An Extension Study to Assess Long-Term Safety of Eplontersen in Adults With Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR-CM)
An Open-Label Extension Study to Assess the Long-Term Safety of Eplontersen (ION-682884) in Patients With Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR-CM)
2 other identifiers
interventional
1,400
19 countries
131
Brief Summary
The purpose of this study is to evaluate the safety and tolerability of extended dosing with eplontersen in participants with ATTR-CM.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P75+ for phase_3
Started Nov 2022
Longer than P75 for phase_3
131 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
July 12, 2022
CompletedStudy Start
First participant enrolled
November 30, 2022
CompletedFirst Posted
Study publicly available on registry
December 28, 2022
CompletedPrimary Completion
Last participant's last visit for primary outcome
August 1, 2029
ExpectedStudy Completion
Last participant's last visit for all outcomes
August 1, 2029
April 17, 2026
April 1, 2026
6.7 years
July 12, 2022
April 16, 2026
Conditions
Keywords
Outcome Measures
Primary Outcomes (4)
Number of Participants With Adverse Events (AE) and Serious Adverse Events (SAE)
Baseline up to 36 months
Change From Baseline in Platelet Count
Baseline up to 36 months
Change From Baseline in Estimated Glomerular Filtration Rate (eGFR)
Baseline up to 36 months
Change From Baseline in Urine Protein Creatinine Ratio (UPCR)
Baseline up to 36 months
Secondary Outcomes (3)
Change From Baseline in Transthyretin (TTR) Serum Levels
Baseline up to 36 months
Change From Baseline in 6-minute Walk Test (6MWT)
Baseline up to 36 months
Change From Baseline in Kansas City Cardiomyopathy Questionnaire (KCCQ)
Baseline up to 36 months
Study Arms (1)
Eplontersen
EXPERIMENTALEplontersen 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.
Interventions
Eplontersen will be administered by SC injection.
Eligibility Criteria
You may qualify if:
- 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.
- Investigator is willing to treat the participant with open-label eplontersen.
- Willingness to adhere to vitamin A supplementation per protocol.
You may not qualify if:
- Permanently discontinued study drug administration while participating in the Index Study (ION 682884-CS2) or IST (ISIS 420915-CS101 Study).
- 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.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Ionis Pharmaceuticals, Inc.lead
- AstraZenecacollaborator
Study Sites (134)
Mayo Clinic
Scottsdale, Arizona, 85259, United States
Cedars-Sinai Medical Center
Beverly Hills, California, 90211, United States
Altman Clinical and Translational Research Institute - Center for Clinical Research
La Jolla, California, 92037, United States
University Of California San Francisco Urology Practice
San Francisco, California, 94143, United States
Stanford University (Leland Stanford Junior University)
Stanford, California, 94305, United States
University of Colorado Hospital - Anschutz Medical Campus
Aurora, Colorado, 80045, United States
MedStar Washington Hospital Center
Washington D.C., District of Columbia, 20010, United States
The George Washington Medical Faculty Associates - Foggy Bottom North Pavilion
Washington D.C., District of Columbia, 20037, United States
Cleveland Clinic Florida
Weston, Florida, 33331, United States
Piedmont Heart of Fayetteville
Atlanta, Georgia, 30309, United States
Emory Heart and Vascular Center - Emory Clifton Campus
Atlanta, Georgia, 30322, United States
Piedmont Atlanta Hospital
Fayetteville, Georgia, 30214, United States
Northwestern University
Chicago, Illinois, 60611, United States
Loyola University Medical Center
Maywood, Illinois, 60153, United States
Indiana University Health University Hospital
Indianapolis, Indiana, 46202, United States
University of Kansas Medical Center (KUMC)
Kansas City, Kansas, 66160, United States
University of Kansas Medical Center
Kansas City, Kansas, 66160, United States
University of Maryland Medical Center
Baltimore, Maryland, 21201, United States
Massachusetts General Hospital
Boston, Massachusetts, 02114, United States
Brigham and Women's Hospital
Boston, Massachusetts, 02115, United States
Boston University School of Medicine
Boston, Massachusetts, 02118, United States
Henry Ford Health System
Detroit, Michigan, 48202, United States
