ION-682884-CS2, Ionis

Condition: Transthyretin-mediated amyloid cardiomyopathy


Key Inclusion Criteria:

  • 18-90 years old
  • Willing to be genetically tested for mutations in the transthyretin gene
  • Transthyretin (TTR) amyloidosis
  • Not pregnant or lactating

Key Exclusion Criteria:

  • Acute coronary syndrome, unstable angina, stroke, transient ischemic attack, coronary revascularization, cardiac device implantation, cardiac valve repair or major surgery within 3 months of screening visit
  • Hospitalization or urgent visit to emergency department/emergency room for worsening of heart failure within 4 weeks prior to or during screening
  • Uncontrolled hypertension

Study Name

A Phase III Global, Double-Blind, Randomized, Placebo Controlled Study to Evaluate the Efficacy and Safety of ION-682884 in Patients With Transthyretin-Mediated Amyloid Cardiomyopathy (IRB no. 0000696)


The purpose of this study is to evaluate the safety and effectiveness of an investigational drug called ION-682884 in individuals who have transthyretin-mediated amyloid cardiomyopathy (ATTR-CM), a disease caused by change in a protein called transthyretin (TTR). This change in protein can cause the TTR to build up in the heart, which can result in a condition called cardiomyopathy. Participants will be randomly assigned to receive either ION-682884 or a placebo (inactive substance), and they will take Vitamin A supplements for the duration of the study. Researchers will compare the effects of ION-682884 against the effects of placebo.

Participants may receive any medication approved by the U.S. Food and Drug Administration for their amyloid cardiomyopathy condition while participating in this trial.

Principal Investigator