Hope for rare heart disease: new drug shows promise in major trial

NCT ID NCT03997383

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

First seen Nov 01, 2025 · Last updated Apr 23, 2026 · Updated 24 times

Summary

This study tested a drug called patisiran in 360 people with a rare heart condition caused by abnormal protein buildup (ATTR amyloidosis with cardiomyopathy). The goal was to see if the drug could improve how far patients could walk in 6 minutes and their quality of life after 12 months. Participants received either patisiran or a placebo, and the study is now complete.

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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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Locations

Clinical Trial Site

Los Angeles, California, 90048, United States
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Chicago, Illinois, 60637, United States
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Skokie, Illinois, 60076, 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
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Boston, Massachusetts, 02118, United States
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Burlington, Massachusetts, 10805, United States
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Rochester, Minnesota, 55905, United States
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St Louis, Missouri, 63110, United States
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New York, New York, 10029, United States
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New York, New York, 10032, United States
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Cleveland, Ohio, 44195, United States
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Philadelphia, Pennsylvania, 19104, United States
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Philadelphia, Pennsylvania, 19140, United States
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Pittsburgh, Pennsylvania, 15212, United States
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Nashville, Tennessee, 37232, United States
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Dallas, Texas, 75246, United States
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Norfolk, Virginia, 23507, United States
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Buenos Aires, C1039AAO, Argentina
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Buenos Aires, C1428AQK, Argentina
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Córdoba, X5000KEH, Argentina
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Rosario, S2000DSR, Argentina
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Rosario, S2000DTC, Argentina
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Rosario, S2000PBJ, Argentina
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San Juan Bautista, B1888AAE, Argentina
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Santa Fe, S3000FWO, Argentina
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Box Hill, 3128, Australia
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Westmead, 2145, Australia
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Woolloongabba, 4021, Australia
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Aalst, 9300, Belgium
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Hasselt, 3500, Belgium
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Liège, 4000, Belgium
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Roeselare, 8800, Belgium
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Porto Alegre, 90035-030, Brazil
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Ribeirão Preto, 14026-900, Brazil
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Ribeirão Preto, 14048-900, Brazil
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Rio de Janeiro, 22280-000, Brazil
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São Paulo, 04012-909, Brazil
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São Paulo, 05403-000, Brazil
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Sofia, 1680, Bulgaria
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Sofia, 1784, Bulgaria
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Stara Zagora, 6000, Bulgaria
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Providencia/ Santiago, 7500587, Chile
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Brno, 656 91, Czechia
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Prague, 120 00, Czechia
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Prague, 128 08, Czechia
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Prague, 140 21, Czechia
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Aarhus N, 8200, Denmark
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Copenhagen, 2100, Denmark
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Odense C, 5000, Denmark
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Clichy, 92110, France
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Créteil, 94010, France
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Rennes, 35033, France
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Toulouse, 31059, France
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Lai Chi Kok, 999077, Hong Kong
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Bologna, 40138, Italy
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Florence, 50134, Italy
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Messina, 98125, Italy
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Pavia, 27100, Italy
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Bunkyō City, 113-8655, Japan
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Fukuoka, 812-8582, Japan
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Kumamoto, 860-8556, Japan
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Kurume, 830-0011, Japan
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Matsumoto, 390-8621, Japan
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Nagoya, 466-8560, Japan
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Suita, 565-8565, Japan
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Mexico City, 14080, Mexico
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Groningen, 9713 GZ, Netherlands
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Maastricht, 6229 HX, Netherlands
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Christchurch, 8011, New Zealand
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Hamilton, 3204, New Zealand
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Gdansk, 80-382, Poland
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Katowice, 40-635, Poland
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Lódz, 90-127, Poland
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Warsaw, 01-192, Poland
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Warsaw, 04-628, Poland
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Porto, 4099-001, Portugal
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Senhora da Hora, 4464-513, Portugal
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Viseu, 3504-509, Portugal
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Seoul, 3080, South Korea
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Stockholm, 17164, Sweden
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Taipei, 11217, Taiwan
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Bellshill, ML4 3NJ, United Kingdom
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Birmingham, B15 2SQ, United Kingdom
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Cardiff, CF15 9SS, United Kingdom
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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

Conditions

Explore the condition pages connected to this study.

TRANSTHYRETIN AMYLOIDOSIS (ATTR) WITH CARDIOMYOPATHY