The purpose of this study is to evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Subcutaneously Administered ALN-AAT in Healthy Adult Subjects and Patients with ZZ Type Alpha-1 Antitrypsin Deficiency Liver Disease.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
SINGLE
Enrollment
26
Single or multiple doses of ALN-AAT by subcutaneous (sc) injection
calculated volume to match active comparator
Richmond Pharmacology, Ltd.
London, United Kingdom
The safety of ALN-AAT evaluated by the proportion of subjects experiencing adverse events (AEs), serious adverse events (SAEs), and AEs leading to study drug discontinuation
Time frame: Part A (SAD phase): through day 70; Part B (MAD) phase: through Day 154; Part C: through Day 224
Profile of Pharmacokinetics (PK) of ALN-AAT
Cmax, tmax, AUC, t1/2
Time frame: Part A (SAD) phase: up to 21 days; Part B (MAD) phase: up to 105 days; Part C: up to 161 days
The effect of ALN-AAT on serum levels of AAT protein
Time frame: Part A (SAD) phase: through day 70; Part B (MAD) phase: through Day 154; Part C: through Day 224
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