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HELIOS-A: A Study of Vutrisiran (ALN-TTRSC02) in Patients With Hereditary Transthyretin Amyloidosis (hATTR Amyloidosis)

NCT03759379 · Status: COMPLETED · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 164

Last updated 2026-01-12

Study results available
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Summary

The purpose of this study is to evaluate the efficacy and safety of vutrisiran (ALN-TTRSC02) in participants with hereditary transthyretin amyloidosis (hATTR amyloidosis). Participants will receive vutrisiran subcutaneous (SC) injection once every 3 months (q3M) or the reference comparator patisiran intravenous (IV) injection once every 3 weeks (q3w) during the 18 month Treatment Period. This study will use the placebo arm of the APOLLO study (NCT01960348) as an external comparator for the primary and most other efficacy endpoints during the 18 Month Treatment Period. Following the 18 Month Treatment Period, all participants will be randomized to receive vutrisiran 50 mg SC injection once every 6 months (q6M) or vutrisiran 25 mg q3M in the Randomized Treatment Extension (RTE) Period. Upon implementation of Amendment 6, participants receiving vutrisiran SC 50 mg q6M will transition to vutrisiran SC 25 mg q3M at their next scheduled dosing.

Conditions

  • Amyloidosis, Hereditary
  • Transthyretin Amyloidosis

Interventions

DRUG

Patisiran

Patisiran will be administered by IV infusion.

DRUG

Vutrisiran

Vutrisiran will be administered by SC injection.

Sponsors & Collaborators

Principal Investigators

  • Medical Director · Alnylam Pharmaceuticals

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
PARALLEL

Eligibility

Min Age
18 Years
Max Age
85 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2019-02-14
Primary Completion
2020-11-10
Completion
2025-11-05
FDA Drug
Yes

Countries

  • United States
  • Argentina
  • Australia
  • Belgium
  • Brazil
  • Bulgaria
  • Canada
  • Cyprus
  • France
  • Germany
  • Greece
  • Italy
  • Japan
  • Malaysia
  • Mexico
  • Netherlands
  • Portugal
  • South Korea
  • Spain
  • Sweden
  • Taiwan
  • United Kingdom

Study Locations

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Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT03759379 on ClinicalTrials.gov