A Phase 2 Open Label Trial of 3K3A-APC in Amyotrophic Lateral Sclerosis
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- 入组人数
- 16
- 试验地点
- 1
- 主要终点
- Number of Participants Who Had Any Serious Adverse Events or Any Adverse Events With Severity Higher Than "Moderate".
研究概览
简要总结
Phase 2 open label trial to investigate the safety and potentially efficacy of 3K3A-APC in patients with Amyotrophic Lateral Sclerosis (ALS).
详细描述
This Phase 2 open label trial seeks to investigate whether a novel therapy named 3K3A-APC is safe and potentially effective in patients with Amyotrophic Lateral Sclerosis (ALS). A total of 16 patients with ALS will be enrolled into 2 dose cohorts with five doses of 15mg or 30mg doses given 12 hours apart in each cohort. The primary study outcomes are to ensure the safety and tolerability of 3K3AAPC in ALS patients, and to determine whether 3K3A-APC is able to reduce the pathological changes that might possibly cause ALS.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Sequential
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 75 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients must have clinically definite ALS (Awaji Criteria)
- •Male or female age 18 years and less than 75 years at time of ALS study
- •Symptom onset less than 36 months before screening
- •Diagnosis of ALS less than 24 months before screening
- •Clinically definite Upper Motor Neuron signs
排除标准
- •Current treatment with anticoagulants (e.g., warfarin, novel oral anticoagulants, heparin) that might preclude safe completion of the lumbar puncture
- •Condition that precludes the safe performance of routine lumbar puncture, such as prohibitive lumbar spinal disease, bleeding diathesis, or clinically significant coagulopathy or thrombocytopenia
- •Use of investigational drugs or devices within 60 days prior to Baseline (dietary supplements taken outside of a clinical trial are not exclusionary, e.g., coenzyme Q10)
- •Prolonged prothrombin time or activated partial thromboplastin time >2xULN
- •Severe hypertension or hypotension
- •Glomerular filtration rate (GFR) <35 mL/min
- •Forced vital capacity (FVC) at screening of <50% of predicted
- •Prior exposure to any exogenous form of APC
- •Inability to lie flat for procedures (MRI, PET, LP)
- •Pregnant or lactating during the study period
研究组 & 干预措施
15mg Dose Group
Participants will receive a fixed dose regimen of five doses of 15mg.
干预措施: 3K3A-APC Protein (Drug)
30mg Dose Group
Participants will receive a fixed dose regimen of five doses of 30mg.
干预措施: 3K3A-APC Protein (Drug)
结局指标
主要结局
Number of Participants Who Had Any Serious Adverse Events or Any Adverse Events With Severity Higher Than "Moderate".
时间窗: 15 Days
Number of participants who had any serious adverse events or any adverse events with severity higher than "moderate", as determined by the Principal Investigator, using the composite safety assessment including clinical laboratory testing (full blood count, biochemistry, coagulation, iron study, CSF analysis and ECG), physical examination and self-reporting of adverse events. All clinical significant findings in the composite safety assessment were reported as adverse events.
Percentage of Change in PERSI Score in the Motor Cortex Before and After Dosing
时间窗: 7 Days
PERSI (Parametric Estimation of Reference Signal Intensity) score is the measurement of microglial activation in the motor cortex utilising serial \[18F\]FEMPA PET imaging. The percentage of change in PERSI score before and after dosing in the two (2) dose cohorts is calculated.
次要结局
- Monocyte Activation(7 Days)
- Chemokine Level(7 Days)
- Soluble CD14 Level(7 Days)
- Diffusion Kurtosis Using MRI Scan(7 Days)
- Cytokine Level(7 Days)
- Kynurenine Level(7 Days)
- Neurofilament Level(7 Days)