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

Alnylam Pharmaceuticals1 site in 1 country26 target enrollmentDecember 2013

ConditionsTTR-mediated Amyloidosis

InterventionsALN-TTRSC (revusiran) for subcutaneous administration

DrugsALN-TTRSC (revusiran) for subcutaneous administration

Overview

Phase: Phase 2
Intervention: ALN-TTRSC (revusiran) for subcutaneous administration
Conditions: TTR-mediated Amyloidosis
Sponsor: Alnylam Pharmaceuticals
Enrollment: 26
Locations: 1
Primary Endpoint: The proportion of subjects experiencing adverse events (AEs), serious adverse events (SAEs) and study drug discontinuation.
Status: Completed
Last Updated: 3 years ago

Overview Investigators Eligibility Criteria Arms & Interventions Outcomes Sites Similar Trials

Overview

Brief 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.

Registry

clinicaltrials.gov

Start Date

December 2013

End Date

January 2015

Last Updated

3 years ago

Study Type

Interventional

Study Design

Single Group

Sex

All

Investigators

Sponsor

Alnylam Pharmaceuticals

Responsible Party

Sponsor

Eligibility 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.

Arms & Interventions

ALN-TTRSC (revusiran)

Intervention: ALN-TTRSC (revusiran) for subcutaneous administration

Outcomes

Primary Outcomes

The proportion of subjects experiencing adverse events (AEs), serious adverse events (SAEs) and study drug discontinuation.

Time Frame: Up to 63 days

Secondary Outcomes

Pharmacokinetics (PK) of ALN-TTRSC (revusiran) (Cmax, tmax, t1/2, AUC, CL, Vss, Vz)(Up to 90 days)
Effect of ALN-TTRSC (revusiran) on transthyretin (TTR) (Determination of % Lowering of TTR to pretreatment/Baseline Levels)(Up to 90 days)