An Extension Study to Assess Long-Term Safety of Eplontersen in Adults with Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR-CM)

Overview

About this study

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

Participation eligibility

Participant eligibility includes age, gender, type and stage of disease, and previous treatments or health concerns. Guidelines differ from study to study, and identify who can or cannot participate. There is no guarantee that every individual who qualifies and wants to participate in a trial will be enrolled. Contact the study team to discuss study eligibility and potential 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. 

Eligibility last updated 5/25/23.   Questions regarding updates should be directed to the study team contact.

Participating Mayo Clinic locations

Study statuses change often. Please contact the study team for the most up-to-date information regarding possible participation.

Mayo Clinic Location Status Contact

Scottsdale/Phoenix, Ariz.

Mayo Clinic principal investigator

Julie Rosenthal, M.D.

Open for enrollment

Contact information:

Julieta Williams

(480) 342-1785

Williams.Julieta@mayo.edu

Rochester, Minn.

Mayo Clinic principal investigator

Daniel Borgeson, M.D.

Open for enrollment

Contact information:

Amyloid Research Team

(507) 266-4426

AMYLOIDTEAMRES@mayo.edu

More information

Publications

Publications are currently not available
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CLS-20550439

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