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Long-term safety assessment of eplontersen for heart disease caused by amyloidosis

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

Phase 3 Interventional Ionis Pharmaceuticals, Inc. · NCT05667493

This study is testing if a new medication called eplontersen is safe for people with heart disease caused by amyloidosis when taken over a long period of time.

Quick facts

PhasePhase 3
Study typeInterventional
Enrollment1400 (estimated)
Ages18 Years and up
SexAll
SponsorIonis Pharmaceuticals, Inc. Industry-sponsored
Locations98 sites (Scottsdale, Arizona and 97 other locations)
Trial IDNCT05667493 on ClinicalTrials.gov

What this trial studies

This multicenter, open-label Phase 3 study aims to evaluate the long-term safety and tolerability of eplontersen in adults diagnosed with transthyretin-mediated amyloid cardiomyopathy (ATTR-CM). Approximately 1400 participants will receive eplontersen once every four weeks for up to 36 months, along with daily vitamin A supplementation. The study includes a screening period, a treatment period, and a post-treatment evaluation period to monitor participants' health and response to the medication.

Who should consider this trial

Good fit: Ideal candidates for this study are adults who have completed a prior study involving eplontersen or have a diagnosis of ATTR-CM.

Not a fit: Patients who have permanently discontinued eplontersen or have new or worsening conditions that affect their suitability for the study may not benefit.

Why it matters

Potential benefit: If successful, this study could provide valuable insights into the long-term safety of eplontersen, potentially improving treatment options for patients with ATTR-CM.

How similar studies have performed: Other studies involving eplontersen have shown promise, indicating that this approach may be effective in managing ATTR-CM.

Eligibility criteria

Show full inclusion / exclusion 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.

Where this trial is running

Scottsdale, Arizona and 97 other locations

+48 more sites — see ClinicalTrials.gov for the full list.

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions Transthyretin-Mediated Amyloid CardiomyopathyAmyloidosis
Last reviewed 2026-06-10 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.