A Phase 2, Single-Arm, Open-Label Extension Study, Evaluating the Long-Term Safety and Clinical Efficacy of SAR447537 (INBRX-101) in Adults with Alpha-1 Antitrypsin Deficiency (AATD) Emphysema
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 发起方
- 入组人数
- 36
- 试验地点
- 9
- 主要终点
- Incidences of all treatment-emergent adverse events (TEAEs), TEAEs ≥Grade 3, serious adverse events (SAEs), TEAEs requiring withdrawal from investigational product (IP) treatment leading to discontinuation from SAR447537, and adverse events of special interest (AESIs) (including infusion-related reactions) leading to discontinuation from and adverse events of special interest (AESIs) (including infusion-related reactions).
研究概览
简要总结
To evaluate the long-term safety and tolerability of SAR447537.
研究设计
- 分配方式
- Not Applicable
- 主要目的
- Open Label Extention Study
- 盲法
- None
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% predicted at screening and post-bronchodilator FEV1/FVC<0.
- •Current non-smoking status.
排除标准
- •For Newly Identified Participants: 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.
- •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.
- •Uncontrolled diabetes mellitus despite adequate antidiabetic pharmacologic treatment with a screening HbA1c value ≥9%
结局指标
主要结局
Incidences of all treatment-emergent adverse events (TEAEs), TEAEs ≥Grade 3, serious adverse events (SAEs), TEAEs requiring withdrawal from investigational product (IP) treatment leading to discontinuation from SAR447537, and adverse events of special interest (AESIs) (including infusion-related reactions) leading to discontinuation from and adverse events of special interest (AESIs) (including infusion-related reactions).
Incidences of all treatment-emergent adverse events (TEAEs), TEAEs ≥Grade 3, serious adverse events (SAEs), TEAEs requiring withdrawal from investigational product (IP) treatment leading to discontinuation from SAR447537, and adverse events of special interest (AESIs) (including infusion-related reactions) leading to discontinuation from and adverse events of special interest (AESIs) (including infusion-related reactions).
次要结局
- Annual rate of change in lung density assessed by serial quantitative CT (15th percentile point [PD15]) at total lung capacity (TLC), centrally read, from Baseline to End of Treatment (EoT).
- Serum PK profile of SAR447537 and fAAT.
- Population PK modeling to assess impact of physiologically relevant participant characteristics (eg, covariates including, but not limited to, age, sex, body size, ethnicity) and disease on PK of SAR447537 through Population PK modeling.
- Incidence of anti-drug antibodies (ADAs) against SAR447537 as well as neutralizing anti-drug antibodies (NAbs) against SAR447537.
研究者
Clinical Sciences and Operations
Scientific
Sanofi AATD Inc.