Experimental drug for rare liver disease shows promise but study halted early
NCT ID NCT04764448
First seen Apr 10, 2026 · Last updated Apr 29, 2026 · Updated 5 times
Summary
This study tested a new drug, belcesiran, in people with a rare genetic condition called Alpha-1 antitrypsin deficiency that causes liver damage. The goal was to see if the drug is safe and can reduce harmful protein buildup in the liver. The trial was stopped early, so results are limited, but the drug aims to control the disease rather than cure it.
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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.
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
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Addenbrooke's Hospital, Cambridge University
Cambridge, United Kingdom
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Auckland Clinical Studies
Grafton, Auckland, 1010, New Zealand
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Beaumont Hospital
Dublin, Ireland
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CHU Bordeaux - Hopital Haut-Leveque - Centre François Magendie
Pessac, France
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CTC Clinical Trial Consultants AB Uppsala
Uppsala, Sweden
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Centre Hospitalier de l'Universite de Montreal (CHUM)
Montreal, Quebec, Canada
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Centro Hospitalar Universitario de Sao Joao
Porto, Portugal
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Centro Hospitalar de Trás-os-Montes e Alto Douro, EPE
Vila Real, Portugal
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Hospital Universitario La Paz
Madrid, Spain
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Hospital Universitario Marques de Valdecilla Santander
Santander, Cantabria, Spain
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Hospital da Senhora da Oliveira - Guimaraes
Creixomil, Portugal
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Leeds Teaching Hospitals NHS Trust
Leeds, United Kingdom
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Leiden University Medical Center
Leiden, Netherlands
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Medical University of South Carolina
Charleston, South Carolina, 29425, United States
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Medizinische Universitaet Innsbruck
Innsbruck, Austria
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Royal Free London NHS Foundation Trust, Royal Free Hospital
London, United Kingdom
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St Vincent's Hospital Melbourne
Melbourne, Australia
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Universitaetsklinikum Aachen, AoeR
Aachen, Germany
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Universitaetsklinikum Schleswig-Holstein Campus Kiel
Kiel, Germany
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Universitaire Ziekenhuizen Leuven
Leuven, Belgium
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University of California - San Diego
La Jolla, California, 92093, United States
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University of Florida
Gainesville, Florida, 32611, United States
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Waikato Hospital
Hamilton, New Zealand
Conditions
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