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 Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
OTHER
Masking
NONE
Belcesiran will be administered subcutaneously (SC) in the treatment arm.
Auckland Clinical Studies
Grafton, Auckland, New Zealand
The incidence of treatment-emergent adverse events
Time frame: up to 152 weeks
The change from baseline in pulmonary function tests (PFTs)
Forced expiratory volume in 1 second (FEV1)
Time frame: up to 152 weeks
The change from baseline in PFTs
Forced vital capacity (FVC)
Time frame: up to 152 weeks
The change from baseline in PFTs
FEV1/FVC
Time frame: up to 152 weeks
The change from baseline in PFTs
diffusing capacity for carbon monoxide (DLCO)
Time frame: up to 152 weeks
The change from baseline in 12-lead electrocardiogram (ECG)
heart rate
Time frame: up to 56 weeks
The change from baseline in ECG
ventricular rate
Time frame: up to 56 weeks
The change from baseline in 12-lead ECG
RR interval
Time frame: up to 56 weeks
The change from baseline in 12-lead ECG
PR interval
Time frame: up to 56 weeks
The change from baseline in 12-lead ECG
QRS duration
Time frame: up to 56 weeks
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The change from baseline in 12-lead ECG
QT interval
Time frame: up to 56 weeks
The change from baseline in 12-lead ECG
corrected QT interval (QTcF, Fridericia correction)
Time frame: up to 56 weeks
The change from baseline in physical examination (PE) findings
body weight
Time frame: up to 56 weeks
The change from baseline in PE findings
body-mass index (BMI) (using height from DCR-A1AT-201 study)
Time frame: up to 56 weeks
The change from baseline in PE findings
physical examination to assess skin, lungs, cardiovascular system, and abdomen (liver and spleen) based on the National Cancer Institute Common Terminology Criteria for Adverse Events (CTCAE; version 5.0) grading scale
Time frame: up to 56 weeks
The change from baseline in vital sign measurements
blood pressure
Time frame: up to 56 weeks
The change from baseline in vital sign measurements
pulse rate
Time frame: up to 56 weeks
The change from baseline in vital sign measurements
respiratory rate
Time frame: up to 56 weeks
The change from baseline in vital sign measurements
oral temperature
Time frame: up to 56 weeks
The change from baseline in clinical laboratory tests: Hematology
Hematology is collected to evaluate the long-term safety of belcesiran
Time frame: up to 152 weeks
The change from baseline in clinical laboratory tests: Clinical Chemistry
Clinical Chemistry is collected to evaluate the long-term safety of belcesiran
Time frame: up to 152 weeks
The change from baseline in clinical laboratory tests: Coagulation
Coagulation is collected to evaluate the long-term safety of belcesiran
Time frame: up to 152 weeks
The change from baseline in clinical laboratory tests: Urinalysis
Urinalysis is collected to evaluate the long-term safety of belcesiran
Time frame: up to 152 weeks
Changes in serum AAT protein concentrations over time
Time frame: up to 152 weeks