HELIOS-B: A Study to Evaluate Vutrisiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy
NCT ID: NCT04153149
Last Updated: 2026-01-12
Study Results
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View full resultsBasic Information
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ACTIVE_NOT_RECRUITING
PHASE3
655 participants
INTERVENTIONAL
2019-11-26
2026-12-02
Brief Summary
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Detailed Description
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Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
QUADRUPLE
Study Groups
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Vutrisiran 25 mg
Participants received vutrisiran 25 milligrams (mg) administered subcutaneously (SC) once every 3 months (q3M) during the 36-month double-blind (DB) period. After the DB period, participants continue receiving vutrisiran, 25 mg, SC injection, q3M for up to 24 months in the open-label treatment extension (OLE) period.
Vutrisiran
Vutrisiran will be administered by SC injection.
Placebo
Participants received vutrisiran matching placebo administered SC q3M during the 36-month DB period. After the DB period, participants receive vutrisiran, 25 mg, SC injection, q3M for up to 24 months in the OLE period.
Vutrisiran
Vutrisiran will be administered by SC injection.
Sterile Normal Saline (0.9% NaCl)
Sterile normal saline (0.9% NaCl) will be administered by SC injection.
Interventions
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Vutrisiran
Vutrisiran will be administered by SC injection.
Sterile Normal Saline (0.9% NaCl)
Sterile normal saline (0.9% NaCl) will be administered by SC injection.
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* Has medical history of heart failure (HF) with at least 1 prior hospitalization for HF OR clinical evidence of HF
Exclusion Criteria
* 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 to valvular heart disease, or cardiomyopathy due to ischemic heart disease
18 Years
85 Years
ALL
No
Sponsors
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Alnylam Pharmaceuticals
INDUSTRY
Responsible Party
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Principal Investigators
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Medical Director
Role: STUDY_DIRECTOR
Alnylam Pharmaceuticals
Locations
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Clinical Trial Site
La Mesa, California, United States
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Los Angeles, California, United States
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Stanford, California, United States
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Washington D.C., District of Columbia, United States
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Gainesville, Georgia, United States
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Chicago, Illinois, United States
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Evanston, Illinois, United States
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Boston, Massachusetts, United States
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Boston, Massachusetts, United States
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Rochester, Minnesota, United States
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Manhasset, New York, United States
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New York, New York, United States
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Durham, North Carolina, United States
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Cleveland, Ohio, United States
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Allentown, Pennsylvania, United States
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Philadelphia, Pennsylvania, United States
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Houston, Texas, United States
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Buenos Aires, Ciudad Autónoma de BuenosAires, Argentina
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Buenos Aires, Pilar, Argentina
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Buenos Aires, , Argentina
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Buenos Aires, , Argentina
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Darlinghurst, New South Wales, Australia
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Northmead, New South Wales, Australia
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Woolloongabba, Queensland, Australia
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Adelaide, South Australia, Australia
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Melbourne, Victoria, Australia
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Graz, , Austria
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Vienna, , Austria
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Vienna, , Austria
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Dendermonde, Oost-Vlaanderen, Belgium
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Ghent, Oost-Vlaanderen, Belgium
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Brussels, , Belgium
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Leuven, , Belgium
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Toronto, Ontario, Canada
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Québec, Quebec, Canada
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Zagreb, , Croatia
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Olomouc, Olomoucký kraj, Czechia
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Prague, Praha, Hlavní Mesto, Czechia
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Odense, Region Syddanmark, Denmark
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Aarhus N, , Denmark
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Créteil, , France
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Marseille, , France
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Paris, , France
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Toulouse, , France
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Frankfurt am Main, Hesse, Germany
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Leipzig, Saxony, Germany
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Essen, , Germany
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Göttingen, , Germany
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Heidelberg, , Germany
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Münster, , Germany
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Würzburg, , Germany
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Budapest, , Hungary
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Budapest, , Hungary
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Szeged, , Hungary
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Dublin, , Ireland
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Dublin, , Ireland
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Haifa, , Israel
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Jerusalem, , Israel
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Ramat Gan, , Israel
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Tsurumai-cho, Aichi-ken, Japan
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Fukuoka, Fukuoka, Japan
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Kurume, Fukuoka, Japan
