for people ages 18 years and up (full criteria)
at La Jolla, California and other locations
study started
completion around



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

Official Title

An Open-Label Extension Study to Assess the Long-Term Safety of Eplontersen (ION-682884) in Patients With Transthyretin-Mediated Amyloid Cardiomyopathy (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.


Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM), Amyloidosis, Cardiomyopathies, Eplontersen


You can join if…

Open to people ages 18 years and up

  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.

You CAN'T join if...

  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.


  • Altman Clinical and Translational Research Institute - Center for Clinical Research accepting new patients
    La Jolla California 92037 United States
  • Cedars-Sinai Medical Center accepting new patients
    Beverly Hills California 90211 United States


accepting new patients
Start Date
Completion Date
Ionis Pharmaceuticals, Inc.
Phase 3 Cardiomyopathy Research Study
Study Type
Expecting 1400 study participants
Last Updated