A Phase 2, Double-blind, Randomized, Active-control, Parallel Group Study to Assess the Pharmacokinetics, Pharmacodynamics, Immunogenicity, and safety of SAR447537 (INBRX-101) Compared to Plasma Derived Apha1-Proteinase Inhibitor (A1PI) Augmentation Therapy in Adults with Alpha-1 Antitrypsin Deficiency (AATD) Emphysema
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 34
- 试验地点
- 65
- 主要终点
- Mean change in average fAAT concentration as measured by anti-neutrophil elastase capacity [ANEC] from baseline to average serum trough fAAT concentration at steady state (Ctrough,ss) in participants treated with SAR447537 compared to A1PI
研究概览
简要总结
To evaluate the pharmacodynamic effect on serum trough functional AAT (fAAT) levels at steady-state, following a treatment period of up to 32-weeks, in participants with AATD emphysema treated with SAR447537 compared to A1PI
详细描述
This is a Phase 2, Double-Blind, Randomized, Active-Control, Parallel Group Study to Assess the Pharmacokinetics, Pharmacodynamics, Immunogenicity, and Safety of SAR447537 (INBRX-101) Compared to Plasma-Derived Alpha1-Proteinase Inhibitor (A1PI) Augmentation Therapy in Adults With Alpha-1 Antitrypsin Deficiency (AATD) Emphysema.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 主要目的
- Screening
- 盲法
- Double (Investigator, Monitor, Carer, Analyst, Subject)
入排标准
- 年龄范围
- 18 years 至 65+ years（65+ Years, 18-64 Years）
- 接受健康志愿者
- 是
入选标准
- •Males or females 18-80 years of age, inclusive, at the time of screening
- •Diagnosis of AATD
- •Evidence of emphysema secondary to AATD
- •FEV1 of ≥ 30% and ≤ 80% predicted at screening
- •Current non-smoking status.
排除标准
- •Receipt of A1PI augmentation therapy within 5 weeks prior to the first dose of study drug
- •Known or suspected allergy to components of SAR447537, A1PI or human IgG
- •Known selective or severe Immunoglobulin A (IgA) deficiency
- •Known or suspected diagnosis of type 1 diabetes or diagnosed with uncontrolled type 2 diabetes
- •Received IV immunoglobulins, monoclonal antibodies and/or other biologic therapies within 30 days
- •On waiting list for lung or liver transplant
- •Acute respiratory tract infection or COPD exacerbation within 4 weeks prior to or during screening
- •Evidence of decompensated cirrhosis
- •Active cancers or has a history of malignancy within 5 years prior to screening
- •History of unstable cor pulmonale
- •Clinically significant congestive heart failure
结局指标
主要结局
Mean change in average fAAT concentration as measured by anti-neutrophil elastase capacity [ANEC] from baseline to average serum trough fAAT concentration at steady state (Ctrough,ss) in participants treated with SAR447537 compared to A1PI
Mean change in average fAAT concentration as measured by anti-neutrophil elastase capacity [ANEC] from baseline to average serum trough fAAT concentration at steady state (Ctrough,ss) in participants treated with SAR447537 compared to A1PI
次要结局
- 1. Mean change in serum fAAT concentration from baseline to fAAT average concentration at steady state (Cavg,ss) in participants treated with INBRX-101 compared to A1PI 2. Percentage of days with fAAT above the lower limit of the normal range during steady-state dosing in participants treated with INBRX-101 compared to A1PI
- 3. Incidence of all treatment emergent adverse events (TEAEs), TEAEs ≥ Grade 3, serious adverse events (SAEs), TEAEs leading to IMP discontinuation, adverse events of special interest (AESI) (including infusion-related reactions)
- 4. Frequency of anti-drug antibodies (ADA) against SAR447537 and endogenous AAT, as well as neutralizing ADA (NAb) against SAR447537 and endogenous AAT 5. Population PK modeling to assess impact of physiologically relevant patient participants characteristics (eg, covariates including, but not limited to, age, sex, body size, ethnicity) and disease on PK
研究者
Clinical Sciences and Operations
Scientific
Sanofi AATD Inc.