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Hope for rare heart disease: experimental drug tested for long-term safety

NCT ID NCT06260709

First seen Nov 01, 2025 · Last updated Jun 23, 2026 · Updated 29 times

Summary

This study tests the long-term safety of an experimental drug called NNC6019-0001 in people with transthyretin amyloid cardiomyopathy, a rare heart disease that can lead to heart failure. About 80 participants who completed an earlier study will receive the drug every 4 weeks for up to 3 years. Researchers will monitor side effects and measure changes in walking ability, heart function, and quality of life.

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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

Locations

  • Ap-Hp-Hopital Henri Mondor

    Créteil, 94000, France

  • Cedars-Sinai Medical Center_Los Angeles

    Los Angeles, California, 90048, United States

  • Centre Hospitalier Universitaire de Toulouse-Hopital Rangueil-1

    Toulouse, 31059, France

  • Centro per lo Studio e la Cura delle Amiloidosi Sistemiche Fondazione IRCCS Policlinico San Matteo

    Pavia, PV, 27100, Italy

  • Ctr for Cardiovascular Innovation

    Vancouver, British Columbia, V5Z 1M9, Canada

  • Fondazione CNR-Regione Toscana Gabriele Monasterio

    Pisa, Pi, 56124, Italy

  • Fondazione Toscana Gabriele Monasterio - Dipartimento Cardiotoracico - UOC Cardiologia e Medicina Cardiovascolare

    Pisa, Pi, 56124, Italy

  • Hiroshima University hospital, Cardiovascular Medicine

    Hiroshima, 734-8551, Japan

  • Hospital Universitario Puerta de Hierro Majadahonda

    Majadahonda, Madrid, 28222, Spain

  • Hospital da Senhora da Oliveira - Guimarães

    Guimarães, 4835-044, Portugal

  • Hospital of the University of Occupational and Environmental Health, Cardiology, Nephrology

    Fukuoka, 807-8556, Japan

  • II. interni klinika VFN - Kardiologie a angiologie

    Prague, 12808, Czechia

  • Kumamoto University Hospital, Cardiovascular Medicine

    Kumamoto-shi, Kumamoto, 860-8556, Japan

  • LMU Klinikum München Klinik und Poliklinik 1

    München, 81377, Germany

  • Mayo Clinic Arizona

    Phoenix, Arizona, 85054, United States

  • Mayo Clinic Jacksonville

    Jacksonville, Florida, 32224, United States

  • Mayo Clinic Rochester

    Rochester, Minnesota, 55905, United States

  • NW Univ-Bluhm Cardiovasc Inst

    Chicago, Illinois, 60611, United States

  • NW Univ-Bluhm Cardiovasc Inst

    Evanston, Illinois, 60208, United States

  • Nagasaki University Hospital, Cardiovascular Medicine

    Nagasaki, 852-8501, Japan

  • Okayama University Hospital_Cardiovascular Medicine

    Okayama-shi, Okayama, 700-8558, Japan

  • Shinshu University Hospital, Department of Neurology

    Nagano, 390-8621, Japan

  • Stanford Hlth Cre-Boswell Clin

    Stanford, California, 94305, United States

  • UMC Groningen

    Groningen, 9713 GZ, Netherlands

  • Unidade Local De Saude De Tras-Os-Montes E Alto Douro E.P.E.

    Vila Real, 5000-508, Portugal

  • Unidade Local de Saude do Alto Ave, E.P.E.

    Guimarães, 4835-044, Portugal

  • Uniklinik Münster, Klinik für Kardiologie I

    Münster, 48149, Germany

  • UniklinikHeidelberg - Innere Med. III - Kardiologie, Angiologie, Pneumologie

    Heidelberg, 69120, Germany

  • Univ of MD Schl of Med

    Baltimore, Maryland, 21201, United States

  • Universitair Medisch Centrum Utrecht

    Utrecht, 3584 CX, Netherlands

  • Universitatsklinikum Wurzburg AöR

    Würzburg, 97078, Germany

  • Universitatsklinikum Würzburg - Zentrum für Herzinsuffizienz

    Würzburg, 97078, Germany

  • University of Calgary_Cardiology

    Calgary, Alberta, T2N 4Z6, Canada

  • Universitätsklinik Heidelberg Innere Medizin III Kardiologie, Angiologie, Pneumologie

    Heidelberg, 69120, Germany

  • Universitätsklinikum Münster - Klinik für Kardiologie I

    Münster, 48149, Germany

What this could mean

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

Active substance

NNC6019-0001

What this could lead to

If successful, this could show that NNC6019-0001 is safe and helps manage symptoms of this heart disease over the long term.

What could go wrong

This is a small, early-phase extension study with no placebo group, so results may not prove effectiveness. The drug may still cause side effects or not improve outcomes.

Conditions

The condition(s) this trial relates to.

wild type ATTR amyloidosis

As listed by the trial registrant

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