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

NCT ID NCT04764448

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

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early This study
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

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.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

16 people

The number who actually took part.

Started

Feb 2021

Finished

May 2024

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 to 75 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * 18 to 75 years, inclusive, at the time of consent. * Documented diagnosis of PiZZ-type alpha-1 antitrypsin deficiency, confirmed by genotyping. Historical genotyping data may be used, if available. * AATD-associated liver disease documented by liver biopsy at Screening. * Consent to undergo paired liver biopsies. * Lung, renal and liver function within acceptable limits * Capable of giving signed informed consent, which includes compliance with the requirements and restrictions listed in the ICF and in this protocol. Exclusion Criteria: * History of chronic liver disease other than non-alcoholic fatty liver disease from any cause other than PiZZ-type alpha-1 antitrypsin deficiency. * Child-Pugh Score B or C. * History of one single severe exacerbation of underlying lung disease in the year prior to randomization. * History of clinically significant respiratory infections (including pneumonia and lower respiratory tract infections), as determined by the Investigator, in the 3 months prior to screening * Use of an RNAi drug at any time.

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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.