NCT04153149

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

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)

• Status: Active Not Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 655 (actual)
• Sponsor: Alnylam Pharmaceuticals · Industry
• Sex: All
• Age: 18 Years – 85 Years
• Healthy volunteers: Not accepted

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 participants with ATTR amyloidosis with cardiomyopathy.

Conditions

• Transthyretin Amyloidosis (ATTR) With Cardiomyopathy

Interventions

Type	Name	Description
DRUG	Vutrisiran	Vutrisiran will be administered by SC injection.
DRUG	Sterile Normal Saline (0.9% NaCl)	Sterile normal saline (0.9% NaCl) will be administered by SC injection.

Timeline

• Start date: 2019-11-26
• Primary completion: 2024-05-08
• Completion: 2026-12-02
• First posted: 2019-11-06
• Last updated: 2026-01-12
• Results posted: 2025-10-21

Locations

119 sites across 32 countries: United States, Argentina, Australia, Austria, Belgium, Canada, Croatia, Czechia, Denmark, France, Germany, Hungary, Ireland, Israel, Japan, Latvia, Lebanon, Lithuania, Malaysia, Moldova, Netherlands, Norway, Peru, Poland, Portugal, Saudi Arabia, Slovenia, South Korea, Spain, Sweden, Thailand, United Kingdom

Regulatory

• FDA-regulated drug study