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

Phases

Phase 3

Study type

Interventional

Source

EU CTIS

Registry ID

CTIS2024-518318-25-00

Acronym

ALN-TTRSC02-003

Enrollment

441

Registered

2024-11-18

Start date

2020-06-24

Completion date

Unknown

Last updated

2025-07-25

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Transthyretin Amyloidosis with Cardiomyopathy (ATTR Amyloidosis with Cardiomyopathy)

Brief summary

Composite outcome of all-cause mortality and recurrent CV events (CV hospitalizations and urgent heart failure [HF] visits) in both the overall population and the vutrisiran monotherapy subgroup (defined as patients not on tafamidis at study baseline).

Detailed description

The following secondary endpoints will be defined in both the overall population and the vutrisiran monotherapy subgroup: - Change from baseline in 6-minute walk test (6-MWT) - Change from baseline in the Kansas City Cardiomyopathy QuestionnaireOverall Summary (KCCQ-OS) - All-cause mortality - Change from baseline in NYHA Class

Interventions

DRUG0.9% Sodium chloride

DRUGAmvuttra 25 mg solution for injection in pre-filled syringe

Eligibility

Sex/Gender

All

Age

18 Years to No maximum

Design outcomes

Primary

Measure	Time frame
Composite outcome of all-cause mortality and recurrent CV events (CV hospitalizations and urgent heart failure [HF] visits) in both the overall population and the vutrisiran monotherapy subgroup (defined as patients not on tafamidis at study baseline).	—

Secondary

Measure	Time frame
The following secondary endpoints will be defined in both the overall population and the vutrisiran monotherapy subgroup: - Change from baseline in 6-minute walk test (6-MWT) - Change from baseline in the Kansas City Cardiomyopathy QuestionnaireOverall Summary (KCCQ-OS) - All-cause mortality - Change from baseline in NYHA Class	—

Countries

Austria, Belgium, Croatia, Czechia, Denmark, France, Germany, Hungary, Ireland, Lithuania, Netherlands, Norway, Poland, Portugal, Spain, Sweden

Outcome results

None listed

Source: EU CTIS · Data processed: Feb 4, 2026