Clinical research study to assess the safety and effectiveness of alpha1-proteinase inhibitor (A1-PI) administered by intravenous application to patients with alpha1-proteinase deficiency and emphysema who have completed the 2-year treatment and observation period in the Phase III/IV Zemaira® CE1226_4001.

An open-label, non-controlled, multicenter, multinational Study to evaluate the Efficacy and Safety of Zemaira® administration in Chronic Augmentation and Maintenance Therapy in Subjects with Emphysema due to Alpha1-Proteinase Inhibitor Deficiency who completed Clinical Study CE1226_4001

Status

Active, not recruiting

Phases

Unknown

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2007-007129-38-IE

Enrollment

140

Registered

2008-01-11

Start date

2008-03-14

Completion date

Unknown

Last updated

2015-09-21

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Chronic augmentation and maintenance therapy in individuals with alpha1-proteinase inhibitor (A1-PI) deficiency and clinical evidence of emphysema. MedDRA version: 17.0 Level: LLT Classification code 10001811 Term: Alpha-1 proteinase inhibitor deficiency System Organ Class: 100000004850

Interventions

Product Name: Alpha1-Proteinase Inhibitor / Zemaira® Product Code: CE1226 Pharmaceutical Form: Powder and solvent for solution for injection INN or Proposed INN: There is no recommended INN Current Sp

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: 1. Subjects who have completed the 2-year treatment and observation period in the Phase 3/4 Zemaira® CE1226_4001 study and are willing to sign informed consent. 2. Males, and non-pregnant, non-lactating females, whose screening pregnancy test is negative and who are using contraceptive methods deemed reliable by the investigator. Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 125 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 15

Exclusion criteria

Exclusion criteria: 1. Individuals residing in the US. 2. Current evidence of alcohol abuse, or abuse of drugs, such as barbiturates, benzodiazepines, amphetamines, cocaine, opioids, and cannabinoids. 3. History of allergy, anaphylactic reaction, or severe systemic response to human plasma derived products, or known mannitol hypersensitivity, or history of prior adverse reaction (AR) to mannitol. 4. Current tobacco smoker (i.e. smoking must be discontinued for at least 6 months prior to study participation). 5. Conditions or behaviors that interfere with attending scheduled study visits, in the opinion of the investigator. 6. History of non-compliance. 7. Administration of any other experimental new drug or participation in an investigation of a marketed product. 8. Inability to perform necessary study procedures.

Design outcomes

Primary

Measure	Time frame
Main Objective: To investigate the effect of Zemaira® on the progression of emphysema, assessed by the decline of lung density, measured by yearly computed tomography (CT).;Secondary Objective: The secondary objectives are to assess the effect of treatment with A1-PI on: 1. change in symptoms score as assessed by Quality of Life (QoL) using the St. George's Respiratory Questionnaire (SGRQ); 2. the assessment of pulmonary function (ie, forced expiratory volume in 1 second [FEV1] and [FEV1/forced vital capacity{FVC}]); 3. reduction in risk of pulmonary exacerbations assessed by the number and annual rate of exacerbations over the study duration; 4. antigenic and functional serum A1-PI levels in relation to disease progression, body mass index (BMI), safety, and mortality. 5. the safety profile of subjects will be assessed throughout the study. The safety of Zemaira® will be assessed by the occurrence of adverse events (AEs).;Primary end point(s): Primary Efficacy Variable: Annual Rate of Change in adjusted P15 from CT Scans The exploratory efficacy variable of primary interest is the annual rate of change, measured by a slope of time and treatment interaction from a mixed effects model, in volume-adjusted lung density. This is assessed by the 15th percentile (P15) of the lung density distribution. The variable of interest is called adjusted P15 measured in g/L. Scans are obtained at total lung capacity (TLC) and functional residual capacity (FRC) inspiration state, and lung volume is assessed for the right, the left, and for both lungs. During the extension study, the CT scans are taken at Month 12 and Month 24 (ie, Month 36 [Visit 5] and Month 48 [Visit 9] when consideration is given to the start of study CE1226_4001). The primary interest of the study will be comparing the annual rate of change in adjusted P15 from CT scans between subjects who had an early start with Zemaira® treatment and subjects who had a late start with treatment over 4 years.;Timepoint(	—

Measure

Time frame

Main Objective: To investigate the effect of Zemaira® on the progression of emphysema, assessed by the decline of lung density, measured by yearly computed tomography (CT).;Secondary Objective: The secondary objectives are to assess the effect of treatment with A1-PI on: 1. change in symptoms score as assessed by Quality of Life (QoL) using the St. George's Respiratory Questionnaire (SGRQ); 2. the assessment of pulmonary function (ie, forced expiratory volume in 1 second [FEV1] and [FEV1/forced vital capacity{FVC}]); 3. reduction in risk of pulmonary exacerbations assessed by the number and annual rate of exacerbations over the study duration; 4. antigenic and functional serum A1-PI levels in relation to disease progression, body mass index (BMI), safety, and mortality. 5. the safety profile of subjects will be assessed throughout the study. The safety of Zemaira® will be assessed by the occurrence of adverse events (AEs).;Primary end point(s): Primary Efficacy Variable: Annual Rate of Change in adjusted P15 from CT Scans The exploratory efficacy variable of primary interest is the annual rate of change, measured by a slope of time and treatment interaction from a mixed effects model, in volume-adjusted lung density. This is assessed by the 15th percentile (P15) of the lung density distribution. The variable of interest is called adjusted P15 measured in g/L. Scans are obtained at total lung capacity (TLC) and functional residual capacity (FRC) inspiration state, and lung volume is assessed for the right, the left, and for both lungs. During the extension study, the CT scans are taken at Month 12 and Month 24 (ie, Month 36 [Visit 5] and Month 48 [Visit 9] when consideration is given to the start of study CE1226_4001). The primary interest of the study will be comparing the annual rate of change in adjusted P15 from CT scans between subjects who had an early start with Zemaira® treatment and subjects who had a late start with treatment over 4 years.;Timepoint(

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Secondary

Measure	Time frame
Secondary end point(s): Key Secondary Efficacy Variable: Absolute and % Change in Adjusted P15 from Baseline to Month 48 The absolute and % change from baseline to the Month 48 will be derived using the values of adjusted P15 of the last assessment of CT scans from study CE1226_3001 and the first assessment of CT scans from study CE1226_4001.;Timepoint(s) of evaluation of this end point: See E.5.2	—

Measure

Time frame

Secondary end point(s): Key Secondary Efficacy Variable: Absolute and % Change in Adjusted P15 from Baseline to Month 48 The absolute and % change from baseline to the Month 48 will be derived using the values of adjusted P15 of the last assessment of CT scans from study CE1226_3001 and the first assessment of CT scans from study CE1226_4001.;Timepoint(s) of evaluation of this end point: See E.5.2

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Countries

Australia, Canada, Czech Republic, Denmark, Estonia, Finland, Germany, Ireland, Poland, Romania, Sweden

Contacts

Public ContactGlobal Project Lead

CSL Behring GmbH

anja.braeunlich@cslbehring.com+49 6421 39-2929

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026