A Study of AIR-001 in Adults With Alpha-1 Antitrypsin Deficiency (AATD) (NCT07431112) | Clinical Trial Compass
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 range
18 Years – 74 Years
Sex
ALL
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.
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Number of participants with treatment-emergent adverse events (TEAEs)
Timeframe: Up to Day 169
2
Incidence of laboratory abnormalities and shifts from baseline, including hematology, chemistry, coagulation, and urinalysis parameters
Timeframe: Baseline through up to Day 169
3
Incidence of abnormal vital signs
Timeframe: Baseline through up to Day 169
4
Incidence of abnormal electrocardiogram (ECG) findings
. 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: