NCT07431112

Brief 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.

Trial Health

80
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
54

participants targeted

Target at P50-P75 for phase_1

Timeline
29mo left

Started Mar 2026

Typical duration for phase_1

Geographic Reach
3 countries

6 active sites

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress14%
Mar 2026Jan 2029

First Submitted

Initial submission to the registry

February 19, 2026

Completed
5 days until next milestone

First Posted

Study publicly available on registry

February 24, 2026

Completed
21 days until next milestone

Study Start

First participant enrolled

March 17, 2026

Completed
2.6 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

November 1, 2028

Expected
2 months until next milestone

Study Completion

Last participant's last visit for all outcomes

January 1, 2029

Last Updated

July 24, 2026

Status Verified

July 1, 2026

Enrollment Period

2.6 years

First QC Date

February 19, 2026

Last Update Submit

July 22, 2026

Conditions

Alpha 1 Antitrypsin Deficiency

Keywords

Lung DiseasesLiver DiseasesRespiratory Tract DiseasesGenetic DiseaseInborn Congenital, Hereditary, Neonatal Diseases and AbnormalitiesSubcutaneous EmphysemaEmphysemaPathologic ProcessesPathological Conditions, Signs and SymptomsAlpha 1 Antitrypsin Deficiency

Outcome Measures

Primary Outcomes (4)

  • 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

Secondary Outcomes (3)

  • Area under the plasma concentration time curve for AIR-001 from time of dosing to the last measurable concentration (AUClast)

    Up to Day 169

  • Maximum concentration of AIR-001 in plasma (Cmax)

    Up to Day 169

  • Change from baseline in levels of total serum AAT protein, including serum M-AAT, Z-AAT, and functional AAT protein

    Up to Day 169

Study Arms (10)

AIR-001 SAD Cohort 1

EXPERIMENTAL

AIR-001 is an RNA-editing oligonucleotide in sterile preservative-free solution administered by subcutaneous (SC) injection as a single dose.

Drug: AIR-001 SAD dose level 1

AIR-001 SAD Cohort 2

EXPERIMENTAL

AIR-001 administered by SC injection as a single dose.

Drug: AIR-001 SAD dose level 2

AIR-001 SAD Cohort 3

EXPERIMENTAL

AIR-001 administered by SC injection as a single dose.

Drug: AIR-001 SAD dose level 3

AIR-001 SAD Cohort 4

EXPERIMENTAL

AIR-001 administered by SC injection, single dose.

Drug: AIR-001 SAD dose level 4

AIR-001 MD Cohort 1

EXPERIMENTAL

AIR-001 is an RNA-editing oligonucleotide in sterile preservative-free solution administered by subcutaneous (SC) injection as multiple doses.

Drug: AIR-001 MD dose level 1

AIR-001 MD Cohort 2

EXPERIMENTAL

AIR-001 administered by SC injection as multiple doses.

Drug: AIR-001 MD dose level 2

AIR-001 MD Cohort 3

EXPERIMENTAL

AIR-001 administered by SC injection as multiple doses.

Drug: AIR-001 MD dose level 3

AIR-001 MD Cohort 4

EXPERIMENTAL

AIR-001 administered by SC injection as multiple doses.

Drug: AIR-001 MD dose level 4

AIR-001 MD Cohort 5

EXPERIMENTAL

AIR-001 administered by SC injection as multiple doses.

Drug: AIR-001 MD dose level 5

AIR-001 MD Cohort 6

EXPERIMENTAL

AIR-001 administered by SC injection as multiple doses.

Drug: AIR-001 MD dose level 6

Interventions

AIR-001 SAD dose level 1DRUG

AIR-001 drug product, SC

AIR-001 SAD Cohort 1
AIR-001 SAD dose level 2DRUG

AIR-001 drug product, SC

AIR-001 SAD Cohort 2
AIR-001 SAD dose level 3DRUG

AIR-001 drug product, SC

AIR-001 SAD Cohort 3
AIR-001 SAD dose level 4DRUG

AIR-001 drug product, SC

AIR-001 SAD Cohort 4
AIR-001 MD dose level 1DRUG

AIR-001 drug product, SC

AIR-001 MD Cohort 1
AIR-001 MD dose level 2DRUG

AIR-001 drug product, SC

AIR-001 MD Cohort 2
AIR-001 MD dose level 3DRUG

AIR-001 drug product, SC

AIR-001 MD Cohort 3
AIR-001 MD dose level 4DRUG

AIR-001 drug product, SC

AIR-001 MD Cohort 4
AIR-001 MD dose level 5DRUG

AIR-001 drug product, SC

AIR-001 MD Cohort 5
AIR-001 MD dose level 6DRUG

AIR-001 drug product, SC

AIR-001 MD Cohort 6

Eligibility Criteria

Age18 Years - 74 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • 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
  • Negative beta human chorionic gonadotropin (β-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 may not qualify if:

  • 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:
  • Alanine aminotransferase (ALT), aspartate aminotransferase (AST), or gamma-glutamyl transferase (GGT) \> 3 x upper limit of normal (ULN)
  • Platelet count ≤ 150 k/μL
  • Estimated glomerular filtration rate (eGFR) ≤ 60 mL/min/1.73m² by Chronic Kidney Disease Epidemiology Collaboration (CKD EPI) equation
  • Urine Albumin-to-Creatinine Ratio \> 300 mg/g
  • Urine Protein-to-Creatinine Ratio \> 500 mg/g
  • Prolonged QT interval on electrocardiogram (ECG), defined as QTcF ≥ 450ms (men) or ≥ 470ms (women)
  • ECG findings at screening that render measurements of QT interval imprecise.
  • +12 more criteria

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (6)

Clinical Study Center

Melbourne, Australia

RECRUITING

Clinical Study Center 1

Tbilisi, Georgia

RECRUITING

Clinical Study Center 2

Tbilisi, Georgia

RECRUITING

Clinical Study Center

Cambridge, United Kingdom

RECRUITING

Clinical Study Center

London, United Kingdom

RECRUITING

Clinical Study Center

Southampton, United Kingdom

RECRUITING

MeSH Terms

Conditions

alpha 1-Antitrypsin DeficiencyLung DiseasesLiver DiseasesRespiratory Tract DiseasesGenetic Diseases, InbornInfant, Newborn, DiseasesCongenital AbnormalitiesSubcutaneous EmphysemaEmphysemaPathologic ProcessesPathological Conditions, Signs and Symptoms

Condition Hierarchy (Ancestors)

Digestive System DiseasesCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

  • Clinical Information

    AIRNA Corporation

    STUDY DIRECTOR

Central Study Contacts

AIRNA Corporation Clinical Trials Information

CONTACT

617-609-8790patients@airna.com

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
SEQUENTIAL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

February 19, 2026

First Posted

February 24, 2026

Study Start

March 17, 2026

Primary Completion (Estimated)

November 1, 2028

Study Completion (Estimated)

January 1, 2029

Last Updated

July 24, 2026

Record last verified: 2026-07

Data Sharing

IPD Sharing
Will not share

Locations

GB(3)GE(2)AU(1)