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

NCT ID NCT04561518

Recruiting now Knowledge-focused Sponsor: Alnylam Pharmaceuticals Source: ClinicalTrials.gov ↗

First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 2 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.

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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

Contacts and locations

Locations

Clinical Trial Site
RECRUITING

La Jolla, California, 92037, United States
Clinical Trial Site
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Los Angeles, California, 90095, United States
Clinical Trial Site
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Jacksonville, Florida, 32224, United States
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Iowa City, Iowa, 52242, United States
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Kansas City, Kansas, 66160, United States
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Baltimore, Maryland, 21224, United States
Clinical Trial Site
COMPLETED

Boston, Massachusetts, 02127, United States
Clinical Trial Site
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New York, New York, 10034, United States
Clinical Trial Site
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Durham, North Carolina, 27710, United States
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Columbus, Ohio, 43210, United States
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Philadelphia, Pennsylvania, 19104, United States
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Austin, Texas, 78756, United States
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Houston, Texas, 77030, United States
Clinical Trial Site
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Salvador, Brazil
Clinical Trial Site
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São Paulo, Brazil
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Sofia, Bulgaria
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Aarhus, Denmark
Clinical Trial Site
COMPLETED

Copenhagen, Denmark
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Bordeaux, France
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Bron, France
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Créteil, France
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Le Kremlin-Bicêtre, France
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Marseille, France
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Berlin, Germany
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Giessen, Germany
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Hanover, Germany
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Jerusalem, Israel
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Ramat Gan, Israel
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Milan, Italy
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Naples, Italy
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Palermo, Italy
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Roma, Italy
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Groningen, Netherlands
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Lisbon, Portugal
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Porto, Portugal
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Barcelona, Spain
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Madrid, Spain
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Palma, Spain
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New Taipei City, Taiwan
Clinical Trial Site
RECRUITING

Taipei, Taiwan

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