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

• Status: Active, not recruiting
• Trial end date: 2024-05-01
• Target enrollment: 0
• Participant gender: All

Summary

The purpose of this study is to evaluate the efficacy and safety of vutrisiran (ALN-TTRSC02) in patients with hereditary transthyretin amyloidosis (hATTR amyloidosis). Participants will receive vutrisiran or the reference comparator patisiran during the Treatment Period. The Treatment Period is followed by a Treatment Extension Period during which all participants in the patisiran group will switch to vutrisiran. This study will use the placebo arm of the APOLLO study (NCT01960348) as an external comparator for the primary and most other efficacy endpoints.

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Alnylam Pharmaceuticals

Criteria

Inclusion Criteria:

• Male or female of 18 to 85 years of age (inclusive);

• Has a diagnosis of hATTR amyloidosis with transthyretin (TTR) mutation;

• Has adequate neurologic impairment score (NIS);

• Has adequate polyneuropathy disability (PND) score;

• Has adequate Karnofsky Performance Status (KPS).

Exclusion Criteria:

• Had a prior liver transplant or is likely to undergo liver transplantation during the study;

• Has known other (non-hATTR) forms of amyloidosis or leptomeningeal amyloidosis;

• Has New York Heart Association heart failure classification >2;

• Clinically significant liver function test abnormalities;

• Has known human immunodeficiency virus (HIV), hepatitis C virus (HCV) or hepatitis B virus (HBV) infection;

• Received an experimental drug within 30 days of dosing;

• Received prior TTR-lowering treatment;

• Has other known causes of neuropathy.