ENDEAVOUR: Phase 3 Multicenter Study of Revusiran (ALN-TTRSC) in Patients With Transthyretin (TTR) Mediated Familial Amyloidotic Cardiomyopathy (FAC)

Not Recruiting

Trial ID: NCT02319005

Purpose

The purpose of this study was to evaluate the safety and efficacy of revusiran (ALN-TTRSC) in patients with transthyretin (TTR) mediated Familial Amyloidotic Cardiomyopathy. Dosing has been discontinued; patients are being followed-up for safety.

Official Title

A Phase 3 Multicenter, Multinational, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of ALN TTRSC in Patients With Transthyretin (TTR) Mediated Familial Amyloidotic Cardiomyopathy (FAC)

Stanford Investigator(s)

Ronald M Witteles
Ronald M Witteles

Professor of Medicine (Cardiovascular Medicine)

Eligibility

Inclusion Criteria:

* Documented TTR mutation
* Amyloid deposits in cardiac or non-cardiac tissue
* Medical history of heart failure
* Evidence of cardiac involvement by echocardiogram

Exclusion Criteria:

* Has known primary amyloidosis (AL), leptomeningeal amyloidosis, non-FAC hereditary cardiomyopathy, hypertensive cardiomyopathy, or cardiomyopathy due to valvular heart disease
* Has known peripheral vascular disease affecting ambulation
* Has a Polyneuropathy Disability score \>2
* Has a New York Heart Association (NYHA) classification of IV

Intervention(s):

drug: Revusiran (ALN-TTRSC)

drug: Sterile Normal Saline (0.9% NaCl)

Not Recruiting

Contact Information

Stanford University
School of Medicine
300 Pasteur Drive
Stanford, CA 94305