HELIOS-B: A Study to Evaluate Vutrisiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy

Last updated: November 13, 2025
Sponsor: Alnylam Pharmaceuticals
Overall Status: Active - Not Recruiting

Phase

3

Condition

Circulation Disorders

Amyloidosis

Treatment

Vutrisiran

Sterile Normal Saline (0.9% NaCl)

Clinical Study ID

NCT04153149
ALN-TTRSC02-003
2019-003153-28
2024-518318-25-00
2023-508366-15-00
2023-508366-15
  • Ages 18-85
  • All Genders

Study Summary

This study will evaluate the efficacy and safety of vutrisiran 25 mg administered subcutaneously (SC) once every 3 months (q3M) compared to placebo in participants with ATTR amyloidosis with cardiomyopathy.

Eligibility Criteria

Inclusion

Inclusion Criteria:

  • Has a documented diagnosis of transthyretin (ATTR) amyloidosis with cardiomyopathy,classified as either hereditary ATTR (hATTR) amyloidosis with cardiomyopathy orwild-type ATTR (wtATTR) amyloidosis with cardiomyopathy meeting pre-specifieddiagnostic criteria

  • Has medical history of heart failure (HF) with at least 1 prior hospitalization forHF OR clinical evidence of HF

Exclusion

Exclusion Criteria:

  • Has known primary amyloidosis or leptomeningeal amyloidosis

  • Has New York Heart Association (NYHA) Class IV heart failure

  • Has NYHA Class III heart failure AND is at high risk based on pre-specified criteria

  • Has a polyneuropathy disability (PND) Score IIIa, IIIb, or IV at the Screening visit

  • Has estimated glomerular filtration rate (eGFR) <30 mL/min/1.73 m^2

  • Has received prior TTR-lowering treatment

  • Has other non-TTR cardiomyopathy, hypertensive cardiomyopathy, cardiomyopathy due tovalvular heart disease, or cardiomyopathy due to ischemic heart disease

Study Design

Total Participants: 655
Treatment Group(s): 2
Primary Treatment: Vutrisiran
Phase: 3
Study Start date:
November 26, 2019
Estimated Completion Date:
December 01, 2026

