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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/study/NCT04764448
Registration number
NCT04764448
Ethics application status
Date submitted
27/01/2021
Date registered
21/02/2021
Date last updated
25/03/2025
Titles & IDs
Public title
A Study of Belcesiran in Patients With AATLD
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Scientific title
A Phase 2, Randomized, Double-blind, Placebo-Controlled Study Investigating Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Two Dose Levels of Belcesiran in Patients With Alpha-1 Antitrypsin Deficiency-Associated Liver Disease
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Secondary ID [1]
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DCR-A1AT-201
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Universal Trial Number (UTN)
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Trial acronym
ESTRELLA
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Alpha 1-Antitrypsin Deficiency
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Condition category
Condition code
Human Genetics and Inherited Disorders
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Other human genetics and inherited disorders
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Respiratory
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Other respiratory disorders / diseases
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Treatment: Drugs - Belcesiran
Other interventions - Placebo
Treatment: Drugs - Belcesiran
Other interventions - Placebo
Treatment: Drugs - Belcesiran
Other interventions - Placebo
Experimental: Belcesiran Cohort 1 -
Placebo comparator: Placebo Cohort 1 -
Experimental: Belcesiran Cohort 2 -
Placebo comparator: Placebo Cohort 2 -
Experimental: Belcesiran Cohort 3 -
Placebo comparator: Placebo Cohort 3 -
Treatment: Drugs: Belcesiran
Administered multiple fixed doses of belcesiran by subcutaneous (sc) injection for 24 weeks. Extension offered to participants.
Other interventions: Placebo
Comparator: Placebo Cohort 1 Administered sterile normal saline (0.9% NaCl) matching volume of belcesiran by subcutaneous (sc) injection for 24 weeks. Extension offered to participants.
Treatment: Drugs: Belcesiran
Administered multiple fixed doses of belcesiran by subcutaneous (sc) injection for 48 weeks. Extension offered to participants.
Other interventions: Placebo
Administered sterile normal saline (0.9% NaCl) matching volumes of belcesiran by subcutaneous (sc) injection for 48 weeks. Extension offered to participants.
Treatment: Drugs: Belcesiran
Administered multiple fixed doses of belcesiran by subcutaneous (sc) injection for 96 weeks.
Other interventions: Placebo
Administered sterile normal saline (0.9% NaCl) matching volumes of belcesiran by subcutaneous (sc) injection for 96 weeks.
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Intervention code [1]
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Treatment: Drugs
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Intervention code [2]
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Other interventions
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Number of Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
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Assessment method [1]
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Number of TEAEs and SAEs is presented. An adverse event (AE) was any untoward medical occurrence in a participant or clinical study participant, temporally associated with the use of study intervention, whether or not considered related to the study intervention. AEs were defined as TEAEs if they had a start date on or after the administration of study drug during the treatment period, or if they occurred prior to the administration of study drug and worsened in severity/grade or relationship to the study intervention after the administration of study intervention during the treatment period. A SAE was defined as any untoward medical occurrence that, at any dose: a) resulted in death, b) is life-threatening, c) required inpatient hospitalization or prolongation of existing hospitalization, d) resulted in persistent disability/incapacity, e) was a congenital anomaly/birth defect.
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Timepoint [1]
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Up to 2.6 years
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Primary outcome [2]
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Number of Participants With TEAEs and SAEs
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Assessment method [2]
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Number of participants with TEAEs and SAEs is presented. An AE was any untoward medical occurrence in a participant or clinical study participant, temporally associated with the use of study intervention, whether or not considered related to the study intervention. AEs were defined as TEAEs if they had a start date on or after the administration of study drug during the treatment period, or if they occurred prior to the administration of study drug and worsened in severity/grade or relationship to the study intervention after the administration of study intervention during the treatment period. A SAE was defined as any untoward medical occurrence that, at any dose: a) resulted in death, b) is life-threatening, c) required inpatient hospitalization or prolongation of existing hospitalization, d) resulted in persistent disability/incapacity, e) was a congenital anomaly/birth defect.
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Timepoint [2]
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Up to 2.6 years
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Primary outcome [3]
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Change From Baseline in Pulmonary Function Tests (PFTs): Forced Vital Capacity (FVC)
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Assessment method [3]
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Change in FVC from baseline (Day 1) to week 96 is presented. FVC is the maximal volume of air exhaled with maximally forced effort from a maximal inspiration, that is, VC performed with a maximally forced expiratory effort.
