A Phase 2, Open-Label Trial to Evaluate the Safety, Pharmacokinetics, Pharmacodynamics and Exploratory Clinical Activity of ALN-TTRSC in Patients With Transthyretin (TTR) Cardiac Amyloidosis

ALN-TTRSC-002

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.

2013-12-01

2014-10-01

2015-01-01

Completed

Early phase I

26

Inclusion Criteria: * 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.

{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE2'], 'designInfo': {'allocation': 'NA', 'interventionModel': 'SINGLE_GROUP', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'NONE'}}, 'enrollmentInfo': {'count': 26, 'type': 'ACTUAL'}}

2023-03-20