An Extension Study to Assess Long-Term Safety of Eplontersen in Adults With Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR-CM)
Launched by IONIS PHARMACEUTICALS, INC. · Dec 19, 2022
Trial Information
Current as of August 19, 2025
Enrolling by invitation
Keywords
ClinConnect Summary
This clinical trial is looking at the long-term safety of a medication called eplontersen for adults with a condition known as Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR-CM), which affects the heart. If you or a family member have participated in an earlier study involving eplontersen or another related study and are between the ages of 65 and 74, you might be eligible to join this new phase of research. To participate, you need to be willing to continue taking the medicine and follow a specific vitamin A supplement regimen.
If you decide to take part, you can expect to receive eplontersen over an extended period, allowing researchers to better understand how safe it is for long-term use. The trial is currently recruiting participants, and everyone involved will be monitored closely to ensure their health and well-being during the study. It's important to note that if you have had any major health changes or have stopped taking the study medication in the past, you may not qualify for this trial.
Gender
ALL
Eligibility 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.
About Ionis Pharmaceuticals, Inc.
Ionis Pharmaceuticals, Inc. is a leading biopharmaceutical company specializing in the discovery and development of innovative RNA-targeted therapies for the treatment of various diseases, including neurological disorders, cardiovascular conditions, and rare diseases. With a robust pipeline of drug candidates, Ionis leverages its proprietary antisense technology to modulate gene expression and address unmet medical needs. The company's commitment to advancing precision medicine is underscored by its collaborative partnerships and a strong focus on research and development, positioning Ionis as a pioneer in the field of RNA therapeutics.
Contacts
Jennifer Cobb
Immunology at National Institute of Allergy and Infectious Diseases (NIAID)
Locations
Chicago, Illinois, United States
Kansas City, Kansas, United States
Boston, Massachusetts, United States
Durham, North Carolina, United States
Seattle, Washington, United States
Boston, Massachusetts, United States
Charleston, South Carolina, United States
Boston, Massachusetts, United States
Saint Louis, Missouri, United States
Copenhagen, , Denmark
Maywood, Illinois, United States
Scottsdale, Arizona, United States
Houston, Texas, United States
Washington, District Of Columbia, United States
Portland, Oregon, United States
Dallas, Texas, United States
Baltimore, Maryland, United States
Chapel Hill, North Carolina, United States
Richmond, Virginia, United States
Weston, Florida, United States
Rochester, Minnesota, United States
Kansas City, Missouri, United States
Hobart, , Australia
Nashville, Tennessee, United States
Lancaster, Pennsylvania, United States
Salt Lake City, Utah, United States
Pessac, , France
Marseille, , France
Münster, , Germany
Columbus, Ohio, United States
Philadelphia, Pennsylvania, United States
Madison, Wisconsin, United States
Würzburg, , Germany
Calgary, Alberta, Canada
Toulouse, , France
Barcelona, , Spain
Woolloongabba, , Australia
Pavia, , Italy
Graz, , Austria
Innsbruck, , Austria
Liverpool, , Australia
Majadahonda, , Spain
Göteborg, , Sweden
Homburg, , Germany
Beverly Hills, California, United States
Cleveland, Ohio, United States
Wien, , Austria
Rehovot, , Israel
Haifa, , Israel
Indianapolis, Indiana, United States
Westmead, , Australia
Köln, , Germany
Santiago De Compostela, , Spain
La Roche Sur Yon, , France
Murcia, , Spain
London, Ontario, Canada
Montréal, Quebec, Canada
Firenze, , Italy
Vancouver, , Canada
New York, New York, United States
Skellefteå, , Sweden
Guimarães, , Portugal
London, , United Kingdom
Kansas City, Kansas, United States
Würzburg, , Germany
Washington, District Of Columbia, United States
Créteil, , France
Porto, , Portugal
Aurora, Colorado, United States
Athens, , Greece
Brugge, , Belgium
Atlanta, Georgia, United States
New York, New York, United States
Cincinnati, Ohio, United States
Ribeirão Preto, , Brazil
São Paulo, , Brazil
Praha, , Czechia
Roma, , Italy
Porto, , Portugal
Hampstead, , United Kingdom
Ciudad Autónoma De Buenos Aires, , Argentina
Murdoch, West Australia, Australia
Joondalup, Western Australia, Australia
La Jolla, California, United States
Atlanta, Georgia, United States
Fayetteville, Georgia, United States
New York, New York, United States
Nantes, , France
Stanford, California, United States
Minneapolis, Minnesota, United States
Ciudad Autonoma De Buenos Aires, , Argentina
Clayton, , Australia
Joondalup, , Australia
Rimouski, , Canada
Praha, , Czechia
Heidelberg, , Germany
Pisa, , Italy
Lisbon, , Portugal
Patients applied
Timeline
First submit
Trial launched
Trial updated
Estimated completion
Not reported
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