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Enrolling by Invitation

NCT Number: NCT05667493

An Extension Study to Assess Long-Term Safety of Eplontersen in Adults 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.

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Key information

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

Hospital Italiano de Buenos Aires, Buenos Aires, Argentina

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About this study

This is a multicenter, open-label extension, Phase 3 study in up to approximately 1400 participants from the 682884-CS2 study. Eligible participants will receive eplontersen once every 4 weeks for up to 36 months or until 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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • 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 criteria

  • 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.

Treatment and study plan

Eplontersen

Drug

Eplontersen will be administered by SC injection.

Other names: ION-682884, IONIS-TTR-LRx

Primary outcomes

  1. Number of Participants With Adverse Events (AE) and Serious Adverse Events (SAE)

    Time frame: Baseline up to 36 months

  2. Change From Baseline in Platelet Count

    Time frame: Baseline up to 36 months

  3. Change From Baseline in Estimated Glomerular Filtration Rate (eGFR)

    Time frame: Baseline up to 36 months

  4. Change From Baseline in Urine Protein Creatinine Ratio (UPCR)

    Time frame: Baseline up to 36 months

Secondary outcomes

  1. Change From Baseline in Transthyretin (TTR) Serum Levels

    Time frame: Baseline up to 36 months

  2. Change From Baseline in 6-minute Walk Test (6MWT)

    Time frame: Baseline up to 36 months

  3. Change From Baseline in Kansas City Cardiomyopathy Questionnaire (KCCQ)

    Time frame: Baseline up to 36 months

Sponsors and collaborators

Lead sponsor

Ionis Pharmaceuticals, Inc.

Industry

Collaborators

  • AstraZeneca

Registry information

Official study 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)

Important dates

Study start
2022
Primary completion
2029
Study completion
2029
First posted
Dec 28, 2022
Registry last updated
Apr 17, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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