A 52-week, Phase 1 Safety, Pharmacokinetic and Pharmacodynamic Trial of Siplizumab in Newly Diagnosed Adult Amyotrophic Lateral Sclerosis (ALS) Patients (AURORA)
试验速览
- 阶段
- 1 期
- 状态
- 终止
- 发起方
- ITB-Med LLC
- 入组人数
- 7
- 试验地点
- 3
- 主要终点
- Evaluation of the safety of TCD601 in adult patients with ALS
研究概览
简要总结
The purpose of this study is to investigate the safety, pharmacokinetics (PK), and pharmacodynamics (PD) of TCD601 (siplizumab) in newly diagnosed adult ALS patients.
详细描述
The purpose of this study is to investigate how safe and tolerable the study drug siplizumab is when given to adult patients with ALS. The study will examine how the drug is distributed in the body and what effects it has on different immune cells in the blood and in the fluid surrounding the motor neurons (cerebrospinal fluid). In addition, the study will monitor the progression of ALS and look for any changes related to the disease that may occur during the study.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Basic Science
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 80 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male or female patients ≥ 18 to 80 years of age.
- •Diagnosis of ALS by revised El Escorial Criteria, at study entry within 24 months of first symptoms.
- •Patients on existing ALS treatment must have been on a stable dose for 28 days.
排除标准
- •Patient with severe systemic infections, current or within the two weeks prior to randomization.
- •Subjects who, in the opinion of the investigator, are not capable of giving informed consent for the study or who are unable or unwilling to adhere to the study requirements outlined in the protocol.
- •Use of other investigational products or treatment in another investigational drug study within 30 days of screening
- •Pregnant or nursing (lactating) women.
研究组 & 干预措施
Treatment
TCD601 (siplizumab) administered with the contemporary standard of care regimen.
干预措施: TCD601 (Biological)
结局指标
主要结局
Evaluation of the safety of TCD601 in adult patients with ALS
时间窗: 12 Months
Assess the incidence of treatment-emergent Adverse Events \[Safety and Tolerability\]
次要结局
- Assessment of clinically relevant changes in vital signs(12 Months)
- Assessment of clinically relevant changes in laboratory measurements(12 Months)
- Evaluation of pharmacodynamics (PD) of TCD601(12 Months)
- Length of time from trial entry to tracheostomy / death(12 Months)
- Evaluation of the pharmacokinetics (PK)(12 Months)
- Evaluation of levels of biomarkers(12 Months)
- Correlation of ALS disease status with leucocyte phenotypic profiles(12 Months)