A Single-arm, Open-label, Phase I Clinical Study to Evaluate the Safety and Efficacy of a Single Intrathecal Injection of RJK002 in Patients With Amyotrophic Lateral Sclerosis (ALS)
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 9
- 试验地点
- 1
- 主要终点
- incidence of adverse events (AEs)/serious adverse events (SAEs)
研究概览
简要总结
The goal of the study aims to evaluate the safety and tolerability of a single intrathecal injection of RJK002 in subjects with amyotrophic lateral sclerosis (ALS), and to determine the recommended Phase II dose (RP2D).
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
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
- •Female or male subjects who are ≥ 18 years of age at screening;
- •Patients with a diagnosis consistent with clinically or laboratory-supported possible, probable, or definite sporadic or familial ALSALS in accordance with Revised EI Escorial diagnostic criteria published by the World Federation of Neurology (WFN);
- •The duration of the disease from the first symptom (any ALS symptom) prior to the screening visit must be less than 2 years (inclusive);
- •The Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) score ≥30 during the screening period, and the three respiratory scores (dyspnea, upright respiration, and respiratory insufficiency) must be ≥3;
- •The forced vital capacity (FVC) of predicted during the screening period is ≥70% at screening;
- •Body mass index (BMI) greater than 18 kg/m2 at screening;
排除标准
- •Subjects with other neurological diseases similar to ALS that affect the evaluation of drug efficacy, such as cervical spondylotic myelopathy, syringomyelia, spinal cord and brain stem tumors, hirayama disease, multifocal motor neuropathy, multiple sclerosis, Guillain-Barre syndrome, Parkinson's disease and dementia;
- •Patients with a diagnosis consistent with clinically or laboratory-supported possible, probable, or definite sporadic or familial ALSALS in accordance with Revised EI Escorial diagnostic criteria published by the World Federation of Neurology (WFN);
- •Subjects who refuse to take food and medication by nasal feeding tube during the study period due to swallowing dysfunction;
研究组 & 干预措施
RJK002 Intrathecal injection
Eligible subjects will receive a single intrathecal administration of investigational product. 9 subjects will be involved in 3 doses cohort from low to high: 6E13 vg/person, 1.2E14 vg/person, and 2.4E14 vg/person, 3 subjects for each cohort.
干预措施: RJK002 Intrathecal injection (Drug)
结局指标
主要结局
incidence of adverse events (AEs)/serious adverse events (SAEs)
时间窗: Each visit within 5 years after administration
Dose limiting toxicities (DLT)
时间窗: 28 days after administration
Measured as any drug-related serious adverse event that meet DLT standard. If a dose has less than 33% DLTs it will be considered tolerable.
次要结局
- Change in the ALS Functional Rating Scale-revised (ALSFRSr) Score(28 days, 56 days, 84 days, 6 months, 12months, 18 months after administration)