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
0
Start Date
December 2021
Completion Date
May 23, 2022
Primary Completion Date
May 23, 2022
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
Anne-Sophie Sejling, 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
Anne-Sophie Sejling, MD, Study Director, Dicerna Pharmaceuticals
Verification Date
June 2022