A Phase 2, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of AL001 in C9orf72-Associated Amyotrophic Lateral Sclerosis
试验速览
- 阶段
- 2 期
- 状态
- 终止
- 发起方
- Alector Inc.
- 入组人数
- 5
- 试验地点
- 3
- 主要终点
- Evaluation of Safety and Tolerability of AL001 Measured by Number of Subjects With Adverse Events
研究概览
简要总结
A phase 2 double-blind, placebo-controlled study of AL001 in participants with C9orf72-associated ALS.
详细描述
This is a phase 2 double-blind, placebo-controlled trial to test the safety, tolerability, pharmacokinetics, and pharmacodynamics of AL001 in participants with C9orf72-associated Amyotrophic Lateral Sclerosis.
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
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
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
- •Confirmation of C9orf72 mutation
- •Diagnosis of ALS by revised El Escorial criteria
- •Time since onset of muscle weakness due to ALS ≤36 months at the time of the Screening Visit
- •Slow Vital Capacity (VC) ≥50% of predicted capacity at the time of the Screening Visit
- •If taking riluzole, must be on a stable dose of riluzole for at least 30 days prior to the Screening Visit. Riluzole naive participants are allowed.
- •If taking edaravone, must have completed at least one cycle of edaravone prior to the Screening Visit and plan to continue edaravone during the study. Edaravone naive participants are allowed.
- •Females must not be pregnant, breastfeeding or planning to conceive within the study period. Males must agree to use acceptable contraception
- •Capable of providing informed consent at the Screening visit and complying with study procedures throughout the study
排除标准
- •Clinically significant, unstable, medical condition (other than ALS)
- •Clinically significant heart disease, liver disease or kidney disease
- •Cognitive impairment or dementia
- •Current uncontrolled hypertension
- •History of unresolved cancer
- •Any experimental gene therapy
- •Any experimental vaccine (any vaccine against COVID-19 either approved or administered under an Emergency Use Authorization is allowed)
研究组 & 干预措施
AL001
AL001 every 4 weeks
干预措施: AL001 (Drug)
Placebo
Placebo every 4 weeks
干预措施: Placebo (Drug)
结局指标
主要结局
Evaluation of Safety and Tolerability of AL001 Measured by Number of Subjects With Adverse Events
时间窗: 24 weeks
Count of participants with adverse events during the study treatment period
Immunogenicity of AL001
时间窗: Week 24
Count of participants positive for Anti-drug Antibodies (ADAs) to AL001 at week 24
Pharmacokinetics (PK) of AL001 in Serum
时间窗: Week 24
Concentration of AL001 in Serum at week 24
Pharmacokinetics (PK) of AL001 in CSF
时间窗: Week 24
Concentration of AL001 in Cerebrospinal fluid (CSF) at week 24
Change From Baseline in Plasma Progranulin
时间窗: 24 weeks
Evaluate the change from baseline to week 24 in plasma progranulin levels
Change From Baseline in CSF Progranulin
时间窗: 24 weeks
Evaluate the change from baseline to week 24 in Cerebrospinal fluid (CSF) progranulin levels
次要结局
- Change From Baseline in Plasma Neurofilament Light Chain(24 weeks)
- Change From Baseline in CSF Neurofilament Light Chain(24 weeks)