The purpose of this study is to determine the pharmacokinetics, pharmacodynamics and exploratory clinical activity of ALN-TTRSC (revusiran) in Patients with Transthyretin (TTR) Cardiac Amyloidosis.
TTR cardiac amyloidosis; Women of child-bearing potential (WOCBP) must have a negative pregnancy test, cannot be breast feeding, and must be willing to use 2 highly effective methods of contraception; Male subjects agree to use appropriate contraception; Adequate blood counts, liver, renal and heart function; Adequate Karnofsky performance status; Adequate New York Heart Association (NYHA) Classification Score; Clinically stable on heart medications; Adequate 6-minute walk test; Willing to give written informed consent and are willing to comply with the study requirements.
Exclusion Criteria:
Known human immunodeficiency virus (HIV) positive status or known or suspected systemic bacterial, viral, parasitic, or fungal infection; Subjects with a history of multiple drug allergies or intolerance to SC injection; Received an investigational agent other than tafamidis, diflunisal, doxycycline or tauroursodeoxycholic acid, or an investigational device within 30 days prior to first dose of study; Uncontrolled hypertension, ischemic heart disease or cardiac arrhythmia; Untreated hypo- or hyperthyroidism; Prior major organ transplant; Considered unfit for the study by the Principal Investigator.
