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
试验速览
- 阶段
- 4 期
- 状态
- 已完成
- 发起方
- CSL Behring
- 入组人数
- 140
- 试验地点
- 22
- 主要终点
- Rate of Change of Adjusted Lung Density
研究概览
简要总结
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).
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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
排除标准
- •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
结局指标
主要结局
Rate of Change of Adjusted Lung Density
时间窗: Up to 2 years
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.
次要结局
- Percent Change in Lung Function as Measured by Percent Predicted FEV1(From baseline up to 2 years)
- Percent Change in Adjusted Lung Density(From baseline to 2 years)
- Change in Subject-reported Symptoms(From baseline to 2 years)
- Absolute Change in Adjusted Lung Density(From baseline to 2 years)
- Percent Change in Lung Function as Measured by Forced Expiratory Volume in 1 Second (FEV1)(From baseline up to 2 years)
- Number of Subjects With Pulmonary Exacerbations(Up to 2 years)
- Percent Change in Lung Function as Measured by Ratio of FEV1/FVC (Forced Vital Capacity)(From baseline up to 2 years)
- Annual Rate in Subject Years of Pulmonary Exacerbations(Up to 2 years)
- Time to First Pulmonary Exacerbation(Up to 2 years)
- Percentage of Subjects With Treatment Emergent Adverse Events(From baseline up to 2.5 years)