RecruitingPhase 1

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

Australia, Georgia, United Kingdom54 participantsStarted 2026-03-17

Plain-language 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.

Who can participate

Age range18 Years – 74 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

✓. 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

Exclusion criteria

✕. Female participants who are nursing or lactating
✕. Participant has received AAT augmentation therapy within 30 days prior to Screening Visit or plans to receive AAT augmentation therapy at any time during study participation.
✕. Known or suspected allergy or intolerance to AIR-001 or its components
✕. Acute respiratory tract infection or clinically-diagnosed chronic obstructive pulmonary disease (COPD) exacerbation that required antibiotic treatment and/or systemic corticosteroids within the 8 weeks prior to dosing.
✕. Positive screening test for COVID-19 and/or Influenza.
✕. Lung disease that requires use of continuous oral corticosteroids, continuous supplemental oxygen, day-time ventilatory support, or any participant who is on a lung transplant waiting list.
✕. Liver Fibrosis score \> 10 kPa defined by screening liver elastography, historical liver biopsy showing ≥ F3 fibrosis (METAVIR or comparable scoring system), or established diagnosis of hepatic cirrhosis.
✕. Any of the following screening laboratory abnormalities:

What they're measuring

Number of participants with treatment-emergent adverse events (TEAEs)

Timeframe: Up to Day 169

Incidence of laboratory abnormalities and shifts from baseline, including hematology, chemistry, coagulation, and urinalysis parameters

Timeframe: Baseline through up to Day 169

Incidence of abnormal vital signs

Timeframe: Baseline through up to Day 169

Incidence of abnormal electrocardiogram (ECG) findings

Timeframe: Baseline through up to Day 169

Trial details

NCT IDNCT07431112

SponsorAIRNA Corporation

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2028-11

Contact for this trial

AIRNA Corporation Clinical Trials Information

617-609-8790 patients@airna.com

View on ClinicalTrials.gov More Alpha 1 Antitrypsin Deficiency trials

Who can participate

Age range18 Years – 74 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

✓. 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

Exclusion criteria

✕. Female participants who are nursing or lactating
✕. Participant has received AAT augmentation therapy within 30 days prior to Screening Visit or plans to receive AAT augmentation therapy at any time during study participation.
✕. Known or suspected allergy or intolerance to AIR-001 or its components
✕. Acute respiratory tract infection or clinically-diagnosed chronic obstructive pulmonary disease (COPD) exacerbation that required antibiotic treatment and/or systemic corticosteroids within the 8 weeks prior to dosing.
✕. Positive screening test for COVID-19 and/or Influenza.
✕. Lung disease that requires use of continuous oral corticosteroids, continuous supplemental oxygen, day-time ventilatory support, or any participant who is on a lung transplant waiting list.
✕. Liver Fibrosis score \> 10 kPa defined by screening liver elastography, historical liver biopsy showing ≥ F3 fibrosis (METAVIR or comparable scoring system), or established diagnosis of hepatic cirrhosis.
✕. Any of the following screening laboratory abnormalities:

What they're measuring

Number of participants with treatment-emergent adverse events (TEAEs)

Timeframe: Up to Day 169

Incidence of laboratory abnormalities and shifts from baseline, including hematology, chemistry, coagulation, and urinalysis parameters

Timeframe: Baseline through up to Day 169

Incidence of abnormal vital signs

Timeframe: Baseline through up to Day 169

Incidence of abnormal electrocardiogram (ECG) findings

Timeframe: Baseline through up to Day 169