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Promising liver drug trial halted early: what it means for patients

NCT ID NCT04764448

Terminated Disease control Sponsor: Dicerna Pharmaceuticals, Inc., a Novo Nordisk company Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study tested a new drug called belcesiran in people with a genetic condition called alpha-1 antitrypsin deficiency that causes liver damage. The goal was to see if the drug is safe and can lower harmful protein levels in the liver. The trial was stopped early, so results are limited.

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.

alpha 1-antitrypsin deficiency

As listed by the trial registrant

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

ALPHA 1-ANTITRYPSIN DEFICIENCY

Contacts and locations

Locations

  • Addenbrooke's Hospital, Cambridge University

    Cambridge, United Kingdom

  • Auckland Clinical Studies

    Grafton, Auckland, 1010, New Zealand

  • Beaumont Hospital

    Dublin, Ireland

  • CHU Bordeaux - Hopital Haut-Leveque - Centre François Magendie

    Pessac, France

  • CTC Clinical Trial Consultants AB Uppsala

    Uppsala, Sweden

  • Centre Hospitalier de l'Universite de Montreal (CHUM)

    Montreal, Quebec, Canada

  • Centro Hospitalar Universitario de Sao Joao

    Porto, Portugal

  • Centro Hospitalar de Trás-os-Montes e Alto Douro, EPE

    Vila Real, Portugal

  • Hospital Universitario La Paz

    Madrid, Spain

  • Hospital Universitario Marques de Valdecilla Santander

    Santander, Cantabria, Spain

  • Hospital da Senhora da Oliveira - Guimaraes

    Creixomil, Portugal

  • Leeds Teaching Hospitals NHS Trust

    Leeds, United Kingdom

  • Leiden University Medical Center

    Leiden, Netherlands

  • Medical University of South Carolina

    Charleston, South Carolina, 29425, United States

  • Medizinische Universitaet Innsbruck

    Innsbruck, Austria

  • Royal Free London NHS Foundation Trust, Royal Free Hospital

    London, United Kingdom

  • St Vincent's Hospital Melbourne

    Melbourne, Australia

  • Universitaetsklinikum Aachen, AoeR

    Aachen, Germany

  • Universitaetsklinikum Schleswig-Holstein Campus Kiel

    Kiel, Germany

  • Universitaire Ziekenhuizen Leuven

    Leuven, Belgium

  • University of California - San Diego

    La Jolla, California, 92093, United States

  • University of Florida

    Gainesville, Florida, 32611, United States

  • Waikato Hospital

    Hamilton, New Zealand

More trials for these conditions

Other studies related to the condition(s) this trial covers.