A Study of AIR-001 in Adults With Alpha-1 Antitrypsin Deficiency (AATD)
Trial Parameters
Brief Summary
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.
Eligibility Criteria
Inclusion Criteria: 1. Male or female participants \>18 years and \<75 years of age at the time of signing informed consent 2. Total serum AAT levels \< 11µM (57 mg/dL) 3. Pi\*ZZ genotype confirmed by DNA sequencing within the SERPINA1 gene with no known co-occurring SERPINA1 null variants 4. Spirometry: Forced expiratory volume in 1 second (FEV1) ≥ 40% of predicted 5. Non-smoker, including vaping, for at least 6 months prior to screening 6. Body mass index between 18-33.0 kg/m² 7. Body weight ≥ 45 kg and ≤110 kg 8. Willing and able to give written informed consent prior to the initiation of any study procedure by the participant 9. Negative beta human chorionic gonadotropin (β-hCG) at enrolment for women of childbearing potential (WOCBP) only. 10. 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 investigation