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New drug patisiran aims to help hearts stiffened by rare protein disease

NCT ID NCT03997383

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
Running, but no longer taking on new participants.
Completed This study
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 phase 3 trial tested the drug patisiran in 360 people with ATTR amyloidosis with cardiomyopathy, a condition where abnormal protein builds up in the heart, making it stiff and weak. Participants received either patisiran or a placebo intravenously. The study measured how far they could walk in 6 minutes and their heart-related quality of life after 12 months to see if the drug helps.

What this could mean

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

Active substance
Patisiran
What this could lead to
If successful, patisiran could become a new treatment option to slow disease progression and improve heart function in people with ATTR amyloidosis with cardiomyopathy.
What could go wrong
This is a single phase 3 trial; results may not confirm benefit or may show limited improvement. As with any drug, there are risks of side effects, and it may not work for all patients.

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

360 people

The number who actually took part.

Started

Sep 2019

Finished

Dec 2025

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: * Documented diagnosis of ATTR amyloidosis with cardiomyopathy, classified as either hereditary ATTR amyloidosis with cardiomyopathy or wild-type ATTR amyloidosis with cardiomyopathy * Medical history of heart failure with at least 1 prior hospitalization for heart failure, or current clinical evidence (signs and symptoms of heart failure) * Clinically stable with no cardiovascular related hospitalizations within 6 weeks of study start * Has never taken tafamidis before (tafamidis naïve) or currently on tafamidis for ≥6 months with evidence of disease progression while on tafamidis treatment * Able to complete ≥150 m on the 6-minute walk test * Screening N-terminal pro B-type natriuretic peptide (NT-proBNP), a blood marker of heart failure severity, \>300 ng/L and \<8500 ng/L; in participants with permanent or persistent atrial fibrillation, screening NT-proBNP\> 600 ng/L and \<8500 ng/L Exclusion Criteria: * Known primary amyloidosis (AL) or leptomeningeal amyloidosis. * Received prior TTR lowering treatment * New York Heart Association heart failure classification of III and at high risk * New York Heart Association heart failure classification of IV * Neuropathy requiring cane or stick to walk, or is wheelchair bound * Estimated glomerular filtration rate (eGFR) \<30 mL/min/1.73m\^2 * Abnormal liver function * Has hepatitis B, hepatitis C or human immunodeficiency virus (HIV) infection * Has non-amyloid disease that significantly affects ability to walk (e.g., severe chronic obstructive pulmonary disease, severe arthritis, or peripheral vascular disease affecting ambulation) * Prior or planned heart, liver, or other organ transplant * Other cardiomyopathy not related to ATTR amyloidosis

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Conditions

The condition(s) this trial relates to.

amyloidosis ATTRV122I amyloidosis cardiomyopathy familial amyloid neuropathy 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

    Los Angeles, California, 90048, United States

  • Clinical Trial Site

    Chicago, Illinois, 60637, United States

  • Clinical Trial Site

    Skokie, Illinois, 60076, United States

  • Clinical Trial Site

    Iowa City, Iowa, 52242, United States

  • Clinical Trial Site

    Kansas City, Kansas, 66160, United States

  • Clinical Trial Site

    Baltimore, Maryland, 21224, United States

  • Clinical Trial Site

    Boston, Massachusetts, 02118, United States

  • Clinical Trial Site

    Burlington, Massachusetts, 10805, United States

  • Clinical Trial Site

    Rochester, Minnesota, 55905, United States

  • Clinical Trial Site

    St Louis, Missouri, 63110, United States

  • Clinical Trial Site

    New York, New York, 10029, United States

  • Clinical Trial Site

    New York, New York, 10032, United States

  • Clinical Trial Site

    Cleveland, Ohio, 44195, United States

  • Clinical Trial Site

    Philadelphia, Pennsylvania, 19104, United States

  • Clinical Trial Site

    Philadelphia, Pennsylvania, 19140, United States

  • Clinical Trial Site

    Pittsburgh, Pennsylvania, 15212, United States

  • Clinical Trial Site

    Nashville, Tennessee, 37232, United States

  • Clinical Trial Site

    Dallas, Texas, 75246, United States

  • Clinical Trial Site

    Norfolk, Virginia, 23507, United States

  • Clinical Trial Site

    Buenos Aires, C1039AAO, Argentina

  • Clinical Trial Site

    Buenos Aires, C1428AQK, Argentina

  • Clinical Trial Site

    Córdoba, X5000KEH, Argentina

  • Clinical Trial Site

    Rosario, S2000DSR, Argentina

  • Clinical Trial Site

    Rosario, S2000DTC, Argentina

  • Clinical Trial Site

    Rosario, S2000PBJ, Argentina

  • Clinical Trial Site

    San Juan Bautista, B1888AAE, Argentina

  • Clinical Trial Site

    Santa Fe, S3000FWO, Argentina

  • Clinical Trial Site

    Box Hill, 3128, Australia

  • Clinical Trial Site

    Westmead, 2145, Australia

  • Clinical Trial Site

    Woolloongabba, 4021, Australia

  • Clinical Trial Site

    Aalst, 9300, Belgium

  • Clinical Trial Site

    Hasselt, 3500, Belgium

  • Clinical Trial Site

    Liège, 4000, Belgium

  • Clinical Trial Site

    Roeselare, 8800, Belgium

  • Clinical Trial Site

    Porto Alegre, 90035-030, Brazil

  • Clinical Trial Site

    Ribeirão Preto, 14026-900, Brazil

  • Clinical Trial Site

    Ribeirão Preto, 14048-900, Brazil

  • Clinical Trial Site

    Rio de Janeiro, 22280-000, Brazil

  • Clinical Trial Site

    São Paulo, 04012-909, Brazil

  • Clinical Trial Site

    São Paulo, 05403-000, Brazil

  • Clinical Trial Site

    Sofia, 1680, Bulgaria

  • Clinical Trial Site

    Sofia, 1784, Bulgaria

  • Clinical Trial Site

    Stara Zagora, 6000, Bulgaria

  • Clinical Trial Site

    Providencia/ Santiago, 7500587, Chile

  • Clinical Trial Site

    Brno, 656 91, Czechia

  • Clinical Trial Site

    Prague, 120 00, Czechia

  • Clinical Trial Site

    Prague, 128 08, Czechia

  • Clinical Trial Site

    Prague, 140 21, Czechia

  • Clinical Trial Site

    Aarhus N, 8200, Denmark

  • Clinical Trial Site

    Copenhagen, 2100, Denmark

  • Clinical Trial Site

    Odense C, 5000, Denmark

  • Clinical Trial Site

    Clichy, 92110, France

  • Clinical Trial Site

    Créteil, 94010, France

  • Clinical Trial Site

    Rennes, 35033, France

  • Clinical Trial Site

    Toulouse, 31059, France

  • Clinical Trial Site

    Lai Chi Kok, 999077, Hong Kong

  • Clinical Trial Site

    Bologna, 40138, Italy

  • Clinical Trial Site

    Florence, 50134, Italy

  • Clinical Trial Site

    Messina, 98125, Italy

  • Clinical Trial Site

    Pavia, 27100, Italy

  • Clinical Trial Site

    Bunkyō City, 113-8655, Japan

  • Clinical Trial Site

    Fukuoka, 812-8582, Japan

  • Clinical Trial Site

    Kumamoto, 860-8556, Japan

  • Clinical Trial Site

    Kurume, 830-0011, Japan

  • Clinical Trial Site

    Matsumoto, 390-8621, Japan

  • Clinical Trial Site

    Nagoya, 466-8560, Japan

  • Clinical Trial Site

    Suita, 565-8565, Japan

  • Clinical Trial Site

    Mexico City, 14080, Mexico

  • Clinical Trial Site

    Groningen, 9713 GZ, Netherlands

  • Clinical Trial Site

    Maastricht, 6229 HX, Netherlands

  • Clinical Trial Site

    Christchurch, 8011, New Zealand

  • Clinical Trial Site

    Hamilton, 3204, New Zealand

  • Clinical Trial Site

    Gdansk, 80-382, Poland

  • Clinical Trial Site

    Katowice, 40-635, Poland

  • Clinical Trial Site

    Lódz, 90-127, Poland

  • Clinical Trial Site

    Warsaw, 01-192, Poland

  • Clinical Trial Site

    Warsaw, 04-628, Poland

  • Clinical Trial Site

    Porto, 4099-001, Portugal

  • Clinical Trial Site

    Senhora da Hora, 4464-513, Portugal

  • Clinical Trial Site

    Viseu, 3504-509, Portugal

  • Clinical Trial Site

    Seoul, 3080, South Korea

  • Clinical Trial Site

    Stockholm, 17164, Sweden

  • Clinical Trial Site

    Taipei, 11217, Taiwan

  • Clinical Trial Site

    Bellshill, ML4 3NJ, United Kingdom

  • Clinical Trial Site

    Birmingham, B15 2SQ, United Kingdom

  • Clinical Trial Site

    Cardiff, CF15 9SS, United Kingdom

  • Clinical Trial Site

    Hexham, NE46 1QJ, United Kingdom

  • Clinical Trial Site

    London, NW3 2PF, United Kingdom

  • Clinical Trial Site

    London, SE1 1YR, United Kingdom

  • Clinical Trial Site

    Manchester, M15 6SE, United Kingdom

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