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Recruiting Phase 1

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

NCT07431112 · tracked via the Priya Life Science Spain tracker
Sponsor
AIRNA Corporation
Phase
Phase 1
Started
2026-03-17
Last updated
2026-10-06

Condition(s) studied

Alpha 1 Antitrypsin Deficiency

Investigational drug(s) / intervention(s)

AIR-001 SAD dose level 1AIR-001 SAD dose level 2AIR-001 SAD dose level 3AIR-001 SAD dose level 4AIR-001 MD dose level 1AIR-001 MD dose level 2AIR-001 MD dose level 3AIR-001 MD dose level 4AIR-001 MD dose level 5AIR-001 MD dose level 6

AIR-001 SAD dose level 1: AIR-001 drug product, SC

AIR-001 SAD dose level 2: AIR-001 drug product, SC

AIR-001 SAD dose level 3: AIR-001 drug product, SC

AIR-001 SAD dose level 4: AIR-001 drug product, SC

AIR-001 MD dose level 1: AIR-001 drug product, SC

AIR-001 MD dose level 2: AIR-001 drug product, SC

AIR-001 MD dose level 3: AIR-001 drug product, SC

AIR-001 MD dose level 4: AIR-001 drug product, SC

AIR-001 MD dose level 5: AIR-001 drug product, SC

AIR-001 MD dose level 6: AIR-001 drug product, SC

Study 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

Sex
ALL
Min age
18 Years
Max age
74 Years
Healthy volunteers
No
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 investigational medicinal product (IMP). 11. Willing and able to comply with the study design schedule, all study procedures, and other requirements Exclusion Criteria: 1. Female participants who are nursing or lactating 2. 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. 3. Known or suspected allergy or intolerance to AIR-001 or its components 4. 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. 5. Positive screening test for COVID-19 and/or Influenza. 6. 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. 7. 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. 8. Any of the following screening laboratory abnormalities: 1. Alanine aminotransferase (ALT), aspartate aminotransferase (AST), or gamma-glutamyl transferase (GGT) \> 3 x upper limit of normal (ULN) 2. Total bilirubin \> ULN (note: for participants with documented Gilbert's syndrome and direct bilirubin ≤ ULN , exclusion criterion is total bilirubin is \> 2.5 mg/dL) 3. INR \> ULN (for participants taking stable doses of anticoagulants, the exclusion criterion is INR \> 3.0) 4. Platelet count ≤ 150 k/μL 5. Estimated glomerular filtration rate (eGFR) ≤ 60 mL/min/1.73m² by Chronic Kidney Disease Epidemiology Collaboration (CKD EPI) equation 6. Urine Albumin-to-Creatinine Ratio \> 300 mg/g 7. Urine Protein-to-Creatinine Ratio \> 500 mg/g 9. Prolonged QT interval on electrocardiogram (ECG), defined as QTcF ≥ 450ms (men) or ≥ 470ms (women) 10. ECG findings at screening that render measurements of QT interval imprecise. 11. History of congestive heart failure, serious cardiac arrythmias requiring anti-arrhythmic medications or unexplained black-outs or fainting episodes with a suspected cardiac origin 12. Positive screening test or known chronic infection with Hepatitis B, Hepatitis C, or HIV. 13. Known history of coagulopathy or bleeding diathesis 14. History or intolerance to subcutaneous (SC) injection including relevant dermatological conditions affecting standard injection sites 15. History or presence of any medical condition, behavioral or psychiatric disorder, or planned surgical procedure or surgical history that may interfere with participation in the study or interpretation of study results, and/or put the participant at significant risk (in the opinion of the investigator) if he/she participates in the study. 16. History of any lung-volume reduction procedure in the 6 months prior to screening. 17. Laboratory value(s) outside the laboratory reference range that is (are) considered to be clinically significant and may affect the safety, efficacy, PK, or PD assessments or interpretation by the Investigator, at screening 18. History of alcohol or drug abuse within the past three months 19. Current or previous participation in any other clinical study where the participant has received a dose of an IMP within 3 months or 5 half-lives of the IMP, whichever is longest, prior to Screening Visit 20. Any previous gene replacement or DNA-editing therapy 21. Any previous use of an RNA-based therapeutic (except for AIR-001 or RNA-based vaccines) within the 6 months prior to the Screening Visit or at any time if stopped due to drug-related adverse event. 22. Use of any new prescription, vaccine, herbal remedy, over-the-counter medication, or supplement, or changes in chronic therapies within the 28 days prior to dosing unless approved by study Medical Monitor.

Primary outcome measure(s)

  • Number of participants with treatment-emergent adverse events (TEAEs) — Up to Day 169
  • Incidence of laboratory abnormalities and shifts from baseline, including hematology, chemistry, coagulation, and urinalysis parameters — Baseline through up to Day 169
  • Incidence of abnormal vital signs — Baseline through up to Day 169
  • Incidence of abnormal electrocardiogram (ECG) findings — Baseline through up to Day 169

Trial sites (12)

FacilityCityRegionStatus
Clinical Study Center Melbourne Australia Recruiting
Clinical Study Center 1 Tbilisi Georgia Withdrawn
Clinical Study Center 2 Tbilisi Georgia Terminated
Clinical Study Center Tübingen Germany Recruiting
Clinical Study Center Leiden Netherlands Recruiting
Clinical Study Center Auckland New Zealand Recruiting
Clinical Study Center Matosinhos Municipality Portugal Recruiting
Clinical Study Center Barcelona Spain Recruiting
Clinical Study Center Stockholm Sweden Recruiting
Clinical Study Center Cambridge United Kingdom Recruiting
Clinical Study Center London United Kingdom Recruiting
Clinical Study Center Southampton United Kingdom Recruiting
Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT07431112 on ClinicalTrials.gov ↗ ← All trials in Spain