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NCT Number: NCT07326592

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)

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).

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Key information

Conditions

Alpha1 Antitrypsin Deficiency Alpha1-Proteinase Inhibitor Deficiency Congenital, Hereditary, and Neonatal Diseases and Abnormalities Digestive System Diseases Emphysema Genetic Diseases, Inborn Liver Diseases Lung Diseases Pathologic Processes Pathological Conditions, Signs and Symptoms Respiratory Tract Diseases Subcutaneous Emphysema alpha 1-Antitrypsin Deficiency

Age range

18 year–65 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 4

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

CE1226

Biological

CE1226 will be administered via intravenous (IV) infusion weekly over 3 years.

Other names: Human alpha1-proteinase inhibitor, Respreeza, Zemaira

Primary outcomes

  1. 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).

Secondary outcomes

  1. Annual rate of change in forced expiratory volume in 1 second percent predicted (FEV1%)

    Time frame: From Baseline to Month 36

    The annual rate of change in FEV1% will be assessed by spirometry.

  2. Annual rate of change in diffusion capacity of carbon monoxide (DLco)

    Time frame: From Baseline to Month 36

    The annual rate of change in DLco will be assessed by gas transfer using site machinery.

  3. Number of severe pulmonary exacerbations

    Time frame: From screening up to Month 36

    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.

  4. Duration of severe pulmonary exacerbations

    Time frame: From screening up to Month 36

    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.

  5. Number of participants experiencing treatment emergent adverse events (TEAEs)

    Time frame: From screening up to Month 36

  6. Percentage of participants experiencing TEAEs

    Time frame: From screening up to Month 36

Study contacts

Contact information is provided by the study sponsor or research team.

Trial Registration Coordinator

CONTACT

[email protected]

+1 610-878-4697

Sponsors and collaborators

Lead sponsor

CSL Behring

Industry

Registry information

Official study 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

Important dates

Study start
2026
Primary completion
2033
Study completion
2033
First posted
Jan 8, 2026
Registry last updated
May 15, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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