Phase 4, Double-blind Study Evaluating the Response on Computed Tomography (CT) Lung Density Decline Rates of Respreeza / Zemaira Weekly for 3 Years in Adults With alpha1 Antitrypsin Deficiency (AATD)
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: CE1226.
- Who it may be relevant to
- Registry conditions: Alpha1 Antitrypsin Deficiency, Alpha1-Proteinase Inhibitor Deficiency, Emphysema. Basic parameters: 18 years — 65 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- Center list to be confirmed — check the primary protocol.
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official title
A Phase 4, Multicenter, Double-blind, Study to Investigate the Efficacy, Safety, and Tolerability of 3 Active Doses of Respreeza® / Zemaira® Weekly Intravenous Infusions Administered Over 3 Years as Longterm Maintenance Therapy in Adult Subjects With Emphysema Related to Alpha1 Antitrypsin Deficiency
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).
Interventions
- Biological CE1226
CE1226 will be administered via intravenous (IV) infusion weekly over 3 years.
Primary outcome measures
- Annual rate of change in adjusted lung density [Time frame: From Baseline to Month 36]
Secondary outcome measures (6)
- Annual rate of change in forced expiratory volume in 1 second percent predicted (FEV1%) [Time frame: From Baseline to Month 36]
- Annual rate of change in diffusion capacity of carbon monoxide (DLco) [Time frame: From Baseline to Month 36]
- Number of severe pulmonary exacerbations [Time frame: From screening up to Month 36]
- Duration of severe pulmonary exacerbations [Time frame: From screening up to Month 36]
- Number of participants experiencing treatment emergent adverse events (TEAEs) [Time frame: From screening up to Month 36]
- Percentage of participants experiencing TEAEs [Time frame: From screening up to Month 36]
Eligibility criteria
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.
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- Randomized
- Model
- Parallel assignment
- Masking
- Quadruple blind
- Primary purpose
- Treatment
Study locations
Center list to be confirmed — check the primary protocol.
Identifiers
NCT: NCT07326592 · CE1226_4003 · 2025-522964-33-00