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 Nov 01, 2025 · Last updated May 15, 2026 · Updated 28 times

Summary

This study is following 1,500 people with ATTR amyloidosis, a rare disease that damages the heart and nerves, to better understand how the condition progresses and how it is managed in everyday medical practice. Researchers will also track the safety and effectiveness of two approved drugs, patisiran and vutrisiran, and monitor people who carry the genetic mutation but do not yet have symptoms. The goal is to gather real-world data to improve future care.

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This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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Contacts and locations

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Study contacts

Contact

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Locations

Clinical Trial Site
RECRUITING

La Jolla, California, 92037, United States
Clinical Trial Site
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Los Angeles, California, 90095, United States
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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
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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
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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
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Taipei, Taiwan

Conditions

Explore the condition pages connected to this study.

ATTR AMYLOIDOSIS TRANSTHYRETIN-MEDIATED AMYLOIDOSIS