Stopped: Development project was discontinued
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.
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Number of Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
Timeframe: Up to 2.6 years
Number of Participants With TEAEs and SAEs
Timeframe: Up to 2.6 years
Change From Baseline in Pulmonary Function Tests (PFTs): Forced Vital Capacity (FVC)
Timeframe: Baseline (Day 1), week 96
Change From Baseline in PFT: Forced Expiratory Volume in One Second (FEV1)
Timeframe: Baseline (Day 1), week 96
Change From Baseline in PFT: FEV1/FVC Ratio
Timeframe: Baseline (Day 1), week 96
Change From Baseline in PFT: Diffusing Capacity of the Lungs for Carbon Monoxide (DLCO)
Timeframe: Baseline (Day 1), week 96
Change From Baseline in 12 -Lead Electrochardiograms (ECGs): Mean Heart Rate
Timeframe: Baseline (Day 1), week 96
Change From Baseline in 12 -Lead ECGs: Mean Ventricular Rate
Timeframe: Baseline (Day 1), week 96
Change From Baseline in 12 -Lead ECGs: PR Interval, QRS Interval, QT Interval, QTcF Interval and RR Interval
Timeframe: Baseline (Day 1), week 96
Number of Participants With Physical Examination Findings
Timeframe: At week 96
Change From Baseline in Vital Signs: Diastolic Blood Pressure and Systolic Blood Pressure
Timeframe: Baseline (Day 1), week 96
Change From Baseline in Vital Signs: Heart Rate
Timeframe: Baseline (Day 1), week 96
Vital Signs: Height at Baseline
Timeframe: Baseline (Day 1)
Change From Baseline in Vital Signs: Respiratory Rate
Timeframe: Baseline (Day 1), week 96
Change From Baseline in Vital Signs: Temperature
Timeframe: Baseline (Day 1), week 96
Change From Baseline in Vital Signs: Weight
Timeframe: Baseline (Day 1), week 96
Change in Clinical Laboratory Tests: Alanine Aminotransferase, Alkaline Phosphatase, Aspartate Aminotransferase, Creatine Kinase, Glutamate Dehydrogenase, Lactate Dehydrogenase, Biomarker Creatine Kinase (M30) and Biomarker Creatine Kinase (M65)
Timeframe: Baseline (Day 1), week 96
Change From Baseline in Clinical Laboratory Tests: Albumin, Apolipoprotein A1, Protein, Biomarker Haptoglobin
Timeframe: Baseline (Day 1), week 96
Change From Baseline in Clinical Laboratory Tests: Bilirubin, Creatinine and Direct Bilirubin
Timeframe: Baseline (Day 1), week 96
Change From Baseline in Clinical Laboratory Tests: Chloride, Cholesterol, Glucose, Potassium, Sodium, Triglycerides and Urea Nitrogen
Timeframe: Baseline (Day 1), week 96
Change From Baseline in Clinical Laboratory Tests: Gamma Glutamyl Transferase
Timeframe: Baseline (Day 1), week 96
Change From Baseline in Clinical Laboratory Tests: Basophils, Eosinophils, Leucocytes, Lymphocytes, Monocytes, Neutrophils and Platelets
Timeframe: Baseline (Day 1), week 96
Change From Baseline in Clinical Laboratory Tests: Basophils/Leucocytes
Timeframe: Baseline (Day 1), week 96
Change From Baseline in Clinical Laboratory Tests: Eosinophils/Leucocytes
Timeframe: Baseline (Day 1), week 96
Change From Baseline in Clinical Laboratory Tests: Haematocrit
Timeframe: Baseline (Day 1), week 96
Change From Baseline in Clinical Laboratory Tests: Lymphocytes/Leucocytes
Timeframe: Baseline (Day 1), week 96
Change From Baseline in Clinical Laboratory Tests: Monocytes/Leucocytes
Timeframe: Baseline (Day 1), week 96
Change From Baseline in Clinical Laboratory Tests: Neutrophils/Leucocytes
Timeframe: Baseline (Day 1), week 96
Change From Baseline in Clinical Laboratory Tests: Reticulocytes/Erythrocytes
Timeframe: Baseline (Day 1), week 96
Change From Baseline in Clinical Laboratory Tests: Erythrocyte Mean Corpuscular Haemoglobin Concentration and Haemoglobin
Timeframe: Baseline (Day 1), week 96
Change From Baseline in Clinical Laboratory Tests: Erythrocyte Mean Corpuscular Haemoglobin
Timeframe: Baseline (Day 1), week 96
Change From Baseline in Clinical Laboratory Tests: Erythrocyte Mean Corpuscular Volume
Timeframe: Baseline (Day 1), week 96
Change From Baseline in Clinical Laboratory Tests: Erythrocytes
Timeframe: Baseline (Day 1), week 96
Change From Baseline in Clinical Laboratory Tests: Specific Gravity
Timeframe: Baseline (Day 1), week 96
Change From Baseline in Clinical Laboratory Tests: Urine Erythrocytes and Urine Leucocytes
Timeframe: Baseline (Day 1), week 96
Change From Baseline in Clinical Laboratory Tests: Potential of Hydrogen (pH)
Timeframe: Baseline (Day 1), week 96
Change From Baseline in Clinical Laboratory Tests: Activated Partial Thromboplastin Time and Prothrombin Time
Timeframe: Baseline (Day 1), week 96
Change From Baseline in Clinical Laboratory Tests: Prothrombin International Normalized Ratio
Timeframe: Baseline (Day 1), week 96
Change in Clinical Laboratory Tests: Alpha Fetoprotein, Biomarker Hyaluronic Acid, Biomarker Matrix Metalloproteinase 9, Biomarker Procollagen 3 N-Terminal Propeptide, Biomarker Tissue Inhibitor of Metalloproteinase 1, Complement C3a and Complement C5a
Timeframe: Baseline (Day 1), week 96
Change From Baseline in Clinical Laboratory Tests: C-Reactive Protein
Timeframe: Baseline (Day 1), week 96
Change From Baseline in Clinical Laboratory Tests: Complement Bb
Timeframe: Baseline (Day 1), week 96
Change From Baseline in Clinical Laboratory Tests: Complement Total (CH50)
Timeframe: Baseline (Day 1), week 96
Cohort 1: Change From Baseline in Serum Alpha-1 Antitrypsin (AAT) Protein Concentrations
Timeframe: Baseline (Day 1), week 24
Cohort 2: Change From Baseline in Serum AAT Protein Concentrations
Timeframe: Baseline (Day 1), week 48