A Study to Evaluate Vutrisiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy

February 19, 2026 updated by: Alnylam Pharmaceuticals

An Open-Label Extension Study to Assess the Safety and Efficacy of Vutrisiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy (ATTR Amyloidosis With Cardiomyopathy)

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.

Study Overview

Status

Enrolling by invitation

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Estimated)

700

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Argentina
      • Buenos Aires, Argentina, C1199ABB
        • Clinical Trial Site
      • Buenos Aires, Argentina, C1093AAS
        • Clinical Trial Site
      • Buenos Aires, Argentina, C1428
        • Clinical Trial Site
      • Córdoba, Argentina, X5000KEH
        • Clinical Trial Site
      • Rosario, Argentina, S2000DSR
        • Clinical Trial Site
      • Rosario, Argentina, S2000PBJ
        • Clinical Trial Site
      • Rosario, Argentina, S2000DEJ
        • Clinical Trial Site
  • Austria
      • Vienna, Austria, 1090
        • Clinical Trial Site
      • Vienna, Austria, 1210
        • Clinical Trial Site
  • Belgium
      • Aalst, Belgium, 9300
        • Clinical Trial Site
      • Anderlecht, Belgium, 1070
        • Clinical Trial Site
      • Dendermonde, Belgium, 9200
        • Clinical Trial Site
      • Ghent, Belgium, 9000
        • Clinical Trial Site
      • Hasselt, Belgium, 3500
        • Clinical Trial Site
      • Leuven, Belgium, 3000
        • Clinical Trial Site
      • Liège, Belgium, 4000
        • Clinical Trial Site
      • Roeselare, Belgium, 8800
        • Clinical Trial Site
  • Brazil
      • Ribeirão Preto, Brazil, 14026-900
        • Clinical Trial Site
      • Ribeirão Preto, Brazil, 14051-140
        • Clinical Trial Site
      • Rio de Janeiro, Brazil, 22280-020
        • Clinical Trial Site
      • São Paulo, Brazil, 05403-000
        • Clinical Trial Site
      • São Paulo, Brazil, 04012-180
        • Clinical Trial Site
  • Czechia
      • Prague, Czechia, 120 00
        • Clinical Trial Site
      • Prague, Czechia, 128 21
        • Clinical Trial Site
      • Prague, Czechia, 140 21
        • Clinical Trial Site
  • Denmark
      • Aarhus N, Denmark, 8200
        • Clinical Trial Site
      • København Ø, Denmark, 2100
        • Clinical Trial Site
      • Odense C, Denmark, 5000
        • Clinical Trial Site
  • France
      • Créteil, France, 94000
        • Clinical Trial Site
      • Marseille, France, 13005
        • Clinical Trial Site
      • Paris, France, 75018
        • Clinical Trial Site
      • Rennes, France, 35000
        • Clinical Trial Site
      • Toulouse, France, 31059
        • Clinical Trial Site
  • Italy
      • Bologna, Italy, 40138
        • Clinical Trial Site
      • Florence, Italy, 50134
        • Clinical Trial Site
      • Pavia, Italy, 27100
        • Clinical Trial Site
  • Netherlands
      • Groningen, Netherlands, 9713 GZ
        • Clinical Trial Site
      • Maastricht, Netherlands, 6229 HX
        • Clinical Trial Site
      • Utrecht, Netherlands, 3584 CX
        • Clinical Trial Site
  • Norway
      • Oslo, Norway, 372
        • Clinical Trial Site
  • Portugal
      • Creixomil, Portugal, 4835-044
        • Clinical Trial Site
      • Lisbon, Portugal, 1649-035
        • Clinical Trial Site
      • Porto, Portugal, 4099-001
        • Clinical Trial Site
      • Viseu, Portugal, 3504-509
        • Clinical Trial Site
  • South Korea
      • Seoul, South Korea, 03080
        • Clinical Trial Site
      • Seoul, South Korea, 03722
        • Clinical Trial Site
      • Seoul, South Korea, 06351
        • Clinical Trial Site
  • Spain
      • Barcelona, Spain, 08035
        • Clinical Trial Site
      • Bilbao, Spain, 48013
        • Clinical Trial Site
      • L'Hospitalet de Llobregat, Spain, 8907
        • Clinical Trial Site
      • Madrid, Spain, 28222
        • Clinical Trial Site
      • Málaga, Spain, 29010
        • Clinical Trial Site
  • Sweden
      • Gothenburg, Sweden, 413 45
        • Clinical Trial Site
      • Solna, Sweden, 171 76
        • Clinical Trial Site
      • Umeå, Sweden, 907 37
        • Clinical Trial Site
  • Taiwan
      • Taipei, Taiwan, 112
        • Clinical Trial Site
  • United Kingdom
      • Bellshill, United Kingdom, ML4 3NJ
        • Clinical Trial Site
      • Birmingham, United Kingdom, B15 2SQ
        • Clinical Trial Site
      • Cardiff, United Kingdom, CF15 9SS
        • Clinical Trial Site
      • Hexham, United Kingdom, NE46 1QJ
        • Clinical Trial Site
      • London, United Kingdom, NW3 2QG
        • Clinical Trial Site
      • London, United Kingdom, SE1 1YR
        • Clinical Trial Site
      • Manchester, United Kingdom, M15 6SE
        • Clinical Trial Site
  • United States
    • Arizona
      • Phoenix, Arizona, United States, 85054
        • Clinical Trial Site
    • California
      • Beverly Hills, California, United States, 90211
        • Clinical Trial Site
      • La Jolla, California, United States, 92037
        • Clinical Trial Site
      • Stanford, California, United States, 94305
        • Clinical Trial Site
    • District of Columbia
      • Washington D.C., District of Columbia, United States, 20010
        • Clinical Trial Site
    • Illinois
      • Chicago, Illinois, United States, 60637
        • Clinical Trial Site
      • Glenview, Illinois, United States, 60026
        • Clinical Trial Site
    • Indiana
      • Indianapolis, Indiana, United States, 46202
        • Clinical Trial Site
    • Kansas
      • Kansas City, Kansas, United States, 66160
        • Clinical Trial Site
    • Maryland
      • Baltimore, Maryland, United States, 21287
        • Clinical Trial Site
    • Massachusetts
      • Boston, Massachusetts, United States, 02118
        • Clinical Trial Site
      • Boston, Massachusetts, United States, 02115
        • Clinical Trial Site
    • Minnesota
      • Rochester, Minnesota, United States, 55905
        • Clinical Trial Site
    • Missouri
      • St Louis, Missouri, United States, 63110
        • Clinical Trial Site
    • Nebraska
      • Omaha, Nebraska, United States, 68198
        • Clinical Trial Site
    • New York
      • New York, New York, United States, 10032
        • Clinical Trial Site
      • New York, New York, United States, 10029
        • Clinical Trial Site
    • North Carolina
      • Durham, North Carolina, United States, 27710
        • Clinical Trial Site
    • Ohio
      • Cleveland, Ohio, United States, 44195
        • Clinical Trial Site
      • Columbus, Ohio, United States, 43214
        • Clinical Trial Site
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19104
        • Clinical Trial Site
    • Tennessee
      • Nashville, Tennessee, United States, 37232
        • Clinical Trial Site
    • Texas
      • Dallas, Texas, United States, 75246
        • Clinical Trial Site
    • Utah
      • Salt Lake City, Utah, United States, 84132
        • Clinical Trial Site

