NCT07052903

Brief Summary

The purpose of this study is to:

  • Evaluate the efficacy of nucresiran compared to placebo on reducing all-cause mortality and cardiovascular (CV) events
  • Evaluate the efficacy of nucresiran compared to placebo on additional assessments of CV events and/or death
  • Evaluate the efficacy of nucresiran compared to placebo on patient-reported health status and health-related quality of life

Trial Health

88
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Strong global presence with extensive site network
Enrollment
1,250

participants targeted

Target at P75+ for phase_3

Timeline
80mo left

Started Jul 2025

Longer than P75 for phase_3

Geographic Reach
31 countries

201 active sites

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

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Study Timeline

Key milestones and dates

Study Progress11%
Jul 2025Nov 2032

First Submitted

Initial submission to the registry

June 30, 2025

Completed
2 days until next milestone

Study Start

First participant enrolled

July 2, 2025

Completed
6 days until next milestone

First Posted

Study publicly available on registry

July 8, 2025

Completed
4.9 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

May 28, 2030

Expected
2.5 years until next milestone

Study Completion

Last participant's last visit for all outcomes

November 30, 2032

Last Updated

April 17, 2026

Status Verified

April 1, 2026

Enrollment Period

4.9 years

First QC Date

June 30, 2025

Last Update Submit

April 16, 2026

Conditions

Keywords

ATTR-CMATTR amyloidosis with cardiomyopathyATTRHeriditary ATTRhATTRWild-type ATTRwATTRCardiomyopathyAmyloidosisTTRTransthyretinTTR-mediated amyloidosisRNAiRNAi therapeuticTTR cardiomyopathyV122iTTR amyloidosis

Outcome Measures

Primary Outcomes (1)

  • Composite outcome of all-cause mortality and recurrent cardiovascular [CV] events (CV hospitalizations and urgent heart failure [HF] visits)

    All-cause mortality and recurrent CV events (CV hospitalizations and urgent HF visits) will be compared between treatment groups using an Andersen-Gill model.

    Baseline to end of double-blind period (estimated 32 months, maximum 5 years)

Secondary Outcomes (4)

  • Time to first CV event (CV hospitalizations and urgent HF visits) or all-cause mortality

    Baseline to end of double-blind period (estimated 32 months, maximum 5 years)

  • All-cause mortality

    Baseline to end of double-blind period (estimated 32 months, maximum 5 years)

  • Recurrent CV events (CV hospitalizations and urgent HF visits)

    Baseline to end of double-blind period (estimated 32 months, maximum 5 years)

  • Change from baseline in Kansas City Cardiomyopathy Questionnaire Overall Summary (KCCQ-OS)

    Baseline to Month 30

Study Arms (2)

Placebo

PLACEBO COMPARATOR

Participants will receive placebo administered subcutaneously (SC) once every 6 months (q6M) during the double-blind (DB) period, followed by nucresiran 300 mg administered SC q6M during the open-label extension (OLE) period.

Drug: NucresiranDrug: Sterile Normal Saline (0.9% NaCl)

Nucresiran 300 mg

EXPERIMENTAL

Participants will receive nucresiran 300 mg administered SC Q6M during the DB period, followed by nucresiran 300 mg administered SC q6M during the OLE period.

Drug: Nucresiran

Interventions

Sterile Normal Saline (0.9% NaCl) administered SC once q6M

Placebo

Nucresiran 300 mg administered SC q6M

Also known as: ALN-TTRSC04
Nucresiran 300 mgPlacebo

Eligibility Criteria

Age18 Years - 85 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Has documented diagnosis of ATTR amyloidosis with cardiomyopathy including those with hereditary ATTR (hATTR) or wild-type ATTR (wATTR) amyloidosis.
  • Has medical history of heart failure (HF) with at least 1 prior hospitalization for HF or signs and symptoms that require treatment with a diuretic.
  • Has screening N-terminal prohormone B-type natriuretic peptide (NT-proBNP) \>300 ng/L and \<8500 ng/L; In patients with permanent or persistent atrial fibrillation, screening NT-proBNP \>600 ng/L and \<8500 ng/L.
  • Patients may be receiving approved TTR stabilizers for ATTR amyloidosis (eg, tafamidis, acoramidis) and may be receiving background therapy for HF at the discretion of the Investigator.

