2024-513962-20-00进行中（未招募）3 期
An Open-Label, Multicenter Study to Evaluate the Long-term Safety of Weekly Intravenous Alphal-Proteinase Inhibitor (Human), Modified Process 60 mg/kg in Subjects With Pulmonary Emphysema Due to Alpha1-Antitrypsin Deficiency
试验速览
- 阶段
- 3 期
- 状态
- 进行中（未招募）
- 入组人数
- 120
- 试验地点
- 8
- 主要终点
- The efficacy variables that will be assessed are: •Whole lung 15th percentile point (PD15) using computed tomography (CT) densitometry •Carbon monoxide diffusing capacity (DLCO) •forced expiratory volume in 1 second (FEV1) •St. George’s Respiratory Questionnaire (SGRQ) score •Five level EQ-5D (EQ-5D-5L) score •Incidence and severity of chronic obstructive pulmonary disease (COPD) exacerbations as defined by American Thoracic Society (ATS)/European Respiratory Society (ERS) criteria
研究概览
简要总结
The primary objective of this study is to obtain an additional 2 years of safety data for intravenously administered Alpha-1 MP 60 mg/kg/week in subjects with AATD.
入排标准
- 年龄范围
- 18 years 至 65+ years（18-64 Years, 65+ Years）
- 接受健康志愿者
- 是
入选标准
- •a. Has completed participation in Study GTi1201 (ie, completed Week 156 and Week 160/End-of-Study Visit) Or b. Has experienced a decline in FEV1 at the annualized rate of ≥134.4 mL/year at or after the Week 104 Visit in GTi
- •Is willing and able to provide informed consent.
排除标准
- •Is unable to physically or mentally undergo a CT scan (eg, unable to fit inside the CT scanner, claustrophobic).
- •Has a history of anaphylaxis or severe systemic response to any plasma-derived alpha1-PI preparation or other blood product(s).
- •In the opinion of the investigator, is likely to have compliance problems with the protocol and the procedures of the protocol.
- •Has any medical condition that the investigator feels might confound the results of the study or pose an additional risk to the subject during study participation.
- •Has severe concomitant disease including, but not limited to, congestive heart failure and liver cirrhosis.
- •Has primary and/or secondary (metastatic disease) pulmonary malignancy or other current malignancy with <1 year predicted overall survival
- •Has a metal object (newly received since starting GTi1201) that might interfere with chest CT quality and quantification. Metal objects include, but are not limited to, cardiac pacemaker, defibrillator, metal prosthetic heart valve, metal projectile, metal weapon fragments, or metal shoulder prosthesis.
- •Is a female who is pregnant, breastfeeding or, if of child-bearing potential, unwilling to practice a highly effective method of contraception (oral, injectable, or implanted hormonal methods of contraception; placement of an intrauterine device or intrauterine system; condom or occlusive cap with spermicidal foam/gel/film/cream/suppository; male sterilization; or abstinence) throughout the study.
- •Has clinical signs and symptoms of viral infection requiring virus safety testing at the Week 160/End-of-Study Visit or Early Discontinuation Visit in Study GTi1201, and the virus safety test results are indicative of acute or chronic infection with hepatitis A virus (HAV), hepatitis B virus (HBV), hepatitis C virus (HCV), human immunodeficiency virus (HIV), or parvovirus B19 (B19V). Note: If the virus safety test results are indicative of acute or chronic infection with HAV, HBV, HCV, HIV, or B19V, the subject will be considered a screen failure and must be withdrawn from the study.
- •Has current evidence of smoking, which includes electronic/vapor cigarettes, or has a positive urine cotinine test at the Week 160/End-of-Study Visit in Study GTi1201 that is due to smoking.
- •Has current evidence of chronic alcoholism or illicit drug abuse (addiction).
- •Is currently participating in another investigational product (IP) study.
结局指标
主要结局
The efficacy variables that will be assessed are: •Whole lung 15th percentile point (PD15) using computed tomography (CT) densitometry •Carbon monoxide diffusing capacity (DLCO) •forced expiratory volume in 1 second (FEV1) •St. George’s Respiratory Questionnaire (SGRQ) score •Five level EQ-5D (EQ-5D-5L) score •Incidence and severity of chronic obstructive pulmonary disease (COPD) exacerbations as defined by American Thoracic Society (ATS)/European Respiratory Society (ERS) criteria
The efficacy variables that will be assessed are: •Whole lung 15th percentile point (PD15) using computed tomography (CT) densitometry •Carbon monoxide diffusing capacity (DLCO) •forced expiratory volume in 1 second (FEV1) •St. George’s Respiratory Questionnaire (SGRQ) score •Five level EQ-5D (EQ-5D-5L) score •Incidence and severity of chronic obstructive pulmonary disease (COPD) exacerbations as defined by American Thoracic Society (ATS)/European Respiratory Society (ERS) criteria
次要结局
- The following safety variables will be assessed in this study: •AEs, serious AEs (SAEs), and discontinuations due to AEs and SAEs •Physical examination (complete and respiratory) •Clinical laboratory parameters: hematology, chemistry (includes high-sensitivity C-reactive protein [hs-CRP])
研究者
SCIENTIFIC AND INNOVATION OFFICE DRUG DEVELOPMENT
Scientific
Grifols Therapeutics LLC
研究点 (8)
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