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Timepoint [3]
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Baseline (Day 1), week 96
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Primary outcome [4]
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Change From Baseline in PFT: Forced Expiratory Volume in One Second (FEV1)
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Assessment method [4]
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Change in FEV1 from baseline (Day 1) to week 96 is presented. FEV1 is the maximal volume of air exhaled in the first second of a forced expiration from a position of full inspiration.
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Timepoint [4]
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Baseline (Day 1), week 96
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Primary outcome [5]
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Change From Baseline in PFT: FEV1/FVC Ratio
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Assessment method [5]
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Change in FEV1/FVC ratio from baseline (Day 1) to week 96 is presented. FEV1 is the maximal volume of air exhaled in the first second of a forced expiration from a position of full inspiration. FVC is the maximal volume of air exhaled with maximally forced effort from a maximal inspiration, that is, VC performed with a maximally forced expiratory effort.
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Timepoint [5]
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Baseline (Day 1), week 96
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Primary outcome [6]
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Change From Baseline in PFT: Diffusing Capacity of the Lungs for Carbon Monoxide (DLCO)
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Assessment method [6]
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Change in DLCO from baseline (Day 1) to week 96 is presented. DLCO is a measure of the quantity of carbon monoxide (CO) transferred per minute from alveolar gas to red blood cells (specifically hemoglobin) in pulmonary capillaries. It is expressed as millimoles per minute per kilopascal (mmol/min/kPa).
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Timepoint [6]
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Baseline (Day 1), week 96
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Primary outcome [7]
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Change From Baseline in 12 -Lead Electrochardiograms (ECGs): Mean Heart Rate
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Assessment method [7]
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Change from baseline (Day 1) to week 96 in mean heart rate is presented.
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Timepoint [7]
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Baseline (Day 1), week 96
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Primary outcome [8]
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Change From Baseline in 12 -Lead ECGs: Mean Ventricular Rate
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Assessment method [8]
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Change from baseline (Day 1) to week 96 in mean ventricular rate is presented.
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Timepoint [8]
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Baseline (Day 1), week 96
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Primary outcome [9]
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Change From Baseline in 12 -Lead ECGs: PR Interval, QRS Interval, QT Interval, QTcF Interval and RR Interval
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Assessment method [9]
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Change from baseline (Day 1) to week 96 in PR interval, QRS interval, QT interval, QTcF interval and RR interval is presented.
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Timepoint [9]
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Baseline (Day 1), week 96
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Primary outcome [10]
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Number of Participants With Physical Examination Findings
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Assessment method [10]
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Number of participants with physical examination findings at week 96 is presented. The data is presented under categories:a) Normal, b) Abnormal, not clinically significant, c) Abnormal, clinically significant.
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Timepoint [10]
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At week 96
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Primary outcome [11]
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Change From Baseline in Vital Signs: Diastolic Blood Pressure and Systolic Blood Pressure
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Assessment method [11]
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Change from baseline (Day 1) to week 96 in diastolic blood pressure and systolic blood pressure is presented.
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Timepoint [11]
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Baseline (Day 1), week 96
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Primary outcome [12]
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Change From Baseline in Vital Signs: Heart Rate
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Assessment method [12]
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Change from baseline (Day 1) to week 96 in heart rate is presented.
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Timepoint [12]
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Baseline (Day 1), week 96
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Primary outcome [13]
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Vital Signs: Height at Baseline
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Assessment method [13]
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Height at baseline is presented.
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Timepoint [13]
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Baseline (Day 1)
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Primary outcome [14]
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Change From Baseline in Vital Signs: Respiratory Rate
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Assessment method [14]
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Change from baseline (Day 1) to week 96 in respiratory rate is presented.
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Timepoint [14]
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Baseline (Day 1), week 96
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Primary outcome [15]
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Change From Baseline in Vital Signs: Temperature
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Assessment method [15]
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Change from baseline (Day 1) to week 96 in temperature is presented.
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Timepoint [15]
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Baseline (Day 1), week 96
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Primary outcome [16]
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Change From Baseline in Vital Signs: Weight
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Assessment method [16]
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Change from baseline (Day 1) to week 96 in weight is presented.
