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)

CSL Behring

Status and phase: Not yet enrolling; Phase 4

Conditions: Alpha1-Proteinase Inhibitor Deficiency; Alpha1 Antitrypsin Deficiency; Emphysema

Treatments: Biological: CE1226

Study type: Interventional

Funder types: Industry

Identifiers

NCT07326592

2025-522964-33-00 (EU Trial (CTIS) Number)

CE1226_4003

Details and patient eligibility

About

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).

Enrollment: 270 estimated patients

Sex: All

Ages: 18 to 65 years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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270 participants in 3 patient groups

CE1226 low dose

Active Comparator group

Description:

CE1226 at low dose.

Treatment:

Biological: CE1226

CE1226 medium dose

Experimental group

Description:

CE1226 at medium dose.

Treatment:

Biological: CE1226

CE1226 high dose

Experimental group

Description:

CE1226 at high dose.

Treatment:

Biological: CE1226