HELIOS-B: A Study to Evaluate Vutrisiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy
Phase 3 – large-scale trial before approval
Conditions:
Transthyretin Amyloidosis (ATTR) With Cardiomyopathy
Sponsor: Alnylam Pharmaceuticals
trial.available_in:
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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 participants with ATTR amyloidosis with cardiomyopathy.
Who can participate
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
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