You may not qualify if:

  • Has New York Heart Association (NYHA) Class IV HF; or NYHA Class III heart failure AND ATTR Amyloidosis Disease Stage 3.
  • Has a polyneuropathy disability (PND) Score IIIa, IIIb, or IV.
  • Has an estimated glomerular filtration rate eGFR of \<30 mL/min/1.73m\^2 at screening.
  • Has received prior or currently receiving TTR-lowering therapy

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (207)

Clinical Trial Site

La Jolla, California, 92037, United States

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Stanford, California, 94305, United States

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Norwich, Connecticut, 06360, United States

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

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Brandon, Florida, 33511, United States

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Miami, Florida, 33125, United States

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Atlanta, Georgia, 30309, United States

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Gainesville, Georgia, 30501, United States

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Tucker, Georgia, 30084, United States

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Indianapolis, Indiana, 46202, United States

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Indianapolis, Indiana, 46260, 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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Detroit, Michigan, 48202, 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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Manhasset, New York, 11030, 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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New York, New York, 10065, United States

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The Bronx, New York, 10467, United States

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Charlotte, North Carolina, 28204, United States

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Cleveland, Ohio, 44195, United States

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Lancaster, Pennsylvania, 17602, United States

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Philadelphia, Pennsylvania, 19104, United States

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Dallas, Texas, 75246, United States

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Houston, Texas, 77030, United States

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Plano, Texas, 75024, United States

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Salt Lake City, Utah, 84132, United States

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Charlottesville, Virginia, 22903, United States

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Norfolk, Virginia, 23507, United States

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Spokane, Washington, 99204, United States

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Buenos Aires, C1025ABI, Argentina

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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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Rosario, S2000DEJ, Argentina

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Rosario, S2000DSR, Argentina

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Rosario, S2000GAP, Argentina

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Adelaide, 5000, Australia

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Bedford Park, 5042, Australia

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Box Hill, 3128, Australia

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Chermside, 4032, Australia

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Darlinghurst, 2010, Australia

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Joondalup, 6027, Australia

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Westmead, 2145, Australia

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Braunau am Inn, 5280, Austria

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Graz, 8036, Austria

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Vienna, 1090, Austria

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Vienna, 1160, Austria

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Vienna, 1210, Austria

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Wels, 4600, Austria

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Aalst, 9300, Belgium

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Bruges, 8000, Belgium

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Brussels, 1200, Belgium

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Dendermonde, 9200, Belgium

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Genk, 3600, 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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Belo Horizonte, 30220-140, Brazil

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Porto Alegre, 90035-903, Brazil

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Porto Alegre, 90560-032, Brazil

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Ribeirão Preto, 14026-900, Brazil

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Ribeirão Preto, 14051-140, Brazil

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Salvador, 40170-130, Brazil

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São Paulo, 04038-002, Brazil

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São Paulo, 05403-000, Brazil

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London, N6A 5A5, Canada

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Santiago, 7500588, Chile

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Santiago, 8320000, Chile

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Dongcheng, 100006, China

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Brno, 602 00, Czechia

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Ostrava, 708-52, Czechia

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Prague, 128 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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Chambray-lès-Tours, 37170, France

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Dijon, 21000, France

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La Tronche, 38700, France

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Marseille, 13005, France

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Martinique, 97261, France

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Montpellier, 34295, France

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Paris, 75015, France

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Paris, 75018, France

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Pessac, 33600, France

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Reims, 51100, France

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Rennes, 35000, France

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Rouen, 76031, France

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Saint-Herblain, 44093, France

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Strasbourg, 67091, France

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Toulouse, 31059, France

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Berlin, 13353, Germany

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Cologne, 50937, Germany

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Göttingen, 37075, Germany

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Hanover, 30625, Germany

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Leipzig, 04103, Germany

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Munich, 81377, Germany

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Münster, 48149, Germany

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Würzburg, 97080, Germany

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Athens, 115 27, Greece

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Athens, 115 28, Greece

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Heraklion, 714 09, Greece

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Thessaloniki, 546 36, Greece

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Hong Kong, 999077, Hong Kong

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Budapest, 1085, Hungary

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Be’er Ya‘aqov, 70300, Israel

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Haifa, 3109601, Israel

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Jerusalem, 91120, Israel

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Petah Tikva, 4941492, Israel

