Phase 2 Study to Evaluate ALN-TTRSC (Revusiran) in Patients With Transthyretin (TTR) Cardiac Amyloidosis

• Status: Completed
• Trial end date: 2015-01-01
• Target enrollment: 0
• Participant gender: All

Summary

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.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Alnylam Pharmaceuticals

Criteria

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.