Long-Term Safety of Eplontersen for Transthyretin Amyloid Heart Disease
Studies long-term safety of eplontersen in adults with transthyretin amyloid heart disease.
Plain-language title generated from the registry record
Registry title: An Extension Study to Assess Long-Term Safety of Eplontersen in Adults With Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR-CM)
Key facts
- Where
- Brno and Prague, CzechiaSee the 3 sites
- Phase
- Phase 3
AI summary: who the study is looking for
From the trial's eligibility criteria. Machine-read from the registry text on this page, so it can be wrong. The registry text below and the trial team decide who can take part.
The trial is looking for adults with transthyretin-mediated amyloid cardiomyopathy (ATTR-CM) who finished a related earlier eplontersen study. Everyone gets eplontersen to check long-term safety.
- Age
- 18 years and older
- Sex
- Men and women can join.
- Disease stage
- ATTR-CM (heart muscle disease caused by transthyretin amyloid). No stage is named in the record.
- Earlier treatment
- People must have finished the treatment period and the end-of-treatment visit of the earlier study ION-682884-CS2, or have an ATTR-CM diagnosis and have taken part satisfactorily in study ISIS 420915-CS101.
Main reasons for not taking part
- Stopped the study drug for good during the earlier study ION 682884-CS2 or ISIS 420915-CS101
- A new or worsening health condition that the investigator or sponsor thinks makes joining unsuitable
- Needing medicines that were not allowed in the earlier study
- Not willing to take vitamin A supplements as the study plan says
Read by machine on 9 November 2026.
Where in Czechia
- Fakultni nemocnice u sv. Anny v BrneBrno, CzechiaNo longer taking part
- Institut klinicke a experimentalni medicinyPrague, CzechiaNo longer taking part
- Vseobecna fakultni nemocnice v PrazePrague, CzechiaNo longer taking part
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- Ionis Pharmaceuticals, Inc.
- Trial code
- NCT05667493
- Record updated
- 17 April 2026
- Original description
The purpose of this study is to evaluate the safety and tolerability of extended dosing with eplontersen in participants with ATTR-CM.Read full original descriptionShow less
An Open-Label Extension Study to Assess the Long-Term Safety of Eplontersen (ION-682884) in Patients With Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR-CM)
The purpose of this study is to evaluate the safety and tolerability of extended dosing with eplontersen in participants with ATTR-CM.
Inclusion: 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.
Exclusion: 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.