Allina Health Minneapolis Heart Institute - Abbott Northwestern's Heart Hospital
Minneapolis, Minnesota, 55407, United States
Mayo Clinic
Rochester, Minnesota, 55905, United States
Saint Luke's Hospital of Kansas City
Kansas City, Missouri, 64111, United States
Barnes-Jewish Hospital
St Louis, Missouri, 63110, United States
New York University Langone Cardiology Associates
New York, New York, 10016, United States
Weill Cornell Medicine Cardiology
New York, New York, 10021, United States
Columbia University Irving Medical Center
New York, New York, 10034, United States
University of North Carolina at Chapel Hill
Chapel Hill, North Carolina, 27599, United States
Duke University Medical Center
Durham, North Carolina, 27710, United States
Carl and Edyth Lindner Research Center at The Christ Hospital
Cincinnati, Ohio, 45219, United States
University Hospitals Cleveland Medical Center
Cleveland, Ohio, 44106, United States
Cleveland Clinic Main Campus
Cleveland, Ohio, 44195, United States
The Ohio State University College of Medicine
Columbus, Ohio, 43210, United States
Oregon Health and Science University
Portland, Oregon, 97239, United States
Lancaster General Hospital
Lancaster, Pennsylvania, 17602, United States
Penn Presbyterian Medical Center
Philadelphia, Pennsylvania, 19104, United States
Medical University of South Carolina
Charleston, South Carolina, 29425, United States
Vanderbilt Heart and Vascular Institute
Nashville, Tennessee, 37232, United States
University of Texas Southwestern Medical Center
Dallas, Texas, 75390, United States
Houston Methodist Hospital
Houston, Texas, 77030, United States
University of Utah
Salt Lake City, Utah, 84112, United States
Virginia Commonwealth University
Richmond, Virginia, 23298, United States
University of Washington
Seattle, Washington, 98195, United States
University of Wisconsin - Madison
Madison, Wisconsin, 53792, United States
Hospital Italiano de Buenos Aires
Buenos Aires, C1199ABB, Argentina
Instituto Cardiovascular de Buenos Aires
Buenos Aires, C1428ART, Argentina
Advara HeartCare
Joondalup, Western Australia, 6027, Australia
Advara HeartCare Murdoch
Murdoch, Western Australia, 6150, Australia
Box Hill Hospital
Clayton, 3168, Australia
Royal Hobart Hospital
Hobart, 7000, Australia
Advara HeartCare - Joondalup
Joondalup, 6027, Australia
Advara Heartcare Leabrook
Leabrook, 5068, Australia
Liverpool Hospital
Liverpool, 2170, Australia
Advara HeartCare Murdoch
Murdoch, 6150, Australia
Westmead Hospital
Westmead, 2145, Australia
Princess Alexandra Hospital
Woolloongabba, 4102, Australia
Medizinische Universität Graz
Graz, 8036, Austria
Medizinische Universität Innsbruck
Innsbruck, 6020, Austria
Medizinische Universität Wien
Vienna, 1090, Austria
Algemeen Ziekenhuis Sint-Jan Brugge-Oostende - Campus Sint-Jan
Bruges, 8000, Belgium
Ziekenhuis Oost-Limburg - Campus Sint-Jan
Limbourg, 3600, Belgium
Hospital Moinhos de Vento
Porto Alegre, 90560-030, Brazil
Hospital das Clínicas da Faculdade de Medicina de Ribeirão Preto
Ribeirão Preto, 14040-030, Brazil
A Beneficência Portuguesa de São Paulo - Unidade Mirante
São Paulo, 01323-030, Brazil
Instituto Dante Pazzanese de Cardiologia
São Paulo, 04012-909, Brazil
Instituto do Coração de São Paulo
São Paulo, 05403-000, Brazil
Universidade Estadual de Campinas
São Paulo, 13083881, Brazil
Centro Avançado de Pesquisa e Estudos para o Diagnóstico CAPED
São Paulo, 14026-900, Brazil
University of Calgary
Calgary, Alberta, T2N 4Z6, Canada
University Hospital - London Health Sciences Centre
London, Ontario, N6A 5A5, Canada
Toronto General Hospital
Toronto, Ontario, M5G 2C4, Canada
Centre Hospitalier de l'Université de Montréal (CHUM)
Montreal, Quebec, H2X 0C1, Canada
Hôpital Regional de Rimouski
Rimouski, G5L 5T1, Canada
Gordon and Leslie Diamond Health Care Centre
Vancouver, V5Z 1M9, Canada
Fakultní Nemocnice u sv. Anny v Brně
Brno, 656 91, Czechia
Vseobecna Fakultni Nemocnice v Praze
Prague, 116 36, Czechia
Institut Klinické a Experimentální Medicíny
Prague, 140 21, Czechia
Rigshospitalet
Copenhagen, 2100, Denmark
Hôpitaux Universitaires Henri Mondor
Créteil, 94010, France
Centre Hospitalier Départemental Vendée
La Roche-sur-Yon, 85925, France
Hôpital de la Timone
Marseille, 13005, France
Centre Hospitalier Universitaire De Nantes - Hôpital Nord Laennec
Nantes, 44093, France
Hôpital Haut-Lévêque
Pessac, 33604, France
Hôpital Rangueil
Toulouse, 31059, France
Uniklinik Köln
Cologne, 50937, Germany
