HELIOS-B: A Study to Evaluate Vutrisiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy

Recruiting

Trial ID: NCT04153149

Purpose

This study will evaluate the efficacy and safety of vutrisiran 25 mg administered subcutaneously (SC) once every 3 months (q3M) compared to placebo in patients with ATTR amyloidosis with cardiomyopathy.

Official Title

HELIOS-B: A Phase 3, Randomized, Double-blind, Placebo-controlled, Multicenter Study to Evaluate the Efficacy and Safety of Vutrisiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy (ATTR Amyloidosis With Cardiomyopathy)

Stanford Investigator(s)

Kevin M. Alexander, MD, FACC, FHFSA
Kevin M. Alexander, MD, FACC, FHFSA

Assistant Professor of Medicine (Cardiovascular Medicine) at the Stanford University Medical Center

Eligibility


Inclusion Criteria:

   - Has a documented diagnosis of transthyretin (ATTR) amyloidosis with cardiomyopathy,
   classified as either hereditary ATTR (hATTR) amyloidosis with cardiomyopathy or
   wild-type ATTR (wtATTR) amyloidosis with cardiomyopathy meeting pre-specified
   diagnostic criteria

   - Has medical history of heart failure (HF) with at least 1 prior hospitalization for HF
   OR clinical evidence of HF

Exclusion Criteria:

   - Has known primary amyloidosis or leptomeningeal amyloidosis

   - Has New York Heart Association (NYHA) Class IV heart failure

   - Has NYHA Class III heart failure AND is at high risk based on pre-specified criteria

   - Has a polyneuropathy disability (PND) Score IIIa, IIIb, or IV at the Screening visit

   - Has estimated glomerular filtration rate (eGFR) <30 mL/min/1.73 m^2

   - Has received prior TTR-lowering treatment

   - Has other non-TTR cardiomyopathy, hypertensive cardiomyopathy, cardiomyopathy due to
   valvular heart disease, or cardiomyopathy due to ischemic heart disease

Intervention(s):

drug: Vutrisiran

drug: Sterile Normal Saline (0.9% NaCl)

Recruiting

Contact Information

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