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

Enrolling by invitation · Phase 3

Conditions studied: Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM)

In brief

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

Key facts

Study ID

NCT05667493

Run by

Ionis Pharmaceuticals, Inc.

People needed

1400

Starts

2022-11-30

Expected to finish

2029-08-01

Last updated by the study team

2026-04-17

Who can join

Age: 18 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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.

You may not qualify if…

• 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.

Where it is running

• Cedars-Sinai Medical Center — Beverly Hills, California, United States
• Altman Clinical and Translational Research Institute - Center for Clinical Research — La Jolla, California, United States
• University Of California San Francisco Urology Practice — San Francisco, California, United States
• Stanford University (Leland Stanford Junior University) — Stanford, California, United States
• University of Colorado Hospital - Anschutz Medical Campus — Aurora, Colorado, United States
• MedStar Washington Hospital Center — Washington D.C., District of Columbia, United States
• The George Washington Medical Faculty Associates - Foggy Bottom North Pavilion — Washington D.C., District of Columbia, United States
• Cleveland Clinic Florida — Weston, Florida, United States
• Piedmont Heart of Fayetteville — Atlanta, Georgia, United States
• Emory Heart and Vascular Center - Emory Clifton Campus — Atlanta, Georgia, United States
• Piedmont Atlanta Hospital — Fayetteville, Georgia, United States
• Northwestern University — Chicago, Illinois, United States
• Loyola University Medical Center — Maywood, Illinois, United States
• Indiana University Health University Hospital — Indianapolis, Indiana, United States
• University of Kansas Medical Center (KUMC) — Kansas City, Kansas, United States
• University of Kansas Medical Center — Kansas City, Kansas, United States
• University of Maryland Medical Center — Baltimore, Maryland, United States
• Massachusetts General Hospital — Boston, Massachusetts, United States
• Brigham and Women's Hospital — Boston, Massachusetts, United States
• Boston University School of Medicine — Boston, Massachusetts, United States
• Henry Ford Health System — Detroit, Michigan, United States
• Allina Health Minneapolis Heart Institute - Abbott Northwestern's Heart Hospital — Minneapolis, Minnesota, United States
• Mayo Clinic — Rochester, Minnesota, United States
• Saint Luke's Hospital of Kansas City — Kansas City, Missouri, United States
• Mayo Clinic — Scottsdale, Arizona, United States

Full record on ClinicalTrials.gov

Trial information comes from ClinicalTrials.gov and is refreshed daily. TrialsForMe does not provide medical care and does not run the studies it lists.