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Hope for rare heart disease: new drug aims to slow deadly protein clumps

NCT ID NCT04153149

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

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing This study
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study tests a drug called vutrisiran in 655 adults with a rare heart condition caused by abnormal protein buildup (ATTR amyloidosis with cardiomyopathy). The drug is given as a shot every 3 months and aims to reduce deaths and heart-related hospital stays. The goal is to see if it can help control the disease and improve how far patients can walk in 6 minutes.

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

655 people

The number who actually took part.

Started

Nov 2019

Expected to finish

Dec 2026

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 a documented diagnosis of transthyretin (ATTR) amyloidosis with cardiomyopathy, classified as either hereditary ATTR (hATTR) amyloidosis with cardiomyopathy or wild-type ATTR (wtATTR) amyloidosis with cardiomyopathy meeting pre-specified diagnostic criteria * Has medical history of heart failure (HF) with at least 1 prior hospitalization for HF OR clinical evidence of HF Exclusion Criteria: * Has known primary amyloidosis or leptomeningeal amyloidosis * Has New York Heart Association (NYHA) Class IV heart failure * Has NYHA Class III heart failure AND is at high risk based on pre-specified criteria * Has a polyneuropathy disability (PND) Score IIIa, IIIb, or IV at the Screening visit * Has estimated glomerular filtration rate (eGFR) \<30 mL/min/1.73 m\^2 * Has received prior TTR-lowering treatment * Has other non-TTR cardiomyopathy, hypertensive cardiomyopathy, cardiomyopathy due to valvular heart disease, or cardiomyopathy due to ischemic heart disease

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Conditions

The condition(s) this trial relates to.

amyloidosis ATTRV122I amyloidosis cardiomyopathy familial amyloid neuropathy hereditary amyloidosis wild type ATTR amyloidosis

