New drug aims to cut deaths and heart crises in rare amyloidosis

NCT ID NCT07052903

Disease control Sponsor: Alnylam Pharmaceuticals Source: ClinicalTrials.gov ↗

First seen Jun 24, 2026 · Last updated Sep 17, 2026 · Updated 6 times

Summary

This Phase 3 study tests a drug called nucresiran in 1250 people with ATTR amyloidosis, a rare disease where abnormal proteins damage the heart. Participants receive either nucresiran or a placebo every 6 months. The goal is to see if the drug reduces deaths and heart-related hospitalizations.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance: nucresiran
What this could lead to: If it works, this could provide a new treatment option to reduce deaths and heart-related hospitalizations for people with ATTR amyloidosis and heart problems.
What could go wrong: This is a large Phase 3 trial, but the drug is still experimental. It may not prove better than placebo, and side effects are possible. Results will take time.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

About 1,750 people

The number the study aims to enrol. It can still change while the study runs.

Started

Jul 2025

Expected to finish

Nov 2032

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 to 85 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria * 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. Exclusion Criteria * 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

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Get notified about this study

Sign up to get updates when this study changes or when new studies for Transthyretin amyloidosis with cardiomyopathy are added.

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Conditions

The condition(s) this trial relates to.

amyloidosis cardiomyopathy wild type ATTR amyloidosis

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Transthyretin amyloidosis with cardiomyopathy

How to take part

Only the study team decides who joins. These are the ways to reach them.

The places running it

265 sites in 36 countries. The list below names each one and where it is.
The official record

ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

Open the record ↗
A doctor treating you

A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

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La Jolla, California, 92037, United States
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Laguna Hills, California, 92653, United States
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Stanford, California, 94305, United States
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Aurora, Colorado, 80045, 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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Chicago, Illinois, 60612, United States
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Evanston, Illinois, 60201, 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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Columbus, Ohio, 43210, United States
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Portland, Oregon, 97239, United States
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Lancaster, Pennsylvania, 17602, United States
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Philadelphia, Pennsylvania, 19104, United States
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Charleston, South Carolina, 29425, United States
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Austin, Texas, 78705, 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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Falls Church, Virginia, 22042, United States
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Norfolk, Virginia, 23507, United States
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Richmond, Virginia, 23298, United States
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Seattle, Washington, 98195, 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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Córdoba, X5016KET, 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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Liverpool, 2170, 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, 1100, 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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Edegem, 2650, 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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Campinas, 13083-888, Brazil
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Porto Alegre, 90035-903, Brazil
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Porto Alegre, 90560-032, Brazil
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Recife, 50070-480, Brazil
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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, 20551-030, Brazil
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Rio de Janeiro, 22280-020, 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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Calgary, T2N 4N1, Canada
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London, N6A 5A5, Canada
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Toronto, M4P 1E4, Canada
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Vancouver, V6Z 1Y6, Canada
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Santiago, 7500588, Chile
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Santiago, 8320000, Chile
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Binhai, 300456, China
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Changsha, 410011, China
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Changsha, 410013, China
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Chengdu, 610041, China
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Dongcheng, 100006, China
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Fuzhou, 350004, China
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Guangzhou, 510080, China
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Haidian, 100191, China
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Hangzhou, 310009, China
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Hangzhou, 310014, China
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Hefei, 230061, China
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Hexi District, 300211, China
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Jinan, 271000, China
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Nanchang, 330030, China
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Nanjing, 210029, China
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Shenzhen Shi, 518053, China
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Taiyuan, 030012, China
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Wuhan, 430064, China
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Xicheng, 100034, China
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Yuanjiagang, 400016, 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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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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Chambray-lès-Tours, 37170, France
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Créteil, 94000, 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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Dresden, 01307, Germany
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Göttingen, 37075, Germany
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Hanover, 30625, Germany
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Heidelberg, 69120, Germany
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Leipzig, 04103, Germany
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Magdeburg, 39120, 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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Balatonfüred, 8230, Hungary
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Budapest, 1085, Hungary
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Dublin, D07 R2WY, Ireland
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Dublin, D08 NHY1, Ireland
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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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Lecce, 73100, 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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Tlalpan, 14080, Mexico
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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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Auckland, 2025, New Zealand
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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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Lodz, 93-338, Poland
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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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Bern, 3010, 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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Taichung, 40705, Taiwan
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Taipei, 10002, Taiwan
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Taipei, 112, Taiwan
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Ankara, 06170, Turkey (Türkiye)
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Antalya, 07070, Turkey (Türkiye)
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Istanbul, 34093, Turkey (Türkiye)
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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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