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

NCT ID: NCT06679946

Last Updated: 2025-12-19

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

ENROLLING_BY_INVITATION

Clinical Phase

PHASE3

Total Enrollment

700 participants

Study Classification

INTERVENTIONAL

Study Start Date

2024-12-03

Study Completion Date

2028-05-30

Brief Summary

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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.

Detailed Description

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Conditions

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Transthyretin Amyloidosis (ATTR) With Cardiomyopathy

Keywords

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Amylodosis TTR ATTR Cardiomyopathy Transthyretin TTR-mediated amyloidosis Amyloidosis, Hereditary, Transthyretin-Related RNAi therapeutic Transthyretin amyloid cardiomyopathy TTR cardiomyopathy ATTR-CM Wild-type TTR V122I TTR amyloidosis Amyloidosis, Wild Type hATTR amyloidosis with cardiomyopathy ATTR amyloidosis with cardiomyopathy wtATTR amyloidosis with cardiomyopathy

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Vutrisiran 25 mg

Participants will receive vutrisiran 25 mg administered subcutaneously (SC) once every 3 months (q3M).

Group Type EXPERIMENTAL

Vutrisiran

Intervention Type DRUG

Vutrisiran administered SC q3M

Interventions

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Vutrisiran

Vutrisiran administered SC q3M

Intervention Type DRUG

Other Intervention Names

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ALN-TTRSC02 AMVUTTRA®

Eligibility Criteria

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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
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Alnylam Pharmaceuticals

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Medical Director

Role: STUDY_DIRECTOR

Alnylam Pharmaceuticals

Locations

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Clinical Trial Site

Phoenix, Arizona, United States

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Beverly Hills, California, United States

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La Jolla, 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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Chicago, Illinois, United States

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Glenview, Illinois, United States

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Indianapolis, Indiana, United States

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Kansas City, Kansas, United States

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Baltimore, Maryland, 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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St Louis, Missouri, United States

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Omaha, Nebraska, United States

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New York, 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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Columbus, Ohio, United States

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Philadelphia, Pennsylvania, United States

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Nashville, Tennessee, United States

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Dallas, Texas, United States

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Salt Lake City, Utah, United States

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Rosario, S, Argentina

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Rosario, S, Argentina

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Rosario, S, Argentina

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Córdoba, X, Argentina

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Vienna, , Austria

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Liège, LIE, Belgium

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Hasselt, LIM, Belgium

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Aalst, OVL, Belgium

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Roeselare, WVL, Belgium

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Rio de Janeiro, Rio de Janeiro, Brazil

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Ribeirão Preto, São Paulo, Brazil

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Ribeirão Preto, São Paulo, Brazil

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São Paulo, São Paulo, Brazil

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São Paulo, São Paulo, Brazil

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Prague, , Czechia

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Prague, , Czechia

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Prague, , Czechia

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Aarhus N, , Denmark

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København Ø, , Denmark

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Odense C, , Denmark

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Créteil, Val-de-Marne, France

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Marseille, , France

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Paris, , France

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Rennes, , France

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Toulouse, , France

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Bologna, Emilia-Romagna, Italy

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Florence, , Italy

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Pavia, , Italy

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Maastricht, LI, Netherlands

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Groningen, , Netherlands

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Utrecht, , Netherlands

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Oslo, , Norway

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Creixomil, , Portugal

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Lisbon, , Portugal

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Viseu, , Portugal

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Barcelona, B, Spain

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L'Hospitalet de Llobregat, B, Spain

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Madrid, , Spain

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Gothenburg, , Sweden

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Solna, , Sweden

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Umeå, , Sweden

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Taipei, , Taiwan

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Birmingham, BIR, United Kingdom

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London, LND, United Kingdom

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Hexham, NBL, United Kingdom

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Bellshill, , United Kingdom

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Cardiff, , United Kingdom

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Manchester, , United Kingdom

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Countries

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United States Argentina Austria Belgium Brazil Czechia Denmark France Italy Netherlands Norway Portugal Spain Sweden Taiwan United Kingdom

References

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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.

Reference Type RESULT
PMID: 39213194 (View on PubMed)

Related Links

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Other Identifiers

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2024-516568-27

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

ALN-TTRSC02-007

Identifier Type: -

Identifier Source: org_study_id