A Double-blind, Randomized, Placebo-controlled, Multicentric, Phase II Study in Adult Patients With Amyotrophic Lateral Sclerosis (ALS) to Assess Efficacy, Safety, Tolerability and Pharmacokinetics of Multiple Intravenous Infusions of NX210c
试验速览
- 阶段
- 2 期
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 80
- 试验地点
- 16
- 主要终点
- The effect of NX210c on blood neurofilament light chain (NfL) or on a blood and cerebrospinal fluid (CSF) biomarker of BBB integrity.
研究概览
简要总结
This study will investigate the efficacy, safety, tolerability and pharmacokinetics (PK) of multiple intravenous infusions of NX210c, at two dose levels, in patients with Amyotrophic lateral sclerosis (ALS).
详细描述
Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disease that predominantly affects motor neurons of both the brain and the spinal cord. This leads to muscular atrophy and paralysis, with the majority of patients succumbing to respiratory failure 3-4 years from symptom onset. To date, therapeutic options for ALS are limited and there is no curative treatment. Management of ALS is otherwise supportive and palliative.
There is strong evidence for blood-brain and blood-spinal cord barrier dysfunction in the early stages of ALS. Preclinical in vitro and in vivo data have shown that NX210c exhibits important properties that may be suitable for the treatment of neurological disorders in humans i.e., blood-brain barrier (BBB) integrity restoration, neurotransmission enhancement and neuroprotection.
Based on the safety data collected in a first-in-human single ascending dose study and a multiple ascending dose (MAD) study in healthy elderly subjects, NX210c has been demonstrated to be well tolerated and without safety concerns.
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
- Double (Participant, Investigator)
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Aged ≥ 18 years, inclusive at screening.
- •Patients diagnosed as having possible, probable, probable laboratory-supported, or definite ALS according to El Escorial Revised criteria.
- •King's Clinical Staging Stage ≤3, disease duration of ≤ 36 calendar months
排除标准
- •Patients with any cognitive or psychological disorder, intellectual disability or other significant impairment that would result in an inability to understand and sign the informed consent.
- •History of any clinically significant or unstable medical, neurological, psychiatric condition, disorder or disease (other than ALS) or social circumstances that, based on the investigator's judgment, would interfere with the conduct of the study or pose a risk to the patient if they were to participate.
研究组 & 干预措施
NX210c 5 mg/kg
Dose: 5 mg/kg
干预措施: NX210c (Drug)
NX210c 10 mg/kg
Dose: 10 mg/kg
干预措施: NX210c (Drug)
Placebo
干预措施: Placebo (Drug)
结局指标
主要结局
The effect of NX210c on blood neurofilament light chain (NfL) or on a blood and cerebrospinal fluid (CSF) biomarker of BBB integrity.
时间窗: From predose to 6-week follow-up
Changes in serum NfL or changes in Albumin CSF/serum quotient (Qalb)
次要结局
未报告次要终点