A Phase 1, Randomized, Double-Blind, Placebo-Controlled Dose Escalating Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of Single Ascending and Multiple Ascending Doses of Prosetin in Healthy Volunteers and Participants With Amyotrophic Lateral Sclerosis (ALS) With an Optional Open-Label Extended Treatment Period for ALS Participants Who Complete 14 Days of Blinded Treatment
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 发起方
- 入组人数
- 72
- 试验地点