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Timepoint [16]
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Baseline (Day 1), week 96
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Primary outcome [17]
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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)
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Assessment method [17]
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Change from baseline (Day 1) to week 96 in alanine aminotransferase, alkaline phosphatase, aspartate aminotransferase, creatine kinase, glutamate dehydrogenase, lactate dehydrogenase, biomarker creatine kinase (M30) and biomarker creatine kinase (M65) is presented.
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Timepoint [17]
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Baseline (Day 1), week 96
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Primary outcome [18]
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Change From Baseline in Clinical Laboratory Tests: Albumin, Apolipoprotein A1, Protein, Biomarker Haptoglobin
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Assessment method [18]
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Change from baseline (Day 1) to week 96 in albumin, apolipoprotein A1, protein and biomarker haptoglobin is presented.
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Timepoint [18]
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Baseline (Day 1), week 96
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Primary outcome [19]
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Change From Baseline in Clinical Laboratory Tests: Bilirubin, Creatinine and Direct Bilirubin
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Assessment method [19]
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Change from baseline (Day 1) to week 96 in bilirubin, creatinine and direct bilirubin is presented.
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Timepoint [19]
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Baseline (Day 1), week 96
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Primary outcome [20]
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Change From Baseline in Clinical Laboratory Tests: Chloride, Cholesterol, Glucose, Potassium, Sodium, Triglycerides and Urea Nitrogen
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Assessment method [20]
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Change from baseline (Day 1) to week 96 in chloride, cholesterol, glucose, potassium, sodium, triglycerides and urea nitrogen is presented.
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Timepoint [20]
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Baseline (Day 1), week 96
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Primary outcome [21]
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Change From Baseline in Clinical Laboratory Tests: Gamma Glutamyl Transferase
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Assessment method [21]
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Change from baseline (Day 1) to week 96 in gamma glutamyl transferase is presented.
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Timepoint [21]
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Baseline (Day 1), week 96
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Primary outcome [22]
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Change From Baseline in Clinical Laboratory Tests: Basophils, Eosinophils, Leucocytes, Lymphocytes, Monocytes, Neutrophils and Platelets
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Assessment method [22]
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Change from baseline (Day 1) to week 96 in basophils, eosinophils, leucocytes, lymphocytes, monocytes, neutrophils and platelets is presented.
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Timepoint [22]
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Baseline (Day 1), week 96
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Primary outcome [23]
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Change From Baseline in Clinical Laboratory Tests: Basophils/Leucocytes
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Assessment method [23]
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Change from baseline (Day 1) to week 96 in basophils/leucocytes is presented.
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Timepoint [23]
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Baseline (Day 1), week 96
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Primary outcome [24]
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Change From Baseline in Clinical Laboratory Tests: Eosinophils/Leucocytes
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Assessment method [24]
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Change from baseline (Day 1) to week 96 in eosinophils/leucocytes is presented.
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Timepoint [24]
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Baseline (Day 1), week 96
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Primary outcome [25]
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Change From Baseline in Clinical Laboratory Tests: Haematocrit
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Assessment method [25]
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Change from baseline (Day 1) to week 96 in haematocrit is presented.
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Timepoint [25]
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Baseline (Day 1), week 96
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Primary outcome [26]
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Change From Baseline in Clinical Laboratory Tests: Lymphocytes/Leucocytes
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Assessment method [26]
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Change from baseline (Day 1) to week 96 in lymphocytes/leucocytes is presented.
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Timepoint [26]
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Baseline (Day 1), week 96
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Primary outcome [27]
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Change From Baseline in Clinical Laboratory Tests: Monocytes/Leucocytes
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Assessment method [27]
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Change from baseline (Day 1) to week 96 in monocytes/leucocytes is presented.
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Timepoint [27]
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Baseline (Day 1), week 96
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Primary outcome [28]
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Change From Baseline in Clinical Laboratory Tests: Neutrophils/Leucocytes
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Assessment method [28]
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Change from baseline (Day 1) to week 96 in neutrophils/leucocytes is presented.
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Timepoint [28]
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Baseline (Day 1), week 96
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Primary outcome [29]
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Change From Baseline in Clinical Laboratory Tests: Reticulocytes/Erythrocytes
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Assessment method [29]
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Change from baseline (Day 1) to week 96 in reticulocytes/erythrocytes is presented.
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Timepoint [29]
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Baseline (Day 1), week 96
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Primary outcome [30]
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Change From Baseline in Clinical Laboratory Tests: Erythrocyte Mean Corpuscular Haemoglobin Concentration and Haemoglobin
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Assessment method [30]
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Change from baseline (Day 1) to week 96 in erythrocyte mean corpuscular haemoglobin concentration and haemoglobin is presented.
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Timepoint [30]
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Baseline (Day 1), week 96
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Primary outcome [31]
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Change From Baseline in Clinical Laboratory Tests: Erythrocyte Mean Corpuscular Haemoglobin
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Assessment method [31]
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Change from baseline (Day 1) to week 96 in erythrocyte mean corpuscular haemoglobin is presented.
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Timepoint [31]
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Baseline (Day 1), week 96
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Primary outcome [32]
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Change From Baseline in Clinical Laboratory Tests: Erythrocyte Mean Corpuscular Volume
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Assessment method [32]
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Change from baseline (Day 1) to week 96 in erythrocyte mean corpuscular volume is presented.
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Timepoint [32]
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Baseline (Day 1), week 96
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Primary outcome [33]
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Change From Baseline in Clinical Laboratory Tests: Erythrocytes
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Assessment method [33]
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Change from baseline (Day 1) to week 96 in erythrocytes is presented.
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Timepoint [33]
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Baseline (Day 1), week 96
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Primary outcome [34]
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Change From Baseline in Clinical Laboratory Tests: Specific Gravity
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Assessment method [34]
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Change from baseline (Day 1) to week 96 in specific gravity is reported.
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Timepoint [34]
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Baseline (Day 1), week 96
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Primary outcome [35]
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Change From Baseline in Clinical Laboratory Tests: Urine Erythrocytes and Urine Leucocytes
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Assessment method [35]
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Change from baseline (Day 1) to week 96 in urine erythrocytes and urine leucocytes is presented.
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Timepoint [35]
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Baseline (Day 1), week 96
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Primary outcome [36]
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Change From Baseline in Clinical Laboratory Tests: Potential of Hydrogen (pH)
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Assessment method [36]
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Change from baseline (Day 1) to week 96 in pH is presented.
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Timepoint [36]
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Baseline (Day 1), week 96
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Primary outcome [37]
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Change From Baseline in Clinical Laboratory Tests: Activated Partial Thromboplastin Time and Prothrombin Time
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Assessment method [37]
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Change from baseline (Day 1) to week 96 in activated partial thromboplastin time and prothrombin time is presented.
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Timepoint [37]
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Baseline (Day 1), week 96
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Primary outcome [38]
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Change From Baseline in Clinical Laboratory Tests: Prothrombin International Normalized Ratio
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Assessment method [38]
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Change from baseline (Day 1) to week 96 in prothrombin international normalized ratio is presented.
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Timepoint [38]
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Baseline (Day 1), week 96
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Primary outcome [39]
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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
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Assessment method [39]
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Change from baseline (Day 1) to week 96 in 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 is presented.
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Timepoint [39]
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Baseline (Day 1), week 96
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Primary outcome [40]
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Change From Baseline in Clinical Laboratory Tests: C-Reactive Protein
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Assessment method [40]
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Change from baseline (Day 1) to week 96 in C-reactive protein is presented.
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Timepoint [40]
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Baseline (Day 1), week 96
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Primary outcome [41]
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Change From Baseline in Clinical Laboratory Tests: Complement Bb
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Assessment method [41]
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Change from baseline (Day 1) to week 96 in complement Bb is presented.
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Timepoint [41]
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Baseline (Day 1), week 96
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Primary outcome [42]
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Change From Baseline in Clinical Laboratory Tests: Complement Total (CH50)
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Assessment method [42]
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Change from baseline (Day 1) to week 96 in CH50 is presented.
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Timepoint [42]
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Baseline (Day 1), week 96
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Primary outcome [43]
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Cohort 1: Change From Baseline in Serum Alpha-1 Antitrypsin (AAT) Protein Concentrations
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Assessment method [43]
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Change from baseline (Day 1) to week 24 in serum AAT protein concentrations in Cohort 1 is presented.
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Timepoint [43]
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Baseline (Day 1), week 24
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Primary outcome [44]
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Cohort 2: Change From Baseline in Serum AAT Protein Concentrations
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Assessment method [44]
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Change from baseline (Day 1) to week 48 in serum AAT protein concentrations in Cohort 2 is presented.
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Timepoint [44]
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Baseline (Day 1), week 48
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Secondary outcome [1]
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Change From Baseline in Liver Fibrosis Ishak Score
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Assessment method [1]
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Change from Baseline up until week 48 in liver fibrosis based on Ishak score is presented. The Ishak staging system for liver fibrosis is a 1995 update to the algorithm initially developed by De Groote et al. Ishak scores range from 0 (no fibrosis) to 6 (cirrhosis) which are as follows: 0-no fibrosis, 1-fibrous expansion of some portal areas, with or without short fibrous septa, 2-Fibrous expansion of most portal areas, with or without short fibrous septa, 3-Fibrous expansion of most portal areas with occasional portal-to-portal bridging, 4-Fibrous expansion of portal areas with marked bridging (portal to portal as well as portal to central), 5-Marked bridging (portal-portal and/or portal-central) with occasional nodules (incomplete cirrhosis), 6-Cirrhosis, probable or definite.
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Timepoint [1]
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Baseline (Day 1), week 48
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Eligibility
Key inclusion criteria
* 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.
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Minimum age
18
Years
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Maximum age
75
Years
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Sex
Both males and females
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Can healthy volunteers participate?
No
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Key exclusion criteria
* 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.
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Study design
Purpose of the study
Other
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Allocation to intervention
Randomised controlled trial
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Blinded (masking used)
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Who is / are masked / blinded?
The people receiving the treatment/s
The people assessing the outcomes
The people analysing the results/data
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Intervention assignment
Parallel
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Other design features
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Phase
Phase 2
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Stopped early
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
12/02/2021
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
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Actual
29/05/2024
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Sample size
Target
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Accrual to date
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Final
16
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Recruitment in Australia
Recruitment state(s)
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Recruitment hospital [1]
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St Vincent's Hospital Melbourne - Melbourne
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Recruitment postcode(s) [1]
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0
- Melbourne
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Recruitment outside Australia
Country [1]
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0
United States of America
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State/province [1]
0
0
California
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Country [2]
0
0
United States of America
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State/province [2]
0
0
Florida
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Country [3]
0
0
United States of America
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State/province [3]
0
0
South Carolina
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Country [4]
0
0
Austria
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State/province [4]
0
0
Innsbruck
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Country [5]
0
0
Belgium
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State/province [5]
0
0
Leuven
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Country [6]
0
0
Canada
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State/province [6]
0
0
Quebec
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Country [7]
0
0
France
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State/province [7]
0
0
Pessac
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Country [8]
0
0
Germany
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State/province [8]
0
0
Aachen
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Country [9]
0
0
Germany
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State/province [9]
0
0
Kiel
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Country [10]
0
0
Ireland
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State/province [10]
0
0
Dublin
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Country [11]
0
0
Netherlands
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State/province [11]
0
0
Leiden
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Country [12]
0
0
New Zealand
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State/province [12]
0
0
Auckland
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Country [13]
0
0
New Zealand
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State/province [13]
0
0
Hamilton
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Country [14]
0
0
Portugal
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State/province [14]
0
0
Creixomil
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Country [15]
0
0
Portugal
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State/province [15]
0
0
Porto
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Country [16]
0
0
Portugal
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State/province [16]
0
0
Vila Real
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Country [17]
0
0
Spain
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State/province [17]
0
0
Cantabria
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Country [18]
0
0
Spain
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State/province [18]
0
0
Madrid
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Country [19]
0
0
Sweden
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State/province [19]
0
0
Uppsala
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Country [20]
0
0
United Kingdom
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State/province [20]
0
0
Cambridge
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Country [21]
0
0
United Kingdom
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State/province [21]
0
0
Leeds
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Country [22]
0
0
United Kingdom
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State/province [22]
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0
London
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Funding & Sponsors
Primary sponsor type
Commercial sector/industry
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Name
Dicerna Pharmaceuticals, Inc., a Novo Nordisk company
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Address
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Country
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Ethics approval
Ethics application status
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Summary
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 (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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Trial website
https://clinicaltrials.gov/study/NCT04764448
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
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Thomas Bowman, MD
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Dicerna Pharmaceuticals / Novo Nordisk
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Attachment
Study protocol
https://cdn.clinicaltrials.gov/large-docs/48/NCT04764448/Prot_000.pdf
Statistical analysis plan
https://cdn.clinicaltrials.gov/large-docs/48/NCT04764448/SAP_001.pdf
Results publications and other study-related documents
No documents have been uploaded by study researchers.
Results are available at
https://clinicaltrials.gov/study/NCT04764448
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