A Phase 1b, Multicenter, Randomized, Placebo-Controlled, Double-Blind Study, Followed by an Open-Label Extension, to Determine the Safety, Pharmacokinetics, and Pharmacodynamics of DNL343 in Participants With Amyotrophic Lateral Sclerosis
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 30
- 试验地点
- 7
- 主要终点
- Incidence of treatment-emergent adverse events (TEAEs) throughout the double-blind period
研究概览
简要总结
This is a Phase 1b, multicenter, randomized, placebo-controlled, double-blind study of 28 days, followed by an 18-month open-label extension, designed to evaluate the safety, pharmacokinetics (PK), and pharmacodynamics (PD) of DNL343 in participants with amyotrophic lateral sclerosis (ALS)
详细描述
This clinical trial information was submitted voluntarily under the applicable law and, therefore, certain submission deadlines may not apply. (That is, clinical trial information for this applicable clinical trial was submitted under section 402(j)(4)(A) of the Public Health Service Act and 42 CFR 11.60 and is not subject to the deadlines established by sections 402(j)(2) and (3) of the Public Health Service Act or 42 CFR 11.24 and 11.44.).
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 80 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosis of sporadic or familial ALS
- •≤ 4 years since ALS symptom onset
- •Stable doses of approved ALS treatments (riluzole and/or edaravone) for at least 2 months prior to screening
- •Participants must be able to swallow the study intervention
- •Vital capacity >50% predicted at screening
- •Women must have been surgically sterilized, be postmenopausal, or for participants of childbearing potential, must not be pregnant, and both the participant and the male partner must use highly effective contraception
- •Men, and sex partner if a woman of childbearing potential, must use highly effective contraception
排除标准
- •Any history of unstable or poorly controlled psychiatric, endocrine, pulmonary, cardiovascular, gastrointestinal, hepatic, pancreatic, renal, metabolic, hematologic, immunologic, or allergic disease, or other major disorders
- •Positive serum pregnancy test or currently lactating or breastfeeding
- •History of malignancy within 5 years
- •History of clinically significant neurologic disorders other than ALS
研究组 & 干预措施
DNL343 (High Dose)
干预措施: DNL343 (Drug)
DNL343 (Low Dose)
干预措施: DNL343 (Drug)
Placebo
干预措施: Placebo (Drug)
结局指标
主要结局
Incidence of treatment-emergent adverse events (TEAEs) throughout the double-blind period
时间窗: 28 Days
次要结局
- PK parameter: Trough concentration (Ctrough) of DNL343 in plasma(19 months)
- PK parameter: Area under the concentration-time curve from time zero to 24 hours (AUC24) of DNL343 in plasma(19 months)
- PK parameter: Maximum concentration (Cmax) of DNL343 in plasma(19 months)
- Cerebrospinal fluid-to-plasma concentration ratio of DNL343 following multiple oral doses(19 months)
- PK parameter: Time to reach maximum concentration (tmax) of DNL343 in plasma(19 months)