A Study of AIR-001 in Adults With Alpha-1 Antitrypsin Deficiency (AATD)
ConditionAlpha 1 Antitrypsin Deficiency
Trial statusRecruiting
Trial phasePhase 1
Trial typeInterventional
Biological sexAll
Age18-74
SponsorAIRNA Corporation
About this trial
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 criteria
This trial does not accept healthy volunteersQualifiers
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
Disqualifiers
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.
Trial design
Design model
Sequential
Treatments tested in this trial
AIR-001 SAD dose level 1
DrugAIR-001 drug product, SC
AIR-001 SAD dose level 2
DrugAIR-001 drug product, SC
AIR-001 SAD dose level 3
DrugAIR-001 drug product, SC
AIR-001 SAD dose level 4
DrugAIR-001 drug product, SC
AIR-001 MD dose level 1
DrugAIR-001 drug product, SC
AIR-001 MD dose level 2
DrugAIR-001 drug product, SC
AIR-001 MD dose level 3
DrugAIR-001 drug product, SC
AIR-001 MD dose level 4
DrugAIR-001 drug product, SC
AIR-001 MD dose level 5
DrugAIR-001 drug product, SC
AIR-001 MD dose level 6
DrugAIR-001 drug product, SC
Treatment groups
54 Participants
are divided into 10 treatment groups10
Treatment groupsSee each treatment group below.
Group A: AIR-001 SAD Cohort 1Experimental treatment 1 intervention
Group B: AIR-001 SAD Cohort 2Experimental treatment 1 intervention
Group C: AIR-001 SAD Cohort 3Experimental treatment 1 intervention
Group D: AIR-001 SAD Cohort 4Experimental treatment 1 intervention
Group E: AIR-001 MD Cohort 1Experimental treatment 1 intervention
Group F: AIR-001 MD Cohort 2Experimental treatment 1 intervention
Group G: AIR-001 MD Cohort 3Experimental treatment 1 intervention
Group H: AIR-001 MD Cohort 4Experimental treatment 1 intervention
Group I: AIR-001 MD Cohort 5Experimental treatment 1 intervention
Group J: AIR-001 MD Cohort 6Experimental treatment 1 intervention
Trial outcomes
Primary outcomes
1
Number of participants with treatment-emergent adverse events (TEAEs)
Time frame
Up to Day 1692
Incidence of laboratory abnormalities and shifts from baseline, including hematology, chemistry, coagulation, and urinalysis parameters
Time frame
Baseline through up to Day 1693
Incidence of abnormal vital signs
Time frame
Baseline through up to Day 1694
Incidence of abnormal electrocardiogram (ECG) findings
Time frame
Baseline through up to Day 169Secondary outcomes
1
Area under the plasma concentration time curve for AIR-001 from time of dosing to the last measurable concentration (AUClast)
Time frame
Up to Day 1692
Maximum concentration of AIR-001 in plasma (Cmax)
Time frame
Up to Day 1693
Change from baseline in levels of total serum AAT protein, including serum M-AAT, Z-AAT, and functional AAT protein
Time frame
Up to Day 169Sponsors and contacts
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Source ClinicalTrials.gov