Status:
TERMINATED
A Study of Belcesiran in Patients With AATLD
Lead Sponsor:
Dicerna Pharmaceuticals, Inc., a Novo Nordisk company
Conditions:
Alpha 1-Antitrypsin Deficiency
Eligibility:
All Genders
18-75 years
Phase:
PHASE2
Brief Summary
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 (AA...
Detailed Description
AATD-associated liver disease is a progressive condition resulting in liver fibrosis, cirrhosis, and in some cases hepatocellular carcinoma. The lack of functional alpha-1 antitrypsin (AAT) in individ...
Eligibility Criteria
Inclusion
- 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
- 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.
Key Trial Info
Start Date :
February 12 2021
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
May 29 2024
Estimated Enrollment :
16 Patients enrolled
Trial Details
Trial ID
NCT04764448
Start Date
February 12 2021
End Date
May 29 2024
Last Update
December 31 2024
Active Locations (23)
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1
University of California - San Diego
La Jolla, California, United States, 92093
2
University of Florida
Gainesville, Florida, United States, 32611
3
Medical University of South Carolina
Charleston, South Carolina, United States, 29425
4
St Vincent's Hospital Melbourne
Melbourne, Australia