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Ancona, 60126, Italy

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Bologna, 40138, Italy

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Brescia, 25123, Italy

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Florence, 50134, Italy

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Forlì, 47121, Italy

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Milan, 20122, Italy

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Naples, 80131, Italy

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Padova, 35128, Italy

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Pavia, 27100, Italy

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Pisa, 56124, Italy

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Rimini, 47923, Italy

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Roma, 00168, Italy

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Rome, 00161, Italy

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Torino, 10126, Italy

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Bunkyō City, 113-8655, Japan

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Fukuoka, 810-0001, Japan

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Fukuoka, 812-8582, Japan

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Kagawa, 761-0793, Japan

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Kashihara, 634-8522, Japan

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Kobe, 650-0017, Japan

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Kumamoto, 860-8556, Japan

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Kurume, 830-0011, Japan

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Matsumoto-shi, 390-8621, Japan

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Nagoya, 466-8560, Japan

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Nankoku, 783-0043, Japan

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Niigata, 951-8520, Japan

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Okayama, 700-8558, Japan

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Sapporo, 060-8543, Japan

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Sapporo, 060-8648, Japan

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Sendai, 980-8574, Japan

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Suita, 564-8565, Japan

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Tokyo, 160-8582, Japan

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Kajang, 43000, Malaysia

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Kuala Lumpur, 59100, Malaysia

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Eindhoven, 5623 EJ, Netherlands

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Groningen, 9713 GZ, Netherlands

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Maastricht, 6229 HX, Netherlands

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Rotterdam, 3015 GD, Netherlands

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Utrecht, 3584 CX, Netherlands

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Christchurch, 8011, New Zealand

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Hamilton, 3204, New Zealand

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Ålesund, 6017, Norway

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Lørenskog, 1478, Norway

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Carnaxide, 2790-134, Portugal

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Creixomil, 4835-044, Portugal

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Leiria, 2410-197, Portugal

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Lisbon, 1169-024, Portugal

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Lisbon, 1649-035, Portugal

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Vila Real, 5000-508, Portugal

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Viseu, 3504-509, Portugal

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Bucharest, 022328, Romania

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Cluj-Napoca, 400001, Romania

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Košice, 040 11, Slovakia

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Incheon, 22332, South Korea

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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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Seoul, 06591, South Korea

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A Coruña, 15006, Spain

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Barcelona, 08035, Spain

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Barcelona, 08036, Spain

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Bilbao, 48013, Spain

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El Palmar, 30120, Spain

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Huelva, 21005, Spain

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Jaén, 23007, Spain

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L'Hospitalet de Llobregat, 8907, Spain

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Lleida, 25198, Spain

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Madrid, 28046, Spain

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Madrid, 28222, Spain

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Palma de Mallorca, 07198, Spain

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Pamplona, 31008, Spain

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Salamanca, 37007, Spain

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Santiago de Compostela, 15706, Spain

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Valencia, 46010, Spain

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Valencia, 46026, Spain

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Vigo, 36213, Spain

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Gothenburg, 413 45, Sweden

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Lund, 222 42, Sweden

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Solna, 171 76, Sweden

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Basel, 4051, Switzerland

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Geneva, 1205, Switzerland

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Lucerne, 6000, Switzerland

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Sankt Gallen, 9007, Switzerland

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New Taipei City, 220, Taiwan

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Taipei, 10002, Taiwan

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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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London, NW10 2PB, United Kingdom

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London, NW3 2QG, United Kingdom

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Manchester, M15 6SE, United Kingdom

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MeSH Terms

Conditions

CardiomyopathiesAmyloidosis

Condition Hierarchy (Ancestors)

Heart DiseasesCardiovascular DiseasesProteostasis DeficienciesMetabolic DiseasesNutritional and Metabolic Diseases

Study Officials

  • Medical Director

    Alnylam Pharmaceuticals

    STUDY DIRECTOR

Central Study Contacts

Clinical Trial Information Line

CONTACT

Clinical Trial Information Line

CONTACT

Study Design

Study Type
interventional
Phase
phase 3
Allocation
RANDOMIZED
Masking
QUADRUPLE
Who Masked
PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

June 30, 2025

First Posted

July 8, 2025

Study Start

July 2, 2025

Primary Completion (Estimated)

May 28, 2030

Study Completion (Estimated)

November 30, 2032

Last Updated

April 17, 2026

Record last verified: 2026-04

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.

Locations