Phase 1, Open-Label, Single Ascending Dose and Multiple Dose Study to Assess Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Subcutaneously Administered AIR-001 in Adults With AATD Due to PiZZ Genotype
This is a Phase 1, open-label, single ascending dose (SAD) and multiple dose (MD) study of AIR-001 in participants with alpha-1 antitrypsin deficiency (AATD) due to PiZZ genotype.
• Male or female participants \>18 years and \<75 years of age at the time of signing informed consent
• Total serum AAT levels \< 11µM (57 mg/dL)
• Pi\*ZZ genotype confirmed by DNA sequencing within the SERPINA1 gene with no known co-occurring SERPINA1 null variants
• Spirometry: Forced expiratory volume in 1 second (FEV1) ≥ 40% of predicted
• Non-smoker, including vaping, for at least 6 months prior to screening
• Body mass index between 18-33.0 kg/m²
• Body weight ≥ 45 kg and ≤110 kg
• Willing and able to give written informed consent prior to the initiation of any study procedure by the participant
• Negative beta human chorionic gonadotropin (β-hCG) at enrolment for women of childbearing potential (WOCBP) only.
⁃ Participants who are either a WOCBP or male participant who is heterosexually active with a WOCBP must consent to use a highly effective method of contraception from screening visit until at least 4 weeks after the last dose of investigational medicinal product (IMP).
⁃ Willing and able to comply with the study design schedule, all study procedures, and other requirements