NCT07431112

A Study of AIR-001 in Adults With Alpha-1 Antitrypsin Deficiency (AATD)

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

• Status: Recruiting
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 54 (estimated)
• Sponsor: AIRNA Corporation · Industry
• Sex: All
• Age: 18 Years – 74 Years
• Healthy volunteers: Not accepted

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.

Detailed description

The study is designed to evaluate the safety, tolerability, PK, and PD of AIR-001.

Conditions

• Alpha 1 Antitrypsin Deficiency

Interventions

Type	Name	Description
DRUG	AIR-001 SAD dose level 1	AIR-001 drug product, SC
DRUG	AIR-001 SAD dose level 2	AIR-001 drug product, SC
DRUG	AIR-001 SAD dose level 3	AIR-001 drug product, SC
DRUG	AIR-001 SAD dose level 4	AIR-001 drug product, SC
DRUG	AIR-001 MD dose level 1	AIR-001 drug product, SC
DRUG	AIR-001 MD dose level 2	AIR-001 drug product, SC
DRUG	AIR-001 MD dose level 3	AIR-001 drug product, SC
DRUG	AIR-001 MD dose level 4	AIR-001 drug product, SC
DRUG	AIR-001 MD dose level 5	AIR-001 drug product, SC
DRUG	AIR-001 MD dose level 6	AIR-001 drug product, SC

Timeline

• Start date: 2026-03-17
• Primary completion: 2028-11-01
• Completion: 2029-01-01
• First posted: 2026-02-24
• Last updated: 2026-04-08

Locations

3 sites across 3 countries: Australia, Georgia, United Kingdom