Connect with a study center

  • Clinical Trial Site

    Buenos Aires,
    Argentina

    Site Not Available

  • Clinical Trial Site

    Buenos Aires 3435910,
    Argentina

    Site Not Available

  • Clinical Trial Site

    Adelaide,
    Australia

    Site Not Available

  • Clinical Trial Site

    Adelaide 2078025,
    Australia

    Site Not Available

  • Clinical Trial Site

    Melbourne,
    Australia

    Site Not Available

  • Clinical Trial Site

    Melbourne 2158177,
    Australia

    Site Not Available

  • Clinical Trial Site

    Sydney,
    Australia

    Site Not Available

  • Clinical Trial Site

    Sydney 2147714,
    Australia

    Site Not Available

  • Clinical Trial Site

    Westmead,
    Australia

    Site Not Available

  • Clinical Trial Site

    Westmead 2143973,
    Australia

    Site Not Available

  • Clinical Trial Site

    Woolloongabba,
    Australia

    Site Not Available

  • Clinical Trial Site

    Woolloongabba 6943568,
    Australia

    Site Not Available

  • Clinical Trial Site

    Graz,
    Austria

    Site Not Available

  • Clinical Trial Site

    Vienna,
    Austria

    Site Not Available

  • Clinical Trial Site

    Vienna 2761369,
    Austria

    Site Not Available

  • Clinical Trial Site

    Anderlecht,
    Belgium

    Site Not Available

  • Clinical Trial Site

    Anderlecht 2803201,
    Belgium

    Site Not Available

  • Clinical Trial Site

    Dendermonde,
    Belgium

    Site Not Available

  • Clinical Trial Site

    Dendermonde 2799645,
    Belgium

    Site Not Available

  • Clinical Trial Site

    Ghent,
    Belgium

    Site Not Available

  • Clinical Trial Site

    Ghent 2797656,
    Belgium

    Site Not Available

  • Clinical Trial Site

    Leuven,
    Belgium

    Site Not Available

  • Clinical Trial Site

    Leuven 2792482,
    Belgium

    Site Not Available

  • Clinical Trial Site

    Quebec City,
    Canada

    Site Not Available

  • Clinical Trial Site

    Québec 6325494,
    Canada

    Site Not Available

  • Clinical Trial Site

    Toronto,
    Canada

    Site Not Available

  • Clinical Trial Site

    Toronto 6167865,
    Canada

    Site Not Available

  • Clinical Trial Site

    Bogotá,
    Colombia

    Site Not Available

  • Clinical Trial Site

    Zagreb,
    Croatia

    Site Not Available

  • Clinical Trial Site

    Zagreb 3186886,
    Croatia

    Site Not Available

  • Clinical Trial Site

    Olomouc,
    Czechia

    Site Not Available

  • Clinical Trial Site

    Olomouc 3069011,
    Czechia

    Site Not Available

  • Clinical Trial Site

    Prague,
    Czechia

    Site Not Available

  • Clinical Trial Site

    Prague 3067696,
    Czechia

    Site Not Available

  • Clinical Trial Site

    Aarhus,
    Denmark

    Site Not Available

  • Clinical Trial Site

    Aarhus 2624652,
    Denmark

    Site Not Available

  • Clinical Trial Site

    Odense,
    Denmark

    Site Not Available

  • Clinical Trial Site

    Odense 2615876,
    Denmark

    Site Not Available

  • Clinical Trial Site

    Créteil,
    France

    Site Not Available

  • Clinical Trial Site

    Créteil 3022530,
    France

    Site Not Available

  • Clinical Trial Site

    Marseille,
    France

    Site Not Available

  • Clinical Trial Site

    Marseille 2995469,
    France

    Site Not Available

  • Clinical Trial Site

    Paris,
    France

    Site Not Available

  • Clinical Trial Site

    Paris 2988507,
    France

    Site Not Available

  • Clinical Trial Site

    Toulouse,
    France

    Site Not Available

  • Clinical Trial Site

    Toulouse 2972315,
    France

    Site Not Available

  • Clinical Trial Site

    Essen,
    Germany

    Site Not Available

  • Clinical Trial Site

    Essen 2928810,
    Germany

    Site Not Available

  • Clinical Trial Site

    Göttingen,
    Germany

    Site Not Available

  • Clinical Trial Site

    Göttingen 2918632,
    Germany

    Site Not Available

  • Clinical Trial Site

    Heidelberg,
    Germany

    Site Not Available

  • Clinical Trial Site

    Heidelberg 2907911,
    Germany

    Site Not Available

  • Clinical Trial Site

    Leipzig,
    Germany

    Site Not Available

  • Clinical Trial Site

    Leipzig 2879139,
    Germany

    Site Not Available

  • Clinical Trial Site

    Münster,
    Germany

    Site Not Available

  • Clinical Trial Site

    Münster 2867543,
    Germany

    Site Not Available

  • Clinical Trial Site

    Würzburg,
    Germany

    Site Not Available

  • Clinical Trial Site

    Würzburg 2805615,
    Germany

    Site Not Available

  • Clinical Trial Site

    Budapest,
    Hungary

    Site Not Available

  • Clinical Trial Site

    Budapest 3054643,
    Hungary

    Site Not Available

  • Clinical Trial Site

    Szeged,
    Hungary

    Site Not Available

  • Clinical Trial Site

    Dublin,
    Ireland

    Site Not Available

  • Clinical Trial Site

    Dublin 2964574,
    Ireland

    Site Not Available

  • Clinical Trial Site

    Haifa,
    Israel

    Site Not Available

