A Multiple Ascending Dose Phase 1 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Intrathecally Administered LY4256984 in Participants With Sporadic Amyotrophic Lateral Sclerosis
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 入组人数
- 32
- 试验地点
- 22
- 主要终点
- Number of Participants with One or More Treatment Emergent Adverse Events (TEAEs), Serious Adverse Event(s) (SAEs), and Adverse Event(s) (AEs) Considered by the Investigator to be Related to Study Drug Administration
研究概览
简要总结
The purpose of this study is to evaluate how well LY4256984 is tolerated and what side effects may occur in participants with sporadic amyotrophic lateral sclerosis (ALS). The study drug will be administered intrathecally (IT) into the spine. Blood tests will be performed to check how much LY4256984 gets into the bloodstream and how long it takes the body to eliminate it.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Basic Science
- 盲法
- Double (Participant, Investigator)
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
- •Have a definite, possible, or probable diagnosis of sporadic amyotrophic lateral sclerosis (ALS) made by a physician experienced with the management of ALS
- •ALS symptom onset as determined by the Investigator within 24 months of Screening
- •Have a body mass index (BMI) within the range of greater than or equal to 18.0 and less than or equal to 35.0 kilogram per square meter (kg/m²) (inclusive)
排除标准
- •Have a history or presence of medical illness including, but not limited to, any cardiovascular, renal, hepatic, gastrointestinal, respiratory, hematological, endocrine, psychiatric, or neurological disease, convulsions, or any clinically significant laboratory abnormality
- •Have a history of another neurodegenerative disease or significant dementia/severe cognitive problems
- •Have serum alanine aminotransferase (ALT), aspartate transferase (AST), or total bilirubin levels greater than 2 x upper limit of normal.
- •Have a significant renal impairment (estimated glomerular filtration rate <60 milliliters per minute [mL/min]/1.73 m²).
- •Have a 12-lead electrocardiogram (ECG) abnormality at screening, in the opinion of the investigator, that increases the risks associated with participating in the study
- •Show clinically significant abnormalities in lumbar spine previously known or determined by screening lumbar X-ray or fluoroscopy (if performed)
研究组 & 干预措施
LY4256984 (Cohorts 1-4)
Multiple ascending doses (MAD) of LY4256984 administered intrathecally (IT)
干预措施: LY4256984 (Drug)
Placebo
Administered IT
干预措施: Placebo (Drug)
结局指标
主要结局
Number of Participants with One or More Treatment Emergent Adverse Events (TEAEs), Serious Adverse Event(s) (SAEs), and Adverse Event(s) (AEs) Considered by the Investigator to be Related to Study Drug Administration
时间窗: Baseline up to Approximately Day 253
A summary of TEAEs, SAEs, and AEs regardless of causality, will be reported in the Reported Adverse Events module
次要结局
- Pharmacokinetics (PK): Plasma Area Under the Concentration Versus Time Curve (AUC) of LY4256984(Baseline up to Approximately Day 253)
- PK: Plasma Maximum Concentration (Cmax) of LY4256984(Baseline up to Approximately Day 253)
- PK: Cerebrospinal Fluid Concentrations (CSF) of LY4256984(Baseline up to Approximately Day 253)