The purpose of this first-in human (FIH) study is to assess the safety, tolerability, and pharmacokinetics (PK) of single and multiple doses of KRRO-110 in both healthy adult participants and in clinically stable patients with Alpha-1 antitrypsin deficiency (AATD).
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
DOUBLE
Enrollment
64
KRRO-110 drug product, IV
The Prince Charles Hospital
Brisbane, Queensland, Australia
Nucleus Network Pty Ltd
Melbourne, Victoria, Australia
St. Vincent's Hospital Melbourne
Melbourne, Victoria, Australia
New Zealand Clinical Research
Auckland, New Zealand
Safety and Tolerability
Type, frequency, and severity of treatment-emergent adverse events (TEAEs)
Time frame: From Day 1 to Day 43 in the SAD and from Day 1 to Day 85 in the MAD
Pharmacokinetics (PK) of KRRO-110
PK profile of KRRO-110 including maximum blood concentration (Cmax)
Time frame: Day 1 to Day 43 (SAD), and Day 1 to Day 85 (MAD)
Pharmacodynamics (PD) of KRRO-110
PD profile of KRRO-110 based on change from Baseline in alpha-antitrypsin (AAT) enzyme levels at all measured timepoints (PiZZ participants only).
Time frame: Day 1 to Day 43 (SAD), and Day 1 to Day 85 (MAD)
Pharmacokinetics (PK) of KRRO-110
PK profile of KRRO-110 half-life (t1/2), area under the concentration-time curve (AUC), and trough level (MAD cohorts only).
Time frame: Day 1 to Day 43 (SAD), and Day 1 to Day 85 (MAD)
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New Zealand Clinical Research
Christchurch, New Zealand