  • Clinical Trial Site

    Haifa 294801,
    Israel

    Site Not Available

  • Clinical Trial Site

    Jerusalem,
    Israel

    Site Not Available

  • Clinical Trial Site

    Ramat Gan,
    Israel

    Site Not Available

  • Clinical Trial Site

    Aichi,
    Japan

    Site Not Available

  • Clinical Trial Site

    Aichi 11192139,
    Japan

    Site Not Available

  • Clinical Trial Site

    Fukuoka,
    Japan

    Site Not Available

  • Clinical Trial Site

    Fukuoka 1863967,
    Japan

    Site Not Available

  • Clinical Trial Site

    Kagawa,
    Japan

    Site Not Available

  • Clinical Trial Site

    Kagawa 1860837,
    Japan

    Site Not Available

  • Clinical Trial Site

    Kashihara,
    Japan

    Site Not Available

  • Clinical Trial Site

    Kashihara 1859952,
    Japan

    Site Not Available

  • Clinical Trial Site

    Kumamoto,
    Japan

    Site Not Available

  • Clinical Trial Site

    Kumamoto 1858421,
    Japan

    Site Not Available

  • Clinical Trial Site

    Matsumoto,
    Japan

    Site Not Available

  • Clinical Trial Site

    Matsumoto 1857519,
    Japan

    Site Not Available

  • Clinical Trial Site

    Nankoku,
    Japan

    Site Not Available

  • Clinical Trial Site

    Nankoku 11612330,
    Japan

    Site Not Available

  • Clinical Trial Site

    Osaka,
    Japan

    Site Not Available

  • Clinical Trial Site

    Osaka 1853909,
    Japan

    Site Not Available

  • Clinical Trial Site

    Tokyo,
    Japan

    Site Not Available

  • Clinical Trial Site

    Tokyo 1850147,
    Japan

    Site Not Available

  • Clinical Trial Site

    Seoul,
    Korea, Republic of

    Site Not Available

  • Clinical Trial Site

    Riga,
    Latvia

    Site Not Available

  • Clinical Trial Site

    Riga 456172,
    Latvia

    Site Not Available

  • Clinical Trial Site

    Beirut,
    Lebanon

    Site Not Available

  • Clinical Trial Site

    Kaunas,
    Lithuania

    Site Not Available

  • Clinical Trial Site

    Kaunas 598316,
    Lithuania

    Site Not Available

  • Clinical Trial Site

    Kuantan,
    Malaysia

    Site Not Available

  • Clinical Trial Site

    Shah Alam,
    Malaysia

    Site Not Available

  • Clinical Trial Site

    Chisinau,
    Moldova, Republic of

    Site Not Available

  • Clinical Trial Site

    Eindhoven,
    Netherlands

    Site Not Available

  • Clinical Trial Site

    Groningen,
    Netherlands

    Site Not Available

  • Clinical Trial Site

    Groningen 2755251,
    Netherlands

    Site Not Available

  • Clinical Trial Site

    Utrecht,
    Netherlands

    Site Not Available

  • Clinical Trial Site

    Utrecht 2745912,
    Netherlands

    Site Not Available

  • Clinical Trial Site

    Oslo,
    Norway

    Site Not Available

  • Clinical Trial Site

    Oslo 3143244,
    Norway

    Site Not Available

  • Clinical Trial Site

    Callao,
    Peru

    Site Not Available

  • Clinical Trial Site

    Lima,
    Peru

    Site Not Available

  • Clinical Trial Site

    Lima 3936456,
    Peru

    Site Not Available

  • Clinical Trial Site

    San Miguel,
    Peru

    Site Not Available

  • Clinical Trial Site

    San Miguel 3929403,
    Peru

    Site Not Available

  • Clinical Trial Site

    Gdansk 3099434,
    Poland

    Site Not Available

  • Clinical Trial Site

    Gdańsk,
    Poland

    Site Not Available

  • Clinical Trial Site

    Katowice,
    Poland

    Site Not Available

  • Clinical Trial Site

    Kraków,
    Poland

    Site Not Available

  • Clinical Trial Site

    Warszawa,
    Poland

    Site Not Available

  • Clinical Trial Site

    Wroclaw 3081368,
    Poland

    Site Not Available

  • Clinical Trial Site

    Wrocław,
    Poland

    Site Not Available

  • Clinical Trial Site

    Łódź,
    Poland

    Site Not Available

  • Clinical Trial Site

    Coimbra,
    Portugal

    Site Not Available

  • Clinical Trial Site

    Faro,
    Portugal

    Site Not Available

  • Clinical Trial Site

    Guimarães,
    Portugal

    Site Not Available

  • Clinical Trial Site

    Guimarães 2738752,
    Portugal

    Site Not Available

  • Clinical Trial Site

    Lisboa,
    Portugal

    Site Not Available

  • Clinical Trial Site

    Lisbon 2267057,
    Portugal

    Site Not Available

  • Clinical Trial Site

    Porto,
    Portugal

    Site Not Available

  • Clinical Trial Site

    Porto 2735943,
    Portugal

    Site Not Available

  • Clinical Trial Site

    Makkah,
    Saudi Arabia

    Site Not Available

  • Clinical Trial Site

    Riyadh,
    Saudi Arabia

    Site Not Available

  • Clinical Trial Site

    Ljubljana,
    Slovenia

    Site Not Available

  • Clinical Trial Site

    Seoul 1835848,
    South Korea

    Site Not Available

  • Clinical Trial Site

    Barcelona,
    Spain

    Site Not Available

  • Clinical Trial Site

    Barcelona 3128760,
    Spain

    Site Not Available

  • Clinical Trial Site

    Bilbao,
    Spain

    Site Not Available

  • Clinical Trial Site

    Bilbao 3128026,
    Spain

    Site Not Available

