Alpha1 Antitrypsin DeficiencyPhase 4NCT07326592
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)
A Phase 4 study testing CE1226 in people with Alpha1 Antitrypsin Deficiency and Alpha1-Proteinase Inhibitor Deficiency, run by CSL Behring at 2 sites in Denmark.
Trial summary
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… Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Alpha1 Antitrypsin Deficiency, Alpha1-Proteinase Inhibitor Deficiency, Emphysema
- What is being tested?
- CE1226
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 270 participants
- Where
- 2 sitesDenmark
- Study dates
- 29 Sep 2026to 15 Sep 2033
- Phase
- Phase 4
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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).
Who cannot join
- 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.
Trial locations and contacts
2 sites in 1 country. Contact the study team or a site near you to ask about taking part.
Denmark2 sites, 2 recruiting
- Recruiting
Aalborg Universitetshospital, Syd
Aalborg, 09000
- Recruiting
Sygehus Lillebælt, Vejle
Vejle, 7100
About this study
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
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).
- Being tested
- CE1226
- Keywords
- Progressive Emphysema, Chronic Pulmonary Disease
- Medical subject headings
- alpha 1-Antitrypsin Deficiency, Emphysema
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CE1226Biological
Also called Human alpha1-proteinase inhibitor, Respreeza, Zemaira
CE1226 will be administered via intravenous (IV) infusion weekly over 3 years.
| Study group | Role | What they receive |
|---|---|---|
| CE1226 low dose | Active comparator | CE1226 at low dose. Receives: Biological: CE1226 |
| CE1226 medium dose | Experimental | CE1226 at medium dose. Receives: Biological: CE1226 |
| CE1226 high dose | Experimental | CE1226 at high dose. Receives: Biological: CE1226 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Annual rate of change in adjusted lung density
The annual rate of change (expressed as gram per liter per year [g/L/year]) in adjusted lung density (15th percentile lung density [PD15]) will be calculated on CT scan assessments of whole lung at total lung capacity (TLC).
Measured: From Baseline to Month 36
Secondary outcomes (6)
Annual rate of change in forced expiratory volume in 1 second percent predicted (FEV1%)
The annual rate of change in FEV1% will be assessed by spirometry.
Measured: From Baseline to Month 36
Annual rate of change in diffusion capacity of carbon monoxide (DLco)
The annual rate of change in DLco will be assessed by gas transfer using site machinery.
Measured: From Baseline to Month 36
Number of severe pulmonary exacerbations
Pulmonary excerbations will be assessed by the investigator using Rodriguez-Roisin criteria, where an exacerbation is defined as: a sustained worsening of the patient's condition, from a stable state and beyond normal day-to-day variations, necessitating a change in regular medication in a patient with underlying chronic obstructive pulmonary disease (COPD). A severe exacerbation is defined as an event requiring the introduction of corticosteroids and / or antibiotics which results in hospitalization or death.
Measured: From screening up to Month 36
Duration of severe pulmonary exacerbations
Pulmonary excerbations will be assessed by the investigator using Rodriguez-Roisin criteria, where an exacerbation is defined as: a sustained worsening of the patient's condition, from a stable state and beyond normal day-to-day variations, necessitating a change in regular medication in a patient with underlying COPD. A severe exacerbation is defined as an event requiring the introduction of corticosteroids and / or antibiotics which results in hospitalization or death.
Measured: From screening up to Month 36
Number of participants experiencing treatment emergent adverse events (TEAEs)
Measured: From screening up to Month 36
Percentage of participants experiencing TEAEs
Measured: From screening up to Month 36
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Jan 2026
- Study start29 Sep 2026
- Primary completion15 Sep 2033 (estimated)
- Study completion15 Sep 2033 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Alpha1 Antitrypsin DeficiencyStudies of the same condition, open and under way.
- More trials run by CSL BehringThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 4
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- This is a double blind, parallel group assignment study.
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 270 participants (estimated)
Sponsor and investigators
- Lead sponsor
- CSL Behring
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Study Director, Study director, CSL Behring
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07326592
- Sponsor's protocol ID
- CE1226_4003
- Other IDs
- 2025-522964-33-00, 2025
- Status last verified
- Oct 2026
- First submitted
- 7 Jan 2026
- Last updated
- 16 Oct 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: CSL will consider on a case-by-case basis requests to share Individual Patient Data (IPD) with external bona-fide, qualified scientific and medical researchers. For information on the process and requirements for submitting a voluntary data sharing request for IPD, please contact CSL at clinicaltrials@cslbehring.com.
Study at a glance
From the registry record, last updated by the sponsor on 16 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT07326592
- Acronym / sponsor ID
- CE1226_4003
- Keywords
- Progressive Emphysema, Chronic Pulmonary Disease
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 270 participants (estimated)
- Lead sponsor
- CSL BehringIndustry (pharma, biotech, devices)
- Study start
- 29 Sep 2026 (actual)
- Primary completion
- 15 Sep 2033 (estimated)
- Study completion
- 15 Sep 2033 (estimated)
- First posted
- 8 Jan 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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).
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 3
- Model description
- This is a double blind, parallel group assignment study.
| Arm | Type | Receives |
|---|---|---|
| CE1226 low dose | Active comparator | Biological: CE1226CE1226 at low dose. |
| CE1226 medium dose | Experimental | Biological: CE1226CE1226 at medium dose. |
| CE1226 high dose | Experimental | Biological: CE1226CE1226 at high dose. |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (2 listed)
- 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).
Key exclusion criteria (3 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| CE1226Also: Human alpha1-proteinase inhibitor, Respreeza, ZemairaCE1226 will be administered via intravenous (IV) infusion weekly over 3 years. | Biological | CE1226 high dose, CE1226 low dose, CE1226 medium dose |
Primary outcome
Annual rate of change in adjusted lung density
Time frame: From Baseline to Month 36
The annual rate of change (expressed as gram per liter per year [g/L/year]) in adjusted lung density (15th percentile lung density [PD15]) will be calculated on CT scan assessments of whole lung at total lung capacity (TLC).
6 secondary outcomes
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
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Oct 2026.
- Individual participant dataSharing planned: Yes. CSL will consider on a case-by-case basis requests to share Individual Patient Data (IPD) with external bona-fide, qualified scientific and medical researchers. For information on the process and requirements for submitting a voluntary data sharing request for IPD, please contact CSL at clinicaltrials@cslbehring.com.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Study Director study director
CSL Behring
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing CE1226 in people with Alpha1 Antitrypsin Deficiency and Alpha1-Proteinase Inhibitor Deficiency, run by CSL Behring at 2 sites in Denmark.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 16 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in Denmark. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Trial Registration Coordinator (+1 610-878-4697, clinicaltrials@cslbehring.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Annual rate of change in adjusted lung density (time frame: From Baseline to Month 36). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 16 Oct 2026.