hills, California 90211
    United States

    Active - Recruiting

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

    La Jolla, California 92037
    United States

    Active - Recruiting

  • Stanford University (Leland Stanford Junior University)

    Stanford, California 94305
    United States

    Active - Recruiting

  • University of Colorado Hospital - Anschutz Medical Campus

    Aurora, Colorado 80045
    United States

    Active - Recruiting

  • MedStar Washington Hospital Center

    Washington, District of Columbia 20010
    United States

    Active - Recruiting

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

    Washington, District of Columbia 20037
    United States

    Active - Recruiting

  • Cleveland Clinic Florida

    Weston, Florida 33331
    United States

    Active - Recruiting

  • Emory Heart and Vascular Center - Emory Clifton Campus

    Atlanta, Georgia 30322
    United States

    Active - Recruiting

  • Piedmont Heart of Fayetteville

    Atlanta, Georgia 30309
    United States

    Active - Recruiting

  • Piedmont Atlanta Hospital

    Fayetteville, Georgia 30214
    United States

    Active - Recruiting

  • Northwestern University

    Chicago, Illinois 60611
    United States

    Active - Recruiting

  • Loyola University Medical Center

    Maywood, Illinois 60153
    United States

    Active - Recruiting

  • Indiana University Health University Hospital

    Indianapolis, Indiana 46202
    United States

    Active - Recruiting

  • University of Kansas Medical Center

    Kansas City, Kansas 66160
    United States

    Active - Recruiting

  • University of Kansas Medical Center (KUMC)

    Kansas City, Kansas 66160
    United States

    Active - Recruiting

  • University of Maryland Medical Center

    Baltimore, Maryland 21201
    United States

    Active - Recruiting

  • Boston University School of Medicine

    Boston, Massachusetts 02118
    United States

    Active - Recruiting

  • Brigham and Women's Hospital

    Boston, Massachusetts 02115
    United States

    Active - Recruiting

  • Massachusetts General Hospital

    Boston, Massachusetts 02114
    United States

    Active - Recruiting

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

    Minneapolis, Minnesota 55407
    United States

    Active - Recruiting

  • Mayo Clinic

    Rochester, Minnesota 55905
    United States

    Active - Recruiting

  • Saint Luke's Hospital of Kansas City

    Kansas City, Missouri 64111
    United States

    Active - Recruiting

  • Barnes-Jewish Hospital

    Saint Louis, Missouri 63110
    United States

    Active - Recruiting

  • Columbia University Irving Medical Center

    New York, New York 10034
    United States

    Active - Recruiting

  • New York University Langone Cardiology Associates

    New York, New York 10016
    United States

    Active - Recruiting

  • Weill Cornell Medicine Cardiology

    New York, New York 10021
    United States

    Active - Recruiting

  • University of North Carolina at Chapel Hill

    Chapel Hill, North Carolina 27599
    United States

    Active - Recruiting

  • Duke University Medical Center

    Durham, North Carolina 27710
    United States

    Active - Recruiting

  • Carl and Edyth Lindner Research Center at The Christ Hospital

    Cincinnati, Ohio 45219
    United States

    Active - Recruiting

  • Cleveland Clinic Main Campus

    Cleveland, Ohio 44195
    United States

    Active - Recruiting

  • The Ohio State University College of Medicine

    Columbus, Ohio 43210
    United States

    Active - Recruiting

  • Oregon Health and Science University

    Portland, Oregon 97239
    United States

    Active - Recruiting

  • Lancaster General Hospital

    Lancaster, Pennsylvania 17602
    United States

    Active - Recruiting

  • Penn Presbyterian Medical Center

    Philadelphia, Pennsylvania 19104
    United States

    Active - Recruiting

  • Medical University of South Carolina

    Charleston, South Carolina 29425
    United States

    Active - Recruiting

  • Vanderbilt Heart and Vascular Institute

    Nashville, Tennessee 37232
    United States

    Active - Recruiting

  • University of Texas Southwestern Medical Center

    Dallas, Texas 75390
    United States

    Active - Recruiting

  • Houston Methodist Hospital

    Houston, Texas 77030
    United States

    Active - Recruiting

  • University of Utah

    Salt Lake City, Utah 84112
    United States

    Active - Recruiting

  • Virginia Commonwealth University

    Richmond, Virginia 23298
    United States

    Active - Recruiting

  • University of Washington

    Seattle, Washington 98195
    United States

    Active - Recruiting

  • University of Wisconsin - Madison

    Madison, Wisconsin 53792
    United States

    Active - Recruiting

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