NCT01983241进行中（未招募）3 期
A Randomized, Double-Blind, Placebo Controlled Study to Assess the Efficacy and Safety of Two Dose Regimens (60 mg/kg and 120 mg/kg) of Weekly Intravenous Alpha1 Proteinase Inhibitor (Human) in Subjects With Pulmonary Emphysema Due to Alpha1 Antitrypsin Deficiency
适应症
干预措施
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 进行中（未招募）
- 入组人数
- 345
- 试验地点
- 52
- 主要终点
- Change from Baseline in Whole lung PD15 (15th percentile point)
研究概览
简要总结
This is a multi-center, randomized, placebo-controlled, double blind clinical study to assess the efficacy and safety of two separate dose regimens of Alpha-1 MP versus placebo for 156 weeks (i.e., 3 years) using computed tomography (CT) of the lungs as the main measure of efficacy. The two Alpha-1 MP doses to be tested are 60 mg/kg and 120 mg/kg administered weekly by IV infusion for 156 weeks. The study consists of an optional pre-screening phase, Screening Phase, a 156-week Treatment Phase, and an End of Study Visit at Week 160.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 18 Years 至 70 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Have a documented total alpha1-PI serum level < 11 µM.
- •Have a diagnosis of congenital AATD with an allelic combination of ZZ, SZ, Z(null), (null)(null), S(null), or "at-risk" alleles.
- •At the Screening (Week -3) Visit, have a post-bronchodilator forced expiratory volume in 1 second (FEV1) ≥ 30% and < 80% of predicted and FEV1/forced vital capacity (FVC) < 70% (Global Initiative for Chronic Obstructive Lung Disease [GOLD] stage II or III).
- •Have a carbon monoxide diffusing capacity (DLCO) ≤ 60% of predicted (corrected for HgB) within the past 2 years OR evidence of pulmonary emphysema on CT scan within the past 2 years per the Investigator's judgment.
- •Have clinical evidence of pulmonary emphysema per the Investigator's judgment.
排除标准
- •Has received alpha1-PI augmentation therapy for more than 1 month within the six months prior to the Screening Visit.
- •Has received alpha1-PI augmentation therapy within one month of the Screening Visit.
- •Has had a chronic obstructive pulmonary disease (COPD) exacerbation within the 5 weeks prior to the Screening Visit or during the Screening Phase.
- •Unable to physically (e.g., unable to fit inside the CT scanner) or mentally (e.g., claustrophobic) undergo a CT scan.
- •History of lung or liver transplant.
- •Any lung surgery during the past 2 years (excluding lung biopsy).
- •On the waiting list for lung surgery, including lung transplant.
- •Smoking during the past 12 months or a positive urine cotinine test at screening that is due to smoking. Maybe on Nicotine replacement, including vapor cigarettes.
- •History of anaphylaxis or severe systemic response to any plasma-derived alpha1-PI preparation or other blood product(s).
- •Use of systemic steroids above a stable dose equivalent to 5 mg/day prednisone (i.e., 10 mg every 2 days) within the 5 weeks prior to the Screening Visit (inhaled steroids are not considered systemic steroids) or during the Screening Phase.
- •Use of systemic or aerosolized antibiotics for a COPD exacerbation within the 5 weeks prior to the Screening Visit or during the Screening Phase.
- •Known selective or severe Immunoglobulin A (IgA) deficiency.
研究组 & 干预措施
Alpha-1 MP 120 mg/kg
Experimental
Alpha-1 MP 120 mg/kg administered weekly by IV infusion for 156 weeks
干预措施: Alpha-1 MP (Biological)
Alpha-1 MP 60 mg/kg
Experimental
Alpha-1 MP 60 mg/kg administered weekly by IV infusion for 156 weeks
干预措施: Alpha-1 MP (Biological)
Placebo
Placebo Comparator
0.9% Sodium Chloride for Injection, USP, administered weekly by IV infusion for 156 weeks
干预措施: 0.9% Sodium Chloride for Injection, USP (Other)
结局指标
主要结局
Change from Baseline in Whole lung PD15 (15th percentile point)
时间窗: Week -3 (baseline measure), Week 52, Week 104, Week 130, Week 156
Whole lung PD15 measured by CT scan
次要结局
- Discontinuations from the study due to AEs(Week -3 through Week 160)
- Changes from baseline in forced expiratory volume in 1 second (FEV1)(Weeks 26, 52, 78, 104, 130 and 156)
- Severe COPD Exacerbations(Week -3 through Week 160)
- Change from Baseline in PD15 of the basal lung region(Week -3 (baseline measure), Week 52, Week 104, Week 130, Week 156)
- Change from baseline in carbon monoxide diffusing capacity (DLco)(Weeks 26, 52, 78, 104, 130 and 156)
- Adverse Events (AEs)(Week -3 through Week 160)
- Serious Adverse Events (SAEs)(Week -3 through Week 160)
- Change from baseline in Saint George's Respiratory Questionnaire: Minimum value = 0, maximum value = 100, higher scores indicate worse condition(Weeks 26, 52, 78, 104, 130 and 156)
- Change from baseline in the EuroQoL (Quality of Life)- 5 Dimension- 5 Level (EQ-5D-5L): Minimum value (for each one of the 5 levels) = 1, maximum value (for each one of the 5 levels) = 5, higher scores indicate worse condition(Weeks 26, 52, 78, 104, 130 and 156)
研究者
研究点 (52)
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