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
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.