A Study to Evaluate Vutrisiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy
An Open-Label Extension Study to Assess the Safety and Efficacy of Vutrisiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy (ATTR Amyloidosis With Cardiomyopathy)
2 other identifiers
interventional
700
17 countries
87
Brief Summary
The purpose of this study is to obtain safety, efficacy, and pharmacodynamic data on the use of vutrisiran in patients with ATTR amyloidosis with cardiomyopathy who continued on extended use of vutrisiran, or switched from patisiran.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P75+ for phase_3
Started Dec 2024
Typical duration for phase_3
87 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
November 6, 2024
CompletedFirst Posted
Study publicly available on registry
November 8, 2024
CompletedStudy Start
First participant enrolled
December 3, 2024
CompletedPrimary Completion
Last participant's last visit for primary outcome
March 31, 2028
ExpectedStudy Completion
Last participant's last visit for all outcomes
March 31, 2028
February 20, 2026
February 1, 2026
3.3 years
November 6, 2024
February 19, 2026
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Frequency of Adverse Events (AEs)
An AE is any untoward medical occurrence in a patient or clinical investigational subject administered a medicinal product and which does not necessarily have a causal relationship with this treatment.
Up to 36 Months
Secondary Outcomes (6)
Change from Baseline in the Kansas City Cardiomyopathy Questionnaire Overall Summary (KCCQ-OS)
Baseline up to 36 Months
Change from Baseline in New York Heart Association (NYHA) Class
Baseline up to 36 Months
Change From Baseline in the Cardiac Biomarker: N-terminal Prohormone of B-type Natriuretic Peptide (NT-proBNP)
Baseline up to 36 Months
Change From Baseline in the Cardiac Biomarker: Troponin I
Baseline up to 36 Months
All-Cause Mortality and Recurrent Cardiovascular (CV) Events (CV Hospitalizations and Urgent Heart Failure [HF] Visits)
Up to 36 Months
- +1 more secondary outcomes
Study Arms (1)
Vutrisiran 25 mg
EXPERIMENTALParticipants will receive vutrisiran 25 mg administered subcutaneously (SC) once every 3 months (q3M).
Interventions
Eligibility Criteria
You may qualify if:
- Patients who are participating in the patisiran parent studies ALN-TTR02-011 or ALN-TTR02-014 or who have completed the 24-month OLE Period in the vutrisiran parent study ALN-TTRSC02-003
You may not qualify if:
- Has permanently discontinued study drug administration while participating in the parent studies, ALN-TTR02-011, ALN-TTR02-014, or ALN-TTRSC02-003
- Future or current participation in another investigational device or drug study, scheduled to occur during this study
- Has other medical conditions or comorbidities which, in the opinion of the Investigator, would interfere with study compliance or data interpretation
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (87)
Clinical Trial Site
Phoenix, Arizona, 85054, United States
Clinical Trial Site
Beverly Hills, California, 90211, United States
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La Jolla, California, 92037, United States
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Stanford, California, 94305, United States
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Washington D.C., District of Columbia, 20010, United States
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Chicago, Illinois, 60637, United States
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Glenview, Illinois, 60026, United States
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Indianapolis, Indiana, 46202, United States
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Kansas City, Kansas, 66160, United States
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Baltimore, Maryland, 21287, United States
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Boston, Massachusetts, 02115, United States
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Boston, Massachusetts, 02118, United States
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Rochester, Minnesota, 55905, United States
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St Louis, Missouri, 63110, United States
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Omaha, Nebraska, 68198, United States
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New York, New York, 10029, United States
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New York, New York, 10032, United States
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Durham, North Carolina, 27710, United States
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Cleveland, Ohio, 44195, United States
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Columbus, Ohio, 43214, United States
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Philadelphia, Pennsylvania, 19104, United States
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Nashville, Tennessee, 37232, United States
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Dallas, Texas, 75246, United States
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Salt Lake City, Utah, 84132, United States
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Buenos Aires, C1093AAS, Argentina
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Buenos Aires, C1199ABB, Argentina
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Buenos Aires, C1428, Argentina
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Córdoba, X5000KEH, Argentina
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Rosario, S2000DEJ, Argentina
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Rosario, S2000DSR, Argentina
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Rosario, S2000PBJ, Argentina
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Vienna, 1090, Austria
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Vienna, 1210, Austria
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Aalst, 9300, Belgium
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Anderlecht, 1070, Belgium
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Dendermonde, 9200, Belgium
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Ghent, 9000, Belgium
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Hasselt, 3500, Belgium
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Leuven, 3000, Belgium
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Liège, 4000, Belgium
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Roeselare, 8800, Belgium
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Ribeirão Preto, 14026-900, Brazil
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Ribeirão Preto, 14051-140, Brazil
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Rio de Janeiro, 22280-020, Brazil
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São Paulo, 04012-180, Brazil
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São Paulo, 05403-000, Brazil
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Prague, 120 00, Czechia
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Prague, 128 21, Czechia
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Prague, 140 21, Czechia
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Aarhus N, 8200, Denmark
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København Ø, 2100, Denmark
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Odense C, 5000, Denmark
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Créteil, 94000, France
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Marseille, 13005, France
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Paris, 75018, France
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Rennes, 35000, France
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Toulouse, 31059, France
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Bologna, 40138, Italy
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Florence, 50134, Italy
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Pavia, 27100, Italy
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Groningen, 9713 GZ, Netherlands
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Maastricht, 6229 HX, Netherlands
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Utrecht, 3584 CX, Netherlands
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Oslo, 372, Norway
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Creixomil, 4835-044, Portugal
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Lisbon, 1649-035, Portugal
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Porto, 4099-001, Portugal
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Viseu, 3504-509, Portugal
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Seoul, 03080, South Korea
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Seoul, 03722, South Korea
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Seoul, 06351, South Korea
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Barcelona, 08035, Spain
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Bilbao, 48013, Spain
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L'Hospitalet de Llobregat, 8907, Spain
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Madrid, 28222, Spain
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Málaga, 29010, Spain
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Gothenburg, 413 45, Sweden
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Solna, 171 76, Sweden
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Umeå, 907 37, Sweden
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Taipei, 112, Taiwan
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Bellshill, ML4 3NJ, United Kingdom
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Birmingham, B15 2SQ, United Kingdom
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Cardiff, CF15 9SS, United Kingdom
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Hexham, NE46 1QJ, United Kingdom
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London, NW3 2QG, United Kingdom
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London, SE1 1YR, United Kingdom
Clinical Trial Site
Manchester, M15 6SE, United Kingdom
Related Publications (1)
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.
PMID: 39213194RESULT
Related Links
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Study Officials
- STUDY DIRECTOR
Medical Director
Alnylam Pharmaceuticals
Study Design
- Study Type
- interventional
- Phase
- phase 3
- Allocation
- NA
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
November 6, 2024
First Posted
November 8, 2024
Study Start
December 3, 2024
Primary Completion (Estimated)
March 31, 2028
Study Completion (Estimated)
March 31, 2028
Last Updated
February 20, 2026
Record last verified: 2026-02
Data Sharing
- IPD Sharing
- Will share
Access to Anonymized individual participant data that support these results is made available 12 months after study completion and not less than 12 months after the product and indication have been approved in the US and/or the EU. Access to data may be declined where there is likelihood a patient could be identified or other feasibility issue, where there is a potential conflict of interest, a planned business activities or an actual or potential competitive risk. Data will be provided contingent upon the approval of a research proposal and the execution of a data sharing agreement. Timeframes for data access may vary and can take up to 6 months or more. Requests for access to data can be submitted via the website www.vivli.org. Questions can also be directed to datasharing@alnylam.com.