Extension Study of Zemaira® i.v. Administration in Subjects With Emphysema Due to alpha1-proteinase Inhibitor Deficiency.

CSL Behring140 enrolled

Overview

This study is a continuation of the placebo-controlled study CE1226\_4001 (NCT00261833) to evaluate the efficacy and safety of Zemaira® intravenous (i.v). administration in subjects with emphysema due to alpha1-proteinase inhibitor deficiency. The long-term verification of a disease-modifying benefit of Zemaira® on the progression of emphysema will be assessed by volume-adjusted lung density, measured yearly by computed tomography (CT).

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

140

Conditions

Emphysema Alpha 1-proteinase Inhibitor Deficiency

Interventions

Alpha1- proteinase inhibitor [human]BIOLOGICAL

Lyophilized preparation of 60 mg/kg body weight intravenously once per week

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

Inclusion Criteria: * Subjects who have completed the 2-year treatment and observation period in the Phase 3/4 Zemaira® CE1226\_4001 study (NCT00261833) and are willing to sign informed consent * Males, and non-pregnant, non-lactating females, whose screening pregnancy test is negative and who are using contraceptive methods deemed reliable by the investigator Exclusion Criteria: * Individuals residing in the US * Current evidence of alcohol abuse or abuse of drugs such as barbiturates, benzodiazepines, amphetamines, cocaine, opioids, and cannabinoids * History of allergy, anaphylactic reaction, or severe systemic response to human plasma derived products, or known mannitol hypersensitivity, or history of prior adverse reaction to mannitol * Current tobacco smoker (smoking must be discontinued for at least 6 months prior to study participation) * Conditions or behaviors that interfere with attending scheduled study visits in the opinion of the investigator * History of non-compliance * Administration of any other experimental new drug or participation in an investigation of a marketed product * Inability to perform necessary study procedures

Locations (21)

Study Site

Adelaide, South Australia, Australia

Study Site

Fitzroy, Victoria, Australia

Outcomes

Primary Outcomes

Rate of Change of Adjusted Lung Density

As measured by centralized, standardized computer tomographic (CT) lung densitometry. CT scans were acquired at 2 inspiration states: TLC (Total Lung Capacity; ie, full inspiration) and FRC (Functional Residual Capacity; ie, full expiration). Results were adjusted for total lung volume and are presented as point estimates for the average rate of decline in the early start and delayed start subgroups from a linear random regression model with country, inspiration state (only for 'TLC and FRC state'), time (time elapsed since Day 1 \[CE1226\_4001\]), treatment and treatment by time interaction as fixed effects and subject and subject by time interaction as random coefficients.

Time frame: Up to 2 years

Secondary Outcomes

Absolute Change in Adjusted Lung Density

Absolute change from baseline to 2 years as measured by centralized, standardized CT lung densitometry. CT scans were acquired at 2 inspiration states: TLC (ie, full inspiration) and FRC (ie, full expiration). Results were adjusted for total lung volume and are presented as point estimates for the average absolute change in the early start and delayed start subgroups from an analysis of covariance (ANCOVA) model with country, treatment, and baseline lung density as fixed effects and inspiration state as a repeated random effect. The baseline is the last assessment from the preceding study CE1226\_4001.

Time frame: From baseline to 2 years

Percent Change in Adjusted Lung Density

Percent change from baseline to 2 years as measured by centralized, standardized CT lung densitometry. CT scans were acquired at 2 inspiration states: TLC (ie, full inspiration) and FRC (ie, full expiration). Results were adjusted for total lung volume and are presented as point estimates for the average percent change in the early start and delayed start subgroups from an analysis of covariance (ANCOVA) model with country, treatment, and baseline lung density as fixed effects and inspiration state as a repeated random effect. The baseline is the last assessment from the preceding study CE1226\_4001.

Time frame: From baseline to 2 years

Data from ClinicalTrials.gov

This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.