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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
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