Clinical Trial
Study Details
Eplontersen will be administered by SC injection.
Outcomes
Study Overview
The purpose of this study is to evaluate the safety and tolerability of extended dosing with eplontersen in participants with ATTR-CM.
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.
Contacts and Locations
Mayo Clinic
Scottsdale, AZ 85259
United States
Cedars-Sinai Medical Center
Beverly Hills, CA 90211
United States
Altman Clinical and Translational Research Institute - Center for Clinical Research
La Jolla, CA 92037
United States
University of Colorado Hospital - Anschutz Medical Campus
Aurora, CO 80045
United States
MedStar Washington Hospital Center
Washington, DC 20010
United States
Cleveland Clinic Florida
Weston, FL 33331
United States
Piedmont Heart of Fayetteville
Atlanta, GA 30309
United States
Piedmont Atlanta Hospital
Fayetteville, GA 30214
United States
Northwestern University
Chicago, IL 60611
United States
Indiana University Health University Hospital
Indianapolis, IN 46202
United States
University of Kansas Medical Center (KUMC)
Kansas City, KS 66160
United States
University of Maryland Medical Center
Baltimore, MD 21201
United States
Massachusetts General Hospital
Boston, MA 02114
United States
Boston University School of Medicine
Boston, MA 02118
United States
Mayo Clinic
Rochester, MN 55905
United States
Barnes-Jewish Hospital
Saint Louis, MO 63110
United States
New York University Langone Cardiology Associates
New York, NY 10016
United States
Weill Cornell Medicine Cardiology
New York, NY 10021
United States
Columbia University Irving Medical Center
New York, NY 10034
United States
University of North Carolina at Chapel Hill
Chapel Hill, NC 27599
United States
Duke University Medical Center
Durham, NC 27710
United States
Cleveland Clinic Main Campus
Cleveland, OH 44195
United States
The Ohio State University College of Medicine
Columbus, OH 43210
United States
Oregon Health and Science University
Portland, OR 97239
United States
Penn Presbyterian Medical Center
Philadelphia, PA 19104
United States
Vanderbilt Heart and Vascular Institute
Nashville, TN 37232
United States
University of Texas Southwestern Medical Center
Dallas, TX 75390
United States
University of Utah
Salt Lake City, UT 84112
United States
Virginia Commonwealth University
Richmond, VA 23298
United States
University of Washington
Seattle, WA 98195
United States
Advara HeartCare Murdoch
Murdoch 6150
Australia
Advara HeartCare
Joondalup WA 6027
Australia
Liverpool Hospital
Liverpool 2170
Australia
Medizinische Universität Graz
8036 Graz
Austria
Medizinische Universität Innsbruck
6020 Innsbruck
Austria
Algemeen Ziekenhuis Sint-Jan Brugge-Oostende - Campus Sint-Jan
8000 Brugge
Belgium
University of Calgary
Calgary AB T2N 4Z6
Canada
University Hospital - London Health Sciences Centre
London ON N6A 5A5
Canada
Centre Hospitalier de l'Université de Montréal (CHUM)
Montréal QC H2X 0C1
Canada
Centre Hospitalier Départemental Vendée
85925 La Roche-sur-Yon
France
Centre Hospitalier Universitaire De Nantes - Hôpital Nord Laennec
44093 Nantes
France
Hôpital Haut-Lévêque
33604 Pessac
France
Universitätsklinikum des Saarlandes
66421 Homburg
Germany
Universitätsklinikum Münster
48149 Münster
Germany
Universitätsklinikum Würzburg
97078 Würzburg
Germany
General Hospital of Athens "Alexandra"
115 28 Athens
Greece
Rambam Health Care Campus
Haifa 3109601
Israel
Kaplan Medical Center
Rehovot 7661041
Israel
Azienda Ospedaliero - Universitaria Careggi
50134 Firenze
Italy
Fondazione IRCCS Policlinico San Matteo
27100 Pavia
Italy
Hospital da Senhora da Oliveira
4835-044 Guimarães
Portugal
Centro Hospitalar Universitário do Porto - Hospital Geral de Santo Antonio
4099-001 Porto
Portugal
Centro Hospitalar Universitário de São João
4200-319 Porto
Portugal
Hospital Universitari Vall d'Hebrón
08035 Barcelona
Spain
Hospital Universitario Puerta de Hierro
28222 Majadahonda
Spain
Hospital Clínico Universitario Virgen de la Arrixaca
30120 Murcia
Spain
Sahlgrenska Universitetssjukhuset
413 45 Göteborg
Sweden
Skellefteå lasarett
931 86 Skellefteå
Sweden
Richmond Pharmacology
London
SE1 1YR
United Kingdom
Participation Criteria
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.