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
Qualifiers
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
Treatments tested in this trial
- AIR-001 SAD dose level 1
- AIR-001 SAD dose level 2
- AIR-001 SAD dose level 3
- AIR-001 SAD dose level 4
- AIR-001 MD dose level 1
- AIR-001 MD dose level 2
- AIR-001 MD dose level 3
- AIR-001 MD dose level 4
- AIR-001 MD dose level 5
- AIR-001 MD dose level 6
Treatment groups
54 Participants
are divided into 10 treatment groups10
Treatment groupsSee each treatment group below.
Sponsors and collaborators
Source ClinicalTrials.gov