As listed by the trial registrant

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

Transthyretin amyloidosis (ATTR) with cardiomyopathy

Contacts and locations

Locations

  • Clinical Trial Site

    Houston, Texas, 77030, United States

  • Clinical Trial Site

    Buenos Aires, Ciudad Autónoma de BuenosAires, C1093AAS, Argentina

  • Clinical Trial Site

    Buenos Aires, Pilar, B1629ODT, Argentina

  • Clinical Trial Site

    Buenos Aires, C1199ABB, Argentina

  • Clinical Trial Site

    Buenos Aires, C1428ART, Argentina

  • Clinical Trial Site

    Darlinghurst, New South Wales, 2010, Australia

  • Clinical Trial Site

    Northmead, New South Wales, 2152, Australia

  • Clinical Trial Site

    Woolloongabba, Queensland, 4102, Australia

  • Clinical Trial Site

    Adelaide, South Australia, 5000, Australia

  • Clinical Trial Site

    Melbourne, Victoria, 3004, Australia

  • Clinical Trial Site

    Graz, 8036, Austria

  • Clinical Trial Site

    Vienna, 1090, Austria

  • Clinical Trial Site

    Vienna, 1210, Austria

  • Clinical Trial Site

    Dendermonde, Oost-Vlaanderen, 9200, Belgium

  • Clinical Trial Site

    Ghent, Oost-Vlaanderen, 9000, Belgium

  • Clinical Trial Site

    Brussels, 1070, Belgium

  • Clinical Trial Site

    Leuven, 3000, Belgium

  • Clinical Trial Site

    Toronto, Ontario, M5G 2C4, Canada

  • Clinical Trial Site

    Québec, Quebec, G1V4G5, Canada

  • Clinical Trial Site

    Zagreb, 10000, Croatia

  • Clinical Trial Site

    Olomouc, Olomouc Region, 779 00, Czechia

  • Clinical Trial Site

    Prague, Praha, Hlavní Mesto, 12000, Czechia

  • Clinical Trial Site

    Odense, Region Syddanmark, 5000, Denmark

  • Clinical Trial Site

    Aarhus N, 8200, Denmark

  • Clinical Trial Site

    Créteil, 94000, France

  • Clinical Trial Site

    Marseille, 13385, France

  • Clinical Trial Site

    Paris, 75877, France

  • Clinical Trial Site

    Toulouse, 31059, France

  • Clinical Trial Site

    Frankfurt am Main, Hesse, 60313, Germany

  • Clinical Trial Site

    Leipzig, Saxony, 04103, Germany

  • Clinical Trial Site

    Essen, 45122, Germany

  • Clinical Trial Site

    Göttingen, 37075, Germany

  • Clinical Trial Site

    Heidelberg, 69120, Germany

  • Clinical Trial Site

    Münster, 48149, Germany

  • Clinical Trial Site

    Würzburg, 97078, Germany

  • Clinical Trial Site

    Budapest, 1088, Hungary

  • Clinical Trial Site

    Budapest, 1134, Hungary

  • Clinical Trial Site

    Szeged, 6725, Hungary

  • Clinical Trial Site

    Dublin, D07 A8NN, Ireland

  • Clinical Trial Site

    Dublin, Dublin 8, Ireland

  • Clinical Trial Site

    Haifa, 3339419, Israel

  • Clinical Trial Site

    Jerusalem, 9112000, Israel

  • Clinical Trial Site

    Ramat Gan, 5262000, Israel

  • Clinical Trial Site

    Tsurumai-cho, Aichi-ken, 466-8560, Japan

  • Clinical Trial Site

    Fukuoka, Fukuoka, 810-0001, Japan

  • Clinical Trial Site

    Kurume, Fukuoka, 830-0011, Japan

  • Clinical Trial Site

    Kita-gun, Kagawa-ken, 761-0793, Japan

  • Clinical Trial Site

    Nankoku, Kochi, 783-8505, Japan

  • Clinical Trial Site

    Matsumoto, Nagano, 390-8621, Japan

  • Clinical Trial Site

    Kashihara, Nara, 634-8522, Japan

  • Clinical Trial Site

    Suita, Osaka, 564-8565, Japan

  • Clinical Trial Site

    Suita, Osaka, 565-0871, Japan

  • Clinical Trial Site

    Bunkyo-ku, Tokyo, 113-8431, Japan

  • Clinical Trial Site

    Bunkyo-ku, Tokyo, 113-8655, Japan

  • Clinical Trial Site

    Shinjuku-Ku, Tokyo, 160-8582, Japan

  • Clinical Trial Site

    Fukuoka, 812-8582, Japan

  • Clinical Trial Site

    Kumamoto, 860-8556, Japan

  • Clinical Trial Site

    Riga, LV-1002, Latvia

  • Clinical Trial Site

    Hamra, Beirut, 1111, Lebanon

  • Clinical Trial Site

    Kaunas, LT-50161, Lithuania

  • Clinical Trial Site

    Kuantan, Pahang, 25100, Malaysia

  • Clinical Trial Site

    Sungai Buloh, Selangor, 47000, Malaysia

  • Clinical Trial Site

    Chisinau, MD2025, Moldova

  • Clinical Trial Site

    Eindhoven, 5623 EJ, Netherlands

  • Clinical Trial Site

    Groningen, 9713 GZ, Netherlands

  • Clinical Trial Site

    Utrecht, 3584 CW, Netherlands

  • Clinical Trial Site

    Oslo, 0424, Norway

  • Clinical Trial Site

    San Isidro, Lima region, 15076, Peru

  • Clinical Trial Site

    Lima, 15088, Peru

  • Clinical Trial Site

    Wroclaw, Lower Silesian Voivodeship, 50-556, Poland

  • Clinical Trial Site

    Lodz, Lódzkie, 90-127, Poland

  • Clinical Trial Site

    Warsaw, Masovian Voivodeship, 01-192, Poland

  • Clinical Trial Site

    Gdansk, Pomeranian Voivodeship, 80-382, Poland

  • Clinical Trial Site

    Katowice, 40-555, Poland

  • Clinical Trial Site

    Lisbon, Lisbon District, 1649-035, Portugal

  • Clinical Trial Site

    Coimbra, 3000-075, Portugal

  • Clinical Trial Site

    Faro, 8000-386, Portugal

  • Clinical Trial Site

    Guimarães, 4835-044, Portugal

  • Clinical Trial Site

    Porto, 4099-001, Portugal

  • Clinical Trial Site

    Mecca, Mecca Region, 21955, Saudi Arabia

  • Clinical Trial Site

    Riyadh, 12372, Saudi Arabia

  • Clinical Trial Site

    Ljubljana, 1000, Slovenia

  • Clinical Trial Site

    Seoul, 03080, South Korea

  • Clinical Trial Site

    Seoul, 03722, South Korea

  • Clinical Trial Site

    Seoul, 06351, South Korea

  • Clinical Trial Site

    Seoul, 06591, South Korea

  • Clinical Trial Site

    L'Hospitalet de Llobregat, Barcelona, 08907, Spain

  • Clinical Trial Site

    Majadahonda, Madrid, 28222, Spain

  • Clinical Trial Site

    Barcelona, 08035, Spain

  • Clinical Trial Site

    Bilbao, 48013, Spain

  • Clinical Trial Site

    Huelva, 21005, Spain

  • Clinical Trial Site

    Málaga, 29010, Spain

  • Clinical Trial Site

    Göteborg, Västerbotten County, 422 46, Sweden

  • Clinical Trial Site

    Gothenburg, 422 46, Sweden

  • Clinical Trial Site

    Chiang Mai, 50200, Thailand

  • Clinical Trial Site

    Bellshill, Lanarkshire, ML4 3NJ, United Kingdom

  • Clinical Trial Site

    London, London, City of, SE1 1YR, United Kingdom

  • Clinical Trial Site

    Londonderry, Londonderry, BT47 6SB, United Kingdom

  • Clinical Trial Site

    Hexham, Northumberland, NE46 1QJ, United Kingdom

  • Clinical Trial Site

    Birmingham, B15 2SQ, United Kingdom

  • Clinical Trial Site

    Cardiff, CF15 9SS, United Kingdom

  • Clinical Trial Site

    London, NW3 2QG, United Kingdom

  • Clinical Trial Site

    Manchester, M15 6SE, United Kingdom

  • Clinical Trial Site

    La Mesa, California, 91942, United States

  • Clinical Trial Site

    Los Angeles, California, 90033, United States

  • Clinical Trial Site

    Stanford, California, 94305, United States

  • Clinical Trial Site

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

  • Clinical Trial Site

    Gainesville, Georgia, 30501, United States

  • Clinical Trial Site

    Chicago, Illinois, 60637, United States

  • Clinical Trial Site

    Evanston, Illinois, 60201, United States

  • Clinical Trial Site

    Boston, Massachusetts, 02115, United States

  • Clinical Trial Site

    Boston, Massachusetts, 02118, United States

  • Clinical Trial Site

    Rochester, Minnesota, 55902, United States

  • Clinical Trial Site

    Manhasset, New York, 11030, United States

  • Clinical Trial Site

    New York, New York, 10032, United States

  • Clinical Trial Site

    Durham, North Carolina, 27710, United States

  • Clinical Trial Site

    Cleveland, Ohio, 44195, United States

  • Clinical Trial Site

    Allentown, Pennsylvania, 18103, United States

  • Clinical Trial Site

    Philadelphia, Pennsylvania, 19104, United States

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