A Study of Belcesiran in Patients With AATLD

Stopped early · Phase 2 · Has a placebo group

Conditions studied: Alpha 1-Antitrypsin Deficiency

In brief

This is a multiple dose, randomized, placebo-controlled, double-blind study of belcesiran to evaluate the safety, tolerability, PK, and PD in adult patients with PiZZ AATD-associated liver disease (AATLD). The study will be conducted in 3 separate cohorts. A total of up to 16 participants may be enrolled in Cohort 1 and 2. A total number of 30 subjects will be enrolled in cohort 3. The 3 cohorts are differentiated by the duration of the treatment period, the number of doses administered, and the timing of the second liver biopsy.

Key facts

Study ID

NCT04764448

Run by

Dicerna Pharmaceuticals, Inc., a Novo Nordisk company

People needed

16

Starts

2021-02-12

Expected to finish

2024-05-29

Last updated by the study team

2026-04-07

Who can join

Age: 18 and older, up to 75. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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.

You may not qualify if…

• 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.

Where it is running

• University of California - San Diego — La Jolla, California, United States
• University of Florida — Gainesville, Florida, United States
• Medical University of South Carolina — Charleston, South Carolina, United States
• St Vincent's Hospital Melbourne — Melbourne, Australia
• Medizinische Universitaet Innsbruck — Innsbruck, Austria
• Universitaire Ziekenhuizen Leuven — Leuven, Belgium
• Centre Hospitalier de l'Universite de Montreal (CHUM) — Montreal, Quebec, Canada
• CHU Bordeaux - Hopital Haut-Leveque - Centre François Magendie — Pessac, France
• Universitaetsklinikum Aachen, AoeR — Aachen, Germany
• Universitaetsklinikum Schleswig-Holstein Campus Kiel — Kiel, Germany
• Beaumont Hospital — Dublin, Ireland
• Leiden University Medical Center — Leiden, Netherlands
• Auckland Clinical Studies — Grafton, Auckland, New Zealand
• Waikato Hospital — Hamilton, New Zealand
• Hospital da Senhora da Oliveira - Guimaraes — Creixomil, Portugal
• Centro Hospitalar Universitario de Sao Joao — Porto, Portugal
• Centro Hospitalar de Trás-os-Montes e Alto Douro, EPE — Vila Real, Portugal
• Hospital Universitario Marques de Valdecilla Santander — Santander, Cantabria, Spain
• Hospital Universitario La Paz — Madrid, Spain
• CTC Clinical Trial Consultants AB Uppsala — Uppsala, Sweden
• Addenbrooke's Hospital, Cambridge University — Cambridge, United Kingdom
• Leeds Teaching Hospitals NHS Trust — Leeds, United Kingdom
• Royal Free London NHS Foundation Trust, Royal Free Hospital — London, United Kingdom

Full record on ClinicalTrials.gov

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