Transthyretin Amyloid Cardiomyopathy Clinical Trial
HELIOS-A: A Study of Vutrisiran (ALN-TTRSC02) in Patients With Hereditary Transthyretin Amyloidosis (hATTR Amyloidosis)
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 SC injection once every 6 months (q6M) or q3M in the Randomized Treatment Extension (RTE) Period.
Eligibility 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.
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There are 12 Locations for this study
San Diego California, 92120, United States
Aurora Colorado, 80045, United States
Chicago Illinois, 97239, United States
Baltimore Maryland, 21205, United States
Boston Massachusetts, 02118, United States
Rochester Minnesota, 55905, United States
Saint Louis Missouri, 63130, United States
New York New York, 10032, United States
Chapel Hill North Carolina, 27599, United States
Columbus Ohio, 43210, United States
Portland Oregon, 97239, United States
Philadelphia Pennsylvania, 19140, United States
Fort Worth Texas, 75246, United States
Buenos Aires , , Argentina
Box Hill , , Australia
Westmead , , Australia
Woolloongabba , , Australia
Bruxelles , , Belgium
Leuven , , Belgium
Rio De Janeiro , , Brazil
Sofia , , Bulgaria
Montréal , , Canada
Vancouver , , Canada
Nicosia , , Cyprus
Bordeaux , , France
Créteil , , France
Marseille , , France
Paris , , France
Mainz , , Germany
Münster , , Germany
Athens , , Greece
Messina , , Italy
Milano , , Italy
Pavia , , Italy
Rome , , Italy
Kumamoto , , Japan
Nagano , , Japan
Nagoya , , Japan
Osaka , , Japan
Junggu , , Korea, Republic of
Kuala Lumpur , , Malaysia
Mexico City , , Mexico
Groningen , , Netherlands
Lisboa , , Portugal
Porto , , Portugal
Barcelona , , Spain
Huelva , , Spain
Madrid , , Spain
Valencia , , Spain
Solna , , Sweden
Umeå , , Sweden
Taipei City , , Taiwan
Taipei , , Taiwan
London , , United Kingdom
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