Universitaetsklinikum Heidelberg - Zentrum für Innere Medizin
Heidelberg, 69120, Germany
Universitätsklinikum des Saarlandes
Homburg, 66421, Germany
Universitätsklinikum Münster
Münster, 48149, Germany
Universitätsklinikum Würzburg
Würzburg, 97078, Germany
General Hospital of Athens "Alexandra"
Athens, 115 28, Greece
Rambam Health Care Campus
Haifa, 3109601, Israel
Hadassah University Hospital Ein Kerem
Jerusalem, 9112001, Israel
Kaplan Medical Center
Rehovot, 7661041, Israel
Azienda Ospedaliero Universitaria Delle Marche
Ancona, 60126, Italy
Azienda Ospedaliero - Universitaria Careggi
Florence, 50134, Italy
Azienda Ospedale Università di Padova
Padova, 35128, Italy
Fondazione IRCCS Policlinico San Matteo
Pavia, 27100, Italy
Fondazione Toscana Gabriele Monasterio per la Ricerca Medica e di Sanita Pubblica
Pisa, 56124, Italy
Azienda Ospedaliera - Universitaria Sant' Andrea
Roma, 00189, Italy
Kurume University Hospital
Kurume-Shi, Fukuoka, 830-0011, Japan
Saiseikai Fukuoka General Hospital
Fukuoka, Hukuoka [Fukuoka], 810-0001, Japan
Saiseikai Fukuoka General Hospital
Fukuoka, 810-0001, Japan
Hamamatsu University Hospital
Hamamatsu, 4313192, Japan
Kumamoto University Hospital
Kumamoto, 860-8556, Japan
Kurume University Hospital
Kurume, 830-0011, Japan
Shinshu University Hospital
Matsumoto, 390-8621, Japan
Okayama University Hospital
Okayama, 700-8558, Japan
Osaka Metropolitan University Hospital
Osaka, 545-8586, Japan
Sapporo Medical University Hospital
Sapporo, 060-8543, Japan
Keio University Hospital
Shinjuku-Ku, 160-8582, Japan
Krakowski Szpital Specjalistyczny im. Jana Pawła II
Krakow, 31- 202, Poland
Hospital da Senhora da Oliveira
Guimarães, 4835-044, Portugal
Unidade Local de Saúde do Alto Ave, E. P. E (Hospital da Senhora da Oliveira Guimarães)
Guimarães, 4835-044, Portugal
Unidade Local de Saúde de São José, E. P. E - Hospital de Santa Marta
Lisbon, 1169-024, Portugal
Centro Hospitalar Universitário do Porto - Hospital Geral de Santo Antonio
Porto, 4099-001, Portugal
Unidade Local de Saúde de Santo António, E. P. E - Hospital Geral de Santo Antonio
Porto, 4099-001, Portugal
Centro Hospitalar Universitário de São João
Porto, 4200-319, Portugal
Unidade Local de Saúde de São João, EPE
Porto, 4200-319, Portugal
Hospital Universitari Vall d'Hebrón
Barcelona, 08035, Spain
Hospital Universitario Puerta de Hierro
Majadahonda, 28222, Spain
Hospital Clínico Universitario Virgen de la Arrixaca
Murcia, 30120, Spain
Complejo Hospitalario Universitario de Santiago (CHUS)
Santiago de Compostela, 15706, Spain
Sahlgrenska Universitetssjukhuset
Gothenburg, 413 45, Sweden
Skellefteå lasarett
Skellefteå, 931 86, Sweden
AES - Synexus - Scotland Clinical Research Centre
Bellshill, England, ML4 3NJ, United Kingdom
AES - Synexus - Midlands
Edgbaston, England, B15 2SQ, United Kingdom
Synexus - Manchester Clinical Research Centre
Manchester, England, M15 6SE, United Kingdom
AES - Synexus - Wales
Cardiff, Wales, CF15 9SS, United Kingdom
Royal Free London NHS Foundation Trust
Hampstead, NW3 2PF, United Kingdom
Synexus - Merseyside Clinical Research Centre
Liverpool, L22 0LG, United Kingdom
Richmond Pharmacology
London, SE1 1YR, United Kingdom
Synexus - Merseyside Clinical Research Centre
Manchester, M15 6SE, United Kingdom
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Study Design
- Study Type
- interventional
- Phase
- phase 3
- Allocation
- NA
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
July 12, 2022
First Posted
December 28, 2022
Study Start
November 30, 2022
Primary Completion (Estimated)
August 1, 2029
Study Completion (Estimated)
August 1, 2029
Last Updated
April 17, 2026
Record last verified: 2026-04
Data Sharing
- IPD Sharing
- Will share
Ionis may share anonymized individual participant data, aggregated clinical data, and other types of data that support the results in this study. Data requests from qualified researchers will be considered once all three of the following criteria are met: (1) 12 months from marketing approval of the study drug in both the United States and European Union; (2) 18 months from conclusion of the study; and (3) 6 months from publication of study article. Access would be via a secure environment and is contingent upon approval of a research proposal and entry into an appropriate data use agreement. Requests to access data can be submitted via the website https://vivli.org/ourmember/ionis/.