Actively Recruiting
A Study of AIR-001 in Adults With Alpha-1 Antitrypsin Deficiency (AATD)
Led by AIRNA Corporation · Updated on 2026-04-28
54
Participants Needed
4
Research Sites
145 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
A Study of AIR-001 in Adults With Alpha-1 Antitrypsin Deficiency (AATD)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Male or female participants >18 years and <75 years of age at the time of signing informed consent
- Total serum AAT levels < 11�b5M (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) �b1 40% of predicted
- Non-smoker, including vaping, for at least 6 months prior to screening
- Body mass index between 18-33.0 kg/m�b2
- Body weight �b1 45 kg and �b1110 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 (�b2-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
You will not qualify if you...
- 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 COPD exacerbation that required antibiotic treatment and/or systemic corticosteroids within 8 weeks prior to dosing
- Positive screening test for COVID-19 and/or Influenza
- Lung disease requiring continuous oral corticosteroids, continuous supplemental oxygen, daytime ventilatory support, or on lung transplant waiting list
- Liver fibrosis score > 10 kPa by screening liver elastography, historical liver biopsy showing �b3 fibrosis, or diagnosed hepatic cirrhosis
- Specific abnormal screening laboratory values including elevated liver enzymes, bilirubin, INR, low platelet count, reduced kidney function, or high urine protein or albumin levels
- Prolonged QT interval on ECG above specified thresholds
- ECG findings that impair QT interval measurement
- History of congestive heart failure, serious cardiac arrhythmias needing medication, or unexplained blackouts or fainting thought to be cardiac
- Positive screening or known chronic infection with Hepatitis B, Hepatitis C, or HIV
- Known history of coagulopathy or bleeding disorder
- History or intolerance to subcutaneous injections including skin conditions affecting injection sites
- Medical, behavioral, psychiatric, or surgical conditions that may interfere with study participation or pose significant risk
- History of lung-volume reduction procedure within 6 months before screening
- Clinically significant abnormal laboratory values at screening
- History of alcohol or drug abuse within past 3 months
- Participation in another clinical study with investigational medicinal product within 3 months or 5 half-lives before screening
- Previous gene replacement or DNA-editing therapy
- Previous use of RNA-based therapy except AIR-001 or RNA vaccines within 6 months prior to screening or if stopped due to drug-related adverse event
- Use of new medications, vaccines, herbal remedies, supplements, or changes in chronic therapies within 28 days prior to dosing unless approved by Medical Monitor
AI-Screening
AI-Powered Screening
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Trial Site Locations
Total: 4 locations
1
Clinical Study Center
Melbourne, Australia
Actively Recruiting
2
Clinical Study Center 1
Tbilisi, Georgia
Actively Recruiting
3
Clinical Study Center 2
Tbilisi, Georgia
Actively Recruiting
4
Clinical Study Center
London, United Kingdom
Actively Recruiting
Research Team
A
AIRNA Corporation Clinical Trials Information
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
10
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