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

• Status: Active, not recruiting
• Trial end date: 2025-06-01
• Target enrollment: 0
• Participant gender: All

Summary

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.

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Alnylam Pharmaceuticals

Criteria

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