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

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.