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Kita-gun, Kagawa-ken, Japan
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Nankoku, Kochi, Japan
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Matsumoto, Nagano, Japan
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Kashihara, Nara, Japan
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Suita, Osaka, Japan
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Suita, Osaka, Japan
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Bunkyo-ku, Tokyo, Japan
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Bunkyo-ku, Tokyo, Japan
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Shinjuku-Ku, Tokyo, Japan
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Fukuoka, , Japan
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Kumamoto, , Japan
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Riga, , Latvia
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Hamra, Beyrouth, Lebanon
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Kaunas, , Lithuania
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Kuantan, Pahang, Malaysia
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Sungai Buloh, Selangor, Malaysia
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Chisinau, , Moldova
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Eindhoven, , Netherlands
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Groningen, , Netherlands
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Utrecht, , Netherlands
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Oslo, , Norway
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San Isidro, Lima region, Peru
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Lima, , Peru
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Wroclaw, Lower Silesian Voivodeship, Poland
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Lodz, Lódzkie, Poland
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Warsaw, Masovian Voivodeship, Poland
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Gdansk, Pomeranian Voivodeship, Poland
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Katowice, , Poland
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Lisbon, Lisbon District, Portugal
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Coimbra, , Portugal
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Faro, , Portugal
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Guimarães, , Portugal
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Porto, , Portugal
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Mecca, Mecca Region, Saudi Arabia
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Riyadh, , Saudi Arabia
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Ljubljana, , Slovenia
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Seoul, , South Korea
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Seoul, , South Korea
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Seoul, , South Korea
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Seoul, , South Korea
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L'Hospitalet de Llobregat, Barcelona, Spain
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Majadahonda, Madrid, Spain
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Barcelona, , Spain
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Bilbao, , Spain
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Huelva, , Spain
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Málaga, , Spain
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Göteborg, Västerbotten County, Sweden
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Gothenburg, , Sweden
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Chiang Mai, , Thailand
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Bellshill, Lanarkshire, United Kingdom
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London, London, City of, United Kingdom
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Londonderry, Londonderry, United Kingdom
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Hexham, Northumberland, United Kingdom
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Birmingham, , United Kingdom
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Cardiff, , United Kingdom
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London, , United Kingdom
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Manchester, , United Kingdom
Countries
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References
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Jering KS, Fontana M, Lairez O, Longhi S, Azevedo O, Morbach C, Bender S, Jay PY, Vest J, Bulwer BE, Prasad N, Solomon SD, Skali H. Effects of vutrisiran on cardiac structure and function in patients with transthyretin amyloidosis with cardiomyopathy: secondary outcomes of the HELIOS-B trial. Nat Med. 2025 Oct;31(10):3560-3568. doi: 10.1038/s41591-025-03851-z. Epub 2025 Aug 6.
Maurer MS, Berk JL, Damy T, Sheikh FH, Gonzalez-Costello J, Morbach C, Delgado D, Bondue A, Azevedo O, Poulsen SH, Jankowska EA, Yang L, Bender S, Eraly SA, Jay PY, Vest J, Fontana M. Impact of Vutrisiran on Cardiac Biomarkers in Patients With Transthyretin Amyloidosis With Cardiomyopathy From HELIOS-B. J Am Coll Cardiol. 2025 Aug 12;86(6):459-475. doi: 10.1016/j.jacc.2025.04.055.
Witteles RM, Garcia-Pavia P, Damy T, Grogan M, Sheikh FH, Morbach C, Bender S, Exter J, Eraly SA, Fontana M. Vutrisiran Improves Survival and Reduces Cardiovascular Events in ATTR Amyloid Cardiomyopathy: HELIOS-B. J Am Coll Cardiol. 2025 Apr 30:S0735-1097(25)06170-4. doi: 10.1016/j.jacc.2025.04.008. Online ahead of print.
Maurer MS, Witteles RM, Garcia-Pavia P, Sheikh FH, Morbach C, Rodriguez Duque D, Aldinc E, Eraly SA, Gillmore JD. Impact of Heart Failure Severity on Vutrisiran Efficacy in Transthyretin Amyloidosis With Cardiomyopathy. J Am Coll Cardiol. 2025 May 27;85(20):1927-1939. doi: 10.1016/j.jacc.2025.03.477. Epub 2025 Mar 17.
Fontana M, Maurer MS, Gillmore JD, Bender S, Aldinc E, Eraly SA, Jay PY, Solomon SD. Outpatient Worsening Heart Failure in Patients With Transthyretin Amyloidosis With Cardiomyopathy in the HELIOS-B Trial. J Am Coll Cardiol. 2025 Feb 25;85(7):753-761. doi: 10.1016/j.jacc.2024.11.015. Epub 2024 Nov 18.
Fontana M, Berk JL, Gillmore JD, Witteles RM, Grogan M, Drachman B, Damy T, Garcia-Pavia P, Taubel J, Solomon SD, Sheikh FH, Tahara N, Gonzalez-Costello J, Tsujita K, Morbach C, Pozsonyi Z, Petrie MC, Delgado D, Van der Meer P, Jabbour A, Bondue A, Kim D, Azevedo O, Hvitfeldt Poulsen S, Yilmaz A, Jankowska EA, Algalarrondo V, Slugg A, Garg PP, Boyle KL, Yureneva E, Silliman N, Yang L, Chen J, Eraly SA, Vest J, Maurer MS; HELIOS-B Trial Investigators. Vutrisiran in Patients with Transthyretin Amyloidosis with Cardiomyopathy. N Engl J Med. 2025 Jan 2;392(1):33-44. doi: 10.1056/NEJMoa2409134. Epub 2024 Aug 30.
Provided Documents
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Document Type: Study Protocol
Document Type: Statistical Analysis Plan
Other Identifiers
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2023-508366-15-00
Identifier Type: CTIS
Identifier Source: secondary_id
ALN-TTRSC02-003
Identifier Type: -
Identifier Source: org_study_id
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