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)

NCT07326592 | Not Yet Recruiting Phase 4 DMC FDA Drug

• 2 other identifiers: CE1226_40032025-522964-33-00
• Study Type: interventional
• Target: 270
• Locations: 0 countries
• Sites: N/A

Brief 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 marketed dose 60 milligrams per kilogram (mg/kg).

Conditions

Alpha1 Antitrypsin Deficiency; Alpha1-Proteinase Inhibitor Deficiency; Emphysema

Keywords

Progressive Emphysema; Chronic Pulmonary Disease

Outcome Measures

Primary Outcomes (1)

• 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).

  From Baseline to Month 36

Secondary Outcomes (6)

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

  From Baseline to Month 36

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

  From Baseline to Month 36

• Number of severe pulmonary exacerbations

  From screening up to Month 36

• Duration of severe pulmonary exacerbations

  From screening up to Month 36

• Number of participants experiencing treatment emergent adverse events (TEAEs)

  From screening up to Month 36

Study Arms (3)

CE1226 low dose

ACTIVE COMPARATOR

CE1226 at low dose.

Biological: CE1226

CE1226 medium dose

EXPERIMENTAL

CE1226 at medium dose.

Biological: CE1226

CE1226 high dose

EXPERIMENTAL

CE1226 at high dose.

Biological: CE1226

Interventions

CE1226 BIOLOGICAL

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

Also known as: Human alpha1-proteinase inhibitor, Respreeza, Zemaira

CE1226 high dose; CE1226 low dose; CE1226 medium dose

Eligibility Criteria

• Age: 18 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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).

You may not qualify if:

• 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.

Sponsors & Collaborators

• CSL Behring: lead

MeSH Terms

Conditions

alpha 1-Antitrypsin Deficiency; Emphysema

Interventions

alpha 1-Antitrypsin; Respreeza

Condition Hierarchy (Ancestors)

Liver Diseases; Digestive System Diseases; Lung Diseases; Respiratory Tract Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Subcutaneous Emphysema; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

Glycoproteins; Glycoconjugates; Carbohydrates; Serpins; Peptides; Amino Acids, Peptides, and Proteins; Acute-Phase Proteins; Blood Proteins; Proteins; Alpha-Globulins; Serum Globulins; Globulins

Study Officials

• Study Director

  CSL Behring

  STUDY DIRECTOR

Central Study Contacts

Trial Registration Coordinator

CONTACT

+1 610-878-4697; clinicaltrials@cslbehring.com

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Masking Details: Sponsor will also be blinded to treatment allocation.
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: This is a double blind, parallel group assignment study.

Study Record Dates

• First Submitted: January 7, 2026
• First Posted: January 8, 2026
• Study Start: April 15, 2026
• Primary Completion (Estimated): September 15, 2033
• Study Completion (Estimated): September 15, 2033
• Last Updated: March 6, 2026

Record last verified: 2026-03

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP
• Time Frame: Requests for IPD will generally be considered once review by major regulatory authorities (i.e. FDA, EMA) is complete and the primary publication is available.
• Access Criteria: Proposed research should seek to answer a previously unanswered important medical or scientific question. Applicable country specific privacy and other laws and regulations will be considered and may prevent sharing of IPD. If the request is approved and the researcher has executed an appropriate data sharing agreement, IPD that has been appropriately anonymized will be available.