  • Clinical Trial Site

    Huelva,
    Spain

    Site Not Available

  • Clinical Trial Site

    Huelva 2516548,
    Spain

    Site Not Available

  • Clinical Trial Site

    Madrid,
    Spain

    Site Not Available

  • Clinical Trial Site

    Madrid 3117735,
    Spain

    Site Not Available

  • Clinical Trial Site

    Málaga,
    Spain

    Site Not Available

  • Clinical Trial Site

    Málaga 2514256,
    Spain

    Site Not Available

  • Clinical Trial Site

    Gothenburg,
    Sweden

    Site Not Available

  • Clinical Trial Site

    Gothenburg 2711537,
    Sweden

    Site Not Available

  • Clinical Trial Site

    Umeå,
    Sweden

    Site Not Available

  • Clinical Trial Site

    Umeå 602150,
    Sweden

    Site Not Available

  • Clinical Trial Site

    Chiang Mai,
    Thailand

    Site Not Available

  • Clinical Trial Site

    Hexham 2647007, Northumberland
    United Kingdom

    Site Not Available

  • Clinical Trial Site

    Birmingham,
    United Kingdom

    Site Not Available

  • Clinical Trial Site

    Birmingham 2655603,
    United Kingdom

    Site Not Available

  • Clinical Trial Site

    Cardiff,
    United Kingdom

    Site Not Available

  • Clinical Trial Site

    Cardiff 2653822,
    United Kingdom

    Site Not Available

  • Clinical Trial Site

    Glasgow,
    United Kingdom

    Site Not Available

  • Clinical Trial Site

    Glasgow 2648579,
    United Kingdom

    Site Not Available

  • Clinical Trial Site

    London,
    United Kingdom

    Site Not Available

  • Clinical Trial Site

    London 2643743,
    United Kingdom

    Site Not Available

  • Clinical Trial Site

    Londonderry,
    United Kingdom

    Site Not Available

  • Clinical Trial Site

    Manchester,
    United Kingdom

    Site Not Available

  • Clinical Trial Site

    Manchester 2643123,
    United Kingdom

    Site Not Available

  • Clinical Trial Site

    Middlesbrough,
    United Kingdom

    Site Not Available

  • Clinical Trial Site

    Middlesbrough 2642607,
    United Kingdom

    Site Not Available

  • Clinical Trial Site

    La Mesa, California 91942
    United States

    Site Not Available

  • Clinical Trial Site

    Los Angeles, California 90033
    United States

    Site Not Available

  • Clinical Trial Site

    Stanford, California 94305
    United States

    Site Not Available

  • Clinical Trial Site

    Stanford 5398563, California 5332921 94305
    United States

    Site Not Available

  • Clinical Trial Site

    Washington, District of Columbia 20010
    United States

    Site Not Available

  • Clinical Trial Site

    Washington D.C. 4140963, District of Columbia 4138106 20010
    United States

    Site Not Available

  • Clinical Trial Site

    Gainesville, Georgia 30501
    United States

    Site Not Available

  • Clinical Trial Site

    Gainesville 4196586, Georgia 4197000 30501
    United States

    Site Not Available

  • Clinical Trial Site

    Chicago, Illinois 60637
    United States

    Site Not Available

  • Clinical Trial Site

    Evanston, Illinois 60201
    United States

    Site Not Available

  • Clinical Trial Site

    Evanston 4891382, Illinois 4896861 60201
    United States

    Site Not Available

  • Clinical Trial Site

    Boston, Massachusetts 02115
    United States

    Site Not Available

  • Clinical Trial Site

    Boston 4930956, Massachusetts 6254926 02118
    United States

    Site Not Available

  • Clinical Trial Site

    Rochester, Minnesota 55902
    United States

    Site Not Available

  • Clinical Trial Site

    Rochester 5043473, Minnesota 5037779 55902
    United States

    Site Not Available

  • Clinical Trial Site

    Manhasset, New York 11030
    United States

    Site Not Available

  • Clinical Trial Site

    New York, New York 10032
    United States

    Site Not Available

  • Clinical Trial Site

    Manhasset 5125766, New York 5128638 11030
    United States

    Site Not Available

  • Clinical Trial Site

    New York 5128581, New York 5128638 10032
    United States

    Site Not Available

  • Clinical Trial Site

    Durham, North Carolina 27710
    United States

    Site Not Available

  • Clinical Trial Site

    Durham 4464368, North Carolina 4482348 27710
    United States

    Site Not Available

  • Clinical Trial Site

    Cleveland, Ohio 44195
    United States

    Site Not Available

  • Clinical Trial Site

    Cleveland 5150529, Ohio 5165418 44195
    United States

    Site Not Available

  • Clinical Trial Site

    Allentown, Pennsylvania 18103
    United States

    Site Not Available

  • Clinical Trial Site

    Philadelphia, Pennsylvania 19104
    United States

    Site Not Available

  • Clinical Trial Site

    Philadelphia 4560349, Pennsylvania 6254927 19104
    United States

    Site Not Available

  • Clinical Trial Site

    Houston, Texas 77030
    United States

    Site Not Available

  • Clinical Trial Site

    Houston 4699066, Texas 4736286 77030
    United States

    Site Not Available

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