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

Alnylam Pharmaceuticals

Status and phase: Active, not recruiting; Phase 3

Conditions: Transthyretin Amyloidosis (ATTR) With Cardiomyopathy

Treatments: Drug: Vutrisiran; Drug: Sterile Normal Saline (0.9% NaCl)

Study type: Interventional

Funder types: Industry

Identifiers

NCT04153149

ALN-TTRSC02-003

2023-508366-15-00 (EU Trial (CTIS) Number)

Details and patient eligibility

About

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.

Enrollment: 655 patients

Sex: All

Ages: 18 to 85 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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655 participants in 2 patient groups, including a placebo group

Vutrisiran 25 mg

Experimental group

Description:

Participants received vutrisiran 25 milligrams (mg) administered subcutaneously (SC) once every 3 months (q3M) during the 36-month double-blind (DB) period. After the DB period, participants continue receiving vutrisiran, 25 mg, SC injection, q3M for up to 24 months in the open-label treatment extension (OLE) period.

Treatment:

Drug: Vutrisiran

Placebo

Placebo Comparator group

Description:

Participants received vutrisiran matching placebo administered SC q3M during the 36-month DB period. After the DB period, participants receive vutrisiran, 25 mg, SC injection, q3M for up to 24 months in the OLE period.

Treatment:

Drug: Sterile Normal Saline (0.9% NaCl)

Drug: Vutrisiran