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

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

Not Recruiting
18-85 years
All
Phase 3

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Overview

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.

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

Study details
    Transthyretin Amyloidosis (ATTR) With Cardiomyopathy

NCT04153149

Alnylam Pharmaceuticals

12 December 2025

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