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

NCT ID NCT06260709

First seen Jun 26, 2026 · Last updated Jun 27, 2026 · Updated 1 time

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. All 80 participants have already completed an earlier study and 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.

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 it works, this could point toward a treatment that reduces heart failure symptoms and improves daily function for people with this rare heart disease.
What could go wrong
This is a small, early-phase extension study with no placebo group, so results may not prove effectiveness. Long-term safety is still unknown.

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.

As listed by the trial registrant

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

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

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