Overview
This is a multicenter, parallel-group, double-blind, randomized phase 4 study designed to identify the optimal dose of CE1226 (2 active doses) to slow disease progression as assessed by reduced rates of annual lung density decline in alpha-1 antitrypsin (AAT) deficient participants over 3 years as compared with the marketed dose 60 milligrams per kilogram (mg/kg).
Eligibility
Inclusion Criteria:
- Age greater than or equal to (\>=) 18 and less than or equal to (\<=) 65 years at the time of providing written informed consent.
- Confirmed diagnosis of emphysema related to AATD with either the PiZZ, PiZ(null), or Pi(null/null) genotype with documented serum AAT levels less than (\<) 11 micrometer (μM) (or \< 50 mg/dL \[milligram/deciliter\]) at any time before the first administration of CE1226 on Day 1 (Baseline).
Exclusion Criteria:
- Participants should not have acute illness or pulmonary exacerbation within 6 weeks before the first administration of CE1226 on Day 1 (Baseline).
- Participants should not have previously received gene therapy for AATD at any point.
- Participants with liver disease secondary to AATD.