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  • 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

Exclusion Criteria:

  • 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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Vutrisiran 25 mg
Participants will receive vutrisiran 25 mg administered subcutaneously (SC) once every 3 months (q3M).
Drug: Vutrisiran
Vutrisiran administered SC q3M
Other Names:
  • ALN-TTRSC02
  • AMVUTTRA®

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Frequency of Adverse Events (AEs)
Time Frame: Up to 36 Months
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 Outcome Measures

Outcome Measure
Measure Description
Time Frame
Change from Baseline in the Kansas City Cardiomyopathy Questionnaire Overall Summary (KCCQ-OS)
Time Frame: Baseline up to 36 Months
The KCCQ is a 23-item self-administered questionnaire quantifying 6 domains (symptoms, physical function, quality of life, social limitation, self-efficacy, and symptom stability) and 2 summary scores (clinical and overall summary [OS]). Scores are transformed to a range of 0-100, in which higher scores reflect better health status.
Baseline up to 36 Months
Change from Baseline in New York Heart Association (NYHA) Class
Time Frame: Baseline up to 36 Months
NYHA classification grades the severity of heart failure symptoms into the following stages: I (no symptoms; ordinary physical activity such as walking and climbing stairs does not cause fatigue or dyspnea), II (symptoms with ordinary physical activity; walking or climbing stairs rapidly, walking uphill, walking or stair climbing after meals, in cold weather, in wind or when under emotional stress causes undue fatigue or dyspnea), III (symptoms with less than ordinary physical activity; walking 1 to 2 blocks on the level and climbing more than 1 flight of stairs in normal conditions causes undue fatigue or dyspnea), IV (symptoms at rest; inability to carry on any physical activity without fatigue or dyspnea).
Baseline up to 36 Months
Change From Baseline in the Cardiac Biomarker: N-terminal Prohormone of B-type Natriuretic Peptide (NT-proBNP)
Time Frame: Baseline up to 36 Months
NT-proBNP will be assessed through measurement of serum levels.
Baseline up to 36 Months
Change From Baseline in the Cardiac Biomarker: Troponin I
Time Frame: Baseline up to 36 Months
Troponin I will be assessed through measurement of serum levels.
Baseline up to 36 Months
All-Cause Mortality and Recurrent Cardiovascular (CV) Events (CV Hospitalizations and Urgent Heart Failure [HF] Visits)
Time Frame: Up to 36 Months
All deaths, hospitalizations and urgent HF visits will be recorded throughout the study as part of SAE and AE monitoring
Up to 36 Months
Change from baseline in serum TTR level
Time Frame: Baseline, Month 6 and Month 12
Baseline, Month 6 and Month 12

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Alnylam Pharmaceuticals

Investigators

  • Study Director: Medical Director, Alnylam Pharmaceuticals

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

December 3, 2024

Primary Completion (Estimated)

March 31, 2028

Study Completion (Estimated)

March 31, 2028

Study Registration Dates

First Submitted

November 6, 2024

First Submitted That Met QC Criteria

November 6, 2024

First Posted (Actual)

November 8, 2024

Study Record Updates

Last Update Posted (Actual)

February 20, 2026

Last Update Submitted That Met QC Criteria

February 19, 2026

Last Verified

February 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • ALN-TTRSC02-007
  • 2024-516568-27 (EudraCT Number)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

YES

IPD Plan Description

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.

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

Yes

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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