An Extension Study of Belcesiran in Patients With Alpha-1 Antitrypsin Deficiency Associated Liver Disease (AATLD)

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Brief Title

An Extension Study of Belcesiran in Patients With Alpha-1 Antitrypsin Deficiency Associated Liver Disease (AATLD)

Official Title

A Phase 2 Open-Label Extension Study to Evaluate the Safety and Pharmacodynamics of Belcesiran in Patients With PiZZ Alpha-1 Antitrypsin Deficiency Associated Liver Disease

Brief Summary

      This is a Phase 2, multicenter, open-label extension of Study DCR-A1AT-201, designed to
      evaluate the long-term safety and further characterize the pharmacodynamics (PD) of
      belcesiran in adult patients with PiZZ AATLD.
    


Study Phase

Phase 2

Study Type

Interventional


Primary Outcome

The incidence of treatment-emergent adverse events

Secondary Outcome

 Changes in serum AAT protein concentrations over time

Condition

Alpha 1-Antitrypsin Deficiency

Intervention

Belcesiran

Study Arms / Comparison Groups

 belcesiran
Description:  Participants who completed the DCR-A1AT-201 treatment period will receive open-label belcesiran administered subcutaneously

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Drug

Estimated Enrollment

54

Start Date

December 2021

Completion Date

December 1, 2025

Primary Completion Date

December 1, 2025

Eligibility Criteria

        Inclusion Criteria:

          1. Must be 18 to 75 years of age inclusive, at the time of signing the Informed Consent
             Form (ICF).

          2. Documented diagnosis of PiZZ-type Alpha-1 Antitrypsin deficiency (AATD), confirmed by
             genotyping.

          3. Lung, renal and liver function within acceptable limits.

          4. Capable of giving signed informed consent, which includes compliance with the
             requirements and restrictions listed in the ICF and in this protocol.

        Exclusion Criteria:

          1. Any condition that, in the opinion of the Investigator, would make the participant
             unsuitable for enrollment or could interfere with participation in or completion of
             the study

          2. Routine use of acetaminophen/paracetamol

          3. Use of systemically acting steroids in the month prior to Screening and throughout the
             study period.

          4. Positive SARS-CoV-2 virus test at Screening

          5. Any other safety laboratory test result considered clinically significant and
             unacceptable by the Investigator

          6. Inability or unwillingness to comply with the specified study procedures, including
             lifestyle considerations
      

Gender

All

Ages

18 Years - 75 Years

Accepts Healthy Volunteers

No

Contacts

Hardean Achneck, MD, , 

Location Countries

New Zealand

Location Countries

New Zealand

Administrative Informations


NCT ID

NCT05146882

Organization ID

DCR-A1AT-202

Secondary IDs

STARLIGHT

Responsible Party

Sponsor

Study Sponsor

Dicerna Pharmaceuticals, Inc.


Study Sponsor

Hardean Achneck, MD, Study Director, Dicerna Pharmaceuticals


Verification Date

December 2021