AATD, Alpha-1 Anti-trypsin Deficiency
Conditions
Brief summary
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).
Interventions
DRUGKRRO-110
KRRO-110 drug product, IV
Sponsors
Korro Bio, Inc.
Study design
Allocation
RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT
Masking
DOUBLE (Subject, Investigator)
Eligibility
Sex/Gender
ALL
Age
18 Years to 70 Years
Healthy volunteers
Yes
Inclusion criteria
Part A SAD cohort Inclusion Criteria (Healthy Volunteers) * Adult male or female participants, 18 to 65 years of age * Documented PiMM genotype * Participants who are willing to and able to provide signed written informed consent PiZZ Genotype (Part A SAD and Part B MAD cohorts) Inclusion Criteria (PiZZ Genotype) * Adult male or female participants 18 to 70 years of age (inclusive) * Documented PiZZ genotype * Baseline blood total AAT level \< 11 µM/L
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Safety and Tolerability | From Day 1 to Day 43 in the SAD and from Day 1 to Day 85 in the MAD | Type, frequency, and severity of treatment-emergent adverse events (TEAEs) |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Pharmacokinetics (PK) of KRRO-110 | Day 1 to Day 43 (SAD), and Day 1 to Day 85 (MAD) | PK profile of KRRO-110 including maximum blood concentration (Cmax) |
| Pharmacodynamics (PD) of KRRO-110 | Day 1 to Day 43 (SAD), and Day 1 to Day 85 (MAD) | PD profile of KRRO-110 based on change from Baseline in alpha-antitrypsin (AAT) enzyme levels at all measured timepoints (PiZZ participants only). |
Countries
Australia, New Zealand
Contacts
STUDY_DIRECTORStudy Director
Korro Bio, Inc.
Outcome results
None listed