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)

NCT ID: NCT07326592

Last Updated: 2026-01-08

Basic Information

• Recruitment Status: NOT_YET_RECRUITING
• Clinical Phase: PHASE4
• Total Enrollment: 270 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2026-01-15
• Study Completion Date: 2033-09-15

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

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

CE1226 low dose

CE1226 at low dose.

Group Type: ACTIVE_COMPARATOR

CE1226

Intervention Type: BIOLOGICAL

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

CE1226 medium dose

CE1226 at medium dose.

Group Type: EXPERIMENTAL

CE1226

Intervention Type: BIOLOGICAL

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

CE1226 high dose

CE1226 at high dose.

Group Type: EXPERIMENTAL

CE1226

Intervention Type: BIOLOGICAL

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

Interventions

CE1226

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

Intervention Type: BIOLOGICAL

Other Intervention Names

Human alpha1-proteinase inhibitor; Respreeza; Zemaira

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.

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 65 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

CSL Behring

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Study Director

Role: STUDY_DIRECTOR

CSL Behring

Central Contacts

Trial Registration Coordinator

Role: CONTACT

clinicaltrials@cslbehring.com

+1 610-878-4697

Other Identifiers

2025-522964-33-00

Identifier Type: CTIS

Identifier Source: secondary_id

CE1226_4003

Identifier Type: -

Identifier Source: org_study_id