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Massive global study aims to unlock secrets of rare heart and nerve disease

NCT ID NCT04561518

Knowledge-focused 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 study
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
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 24, 2026 · Last updated Sep 09, 2026 · Updated 4 times

Summary

This observational study will follow 1,500 people with ATTR amyloidosis, a rare disease that damages the heart and nerves, for many years. Researchers will track how the disease progresses, how patients are treated in real-world settings, and the safety of two approved drugs, patisiran and vutrisiran. The goal is to better understand the condition and improve patient care.

What this could mean

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

What this could lead to
If successful, this study could provide a clearer picture of how ATTR amyloidosis progresses and how well current treatments work in everyday life, helping doctors manage the disease better.
What could go wrong
This is an observational study, not a treatment trial, so it won't directly test new therapies. Results may take years and might not lead to immediate changes in care.

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.

Participants

About 1,500 people

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

Started

Nov 2020

Expected to finish

Dec 2033

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.

Who is studied

Patients with a diagnosis of ATTR amyloidosis, hereditary or wild type, and pre-symptomatic carriers with a known disease-causing TTR variant will be eligible for the study.

Ages

Children (under 18), adults (18 to 64) and older adults (65 and over)

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: * Diagnosis of ATTR amyloidosis or documented known disease-causing TTR variant for the cohort of pre-symptomatic carriers * Germany Only: Patients must be treated per the summary of product characteristics (SmPC) for any approved treatment for ATTR amyloidosis Exclusion Criteria: * Current enrollment in a clinical trial for any investigational agent

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Conditions

The condition(s) this trial relates to.

Amyloid Neuropathies Amyloid Neuropathies, Familial amyloidosis familial amyloid neuropathy hereditary amyloidosis polyneuropathy

As listed by the trial registrant

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

Attr amyloidosis Transthyretin-mediated amyloidosis

How to take part

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

  1. The places running it

    38 sites in 12 countries. The list below names each one and where it is.

  2. 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 ↗

  3. 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

  • Clinical Trial Site

    RECRUITING

    La Jolla, California, 92037, United States

  • Clinical Trial Site

    RECRUITING

    Los Angeles, California, 90095, United States

  • Clinical Trial Site

    RECRUITING

    Jacksonville, Florida, 32224, United States

  • Clinical Trial Site

    RECRUITING

    Iowa City, Iowa, 52242, United States

  • Clinical Trial Site

    RECRUITING

    Kansas City, Kansas, 66160, United States

  • Clinical Trial Site

    RECRUITING

    Baltimore, Maryland, 21224, United States

  • Clinical Trial Site

    COMPLETED

    Boston, Massachusetts, 02127, United States

  • Clinical Trial Site

    RECRUITING

    New York, New York, 10034, United States

  • Clinical Trial Site

    RECRUITING

    Durham, North Carolina, 27710, United States

  • Clinical Trial Site

    RECRUITING

    Columbus, Ohio, 43210, United States

  • Clinical Trial Site

    RECRUITING

    Philadelphia, Pennsylvania, 19104, United States

  • Clinical Trial Site

    RECRUITING

    Austin, Texas, 78756, United States

  • Clinical Trial Site

    RECRUITING

    Houston, Texas, 77030, United States

  • Clinical Trial Site

    RECRUITING

    Salvador, Brazil

  • Clinical Trial Site

    RECRUITING

    São Paulo, Brazil

  • Clinical Trial Site

    RECRUITING

    Sofia, Bulgaria

  • Clinical Trial Site

    RECRUITING

    Aarhus, Denmark

  • Clinical Trial Site

    COMPLETED

    Copenhagen, Denmark

  • Clinical Trial Site

    RECRUITING

    Bordeaux, France

  • Clinical Trial Site

    RECRUITING

    Bron, France

  • Clinical Trial Site

    RECRUITING

    Créteil, France

  • Clinical Trial Site

    RECRUITING

    Le Kremlin-Bicêtre, France

  • Clinical Trial Site

    RECRUITING

    Marseille, France

  • Clinical Trial Site

    RECRUITING

    Berlin, Germany

  • Clinical Trial Site

    RECRUITING

    Giessen, Germany

  • Clinical Trial Site

    RECRUITING

    Hanover, Germany

  • Clinical Trial Site

    RECRUITING

    Jerusalem, Israel

  • Clinical Trial Site

    RECRUITING

    Ramat Gan, Israel

  • Clinical Trial Site

    RECRUITING

    Milan, Italy

  • Clinical Trial Site

    RECRUITING

    Naples, Italy

  • Clinical Trial Site

    RECRUITING

    Palermo, Italy

  • Clinical Trial Site

    RECRUITING

    Roma, Italy

  • Clinical Trial Site

    RECRUITING

    Groningen, Netherlands

  • Clinical Trial Site

    RECRUITING

    Lisbon, Portugal

  • Clinical Trial Site

    RECRUITING

    Porto, Portugal

  • Clinical Trial Site

    RECRUITING

    Barcelona, Spain

  • Clinical Trial Site

    RECRUITING

    Madrid, Spain

  • Clinical Trial Site

    RECRUITING

    Palma, Spain

  • Clinical Trial Site

    RECRUITING

    New Taipei City, Taiwan

  • Clinical Trial Site

    RECRUITING

    Taipei, Taiwan

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