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
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Ap-Hp-Hopital Henri Mondor
Créteil, 94000, France
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Cedars-Sinai Medical Center_Los Angeles
Los Angeles, California, 90048, United States
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Centre Hospitalier Universitaire de Toulouse-Hopital Rangueil-1
Toulouse, 31059, France
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Centro per lo Studio e la Cura delle Amiloidosi Sistemiche Fondazione IRCCS Policlinico San Matteo
Pavia, PV, 27100, Italy
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Ctr for Cardiovascular Innovation
Vancouver, British Columbia, V5Z 1M9, Canada
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Fondazione CNR-Regione Toscana Gabriele Monasterio
Pisa, Pi, 56124, Italy
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Fondazione Toscana Gabriele Monasterio - Dipartimento Cardiotoracico - UOC Cardiologia e Medicina Cardiovascolare
Pisa, Pi, 56124, Italy
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Hiroshima University hospital, Cardiovascular Medicine
Hiroshima, 734-8551, Japan
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Hospital Universitario Puerta de Hierro Majadahonda
Majadahonda, Madrid, 28222, Spain
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Hospital da Senhora da Oliveira - Guimarães
Guimarães, 4835-044, Portugal
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Hospital of the University of Occupational and Environmental Health, Cardiology, Nephrology
Fukuoka, 807-8556, Japan
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II. interni klinika VFN - Kardiologie a angiologie
Prague, 12808, Czechia
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Kumamoto University Hospital, Cardiovascular Medicine
Kumamoto-shi, Kumamoto, 860-8556, Japan
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LMU Klinikum München Klinik und Poliklinik 1
München, 81377, Germany
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Mayo Clinic Arizona
Phoenix, Arizona, 85054, United States
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Mayo Clinic Jacksonville
Jacksonville, Florida, 32224, United States
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Mayo Clinic Rochester
Rochester, Minnesota, 55905, United States
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NW Univ-Bluhm Cardiovasc Inst
Chicago, Illinois, 60611, United States
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NW Univ-Bluhm Cardiovasc Inst
Evanston, Illinois, 60208, United States
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Nagasaki University Hospital, Cardiovascular Medicine
Nagasaki, 852-8501, Japan
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Okayama University Hospital_Cardiovascular Medicine
Okayama-shi, Okayama, 700-8558, Japan
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Shinshu University Hospital, Department of Neurology
Nagano, 390-8621, Japan
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Stanford Hlth Cre-Boswell Clin
Stanford, California, 94305, United States
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UMC Groningen
Groningen, 9713 GZ, Netherlands
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Unidade Local De Saude De Tras-Os-Montes E Alto Douro E.P.E.
Vila Real, 5000-508, Portugal
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Unidade Local de Saude do Alto Ave, E.P.E.
Guimarães, 4835-044, Portugal
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Uniklinik Münster, Klinik für Kardiologie I
Münster, 48149, Germany
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UniklinikHeidelberg - Innere Med. III - Kardiologie, Angiologie, Pneumologie
Heidelberg, 69120, Germany
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Univ of MD Schl of Med
Baltimore, Maryland, 21201, United States
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Universitair Medisch Centrum Utrecht
Utrecht, 3584 CX, Netherlands
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Universitatsklinikum Wurzburg AöR
Würzburg, 97078, Germany
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Universitatsklinikum Würzburg - Zentrum für Herzinsuffizienz
Würzburg, 97078, Germany
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University of Calgary_Cardiology
Calgary, Alberta, T2N 4Z6, Canada
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Universitätsklinik Heidelberg Innere Medizin III Kardiologie, Angiologie, Pneumologie
Heidelberg, 69120, Germany
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Universitätsklinikum Münster - Klinik für Kardiologie I
Münster, 48149, Germany
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- New drug duo aims to stop rare heart disease in its tracks
- Can a smartphone app keep heart patients out of the hospital? new study aims to find out.
- Experimental drug shows promise for stiff heart disease
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