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

Alnylam Pharmaceuticals

Status and phase

Completed

Phase 2

Conditions

TTR-mediated Amyloidosis

Treatments

Drug: ALN-TTRSC (revusiran) for subcutaneous administration

Study type

Interventional

Funder types

Industry

Identifiers

NCT01981837

ALN-TTRSC-002

Details and patient eligibility

About

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.

Enrollment

26 patients

Sex

All

Ages

18 to 80 years old

Volunteers

No Healthy Volunteers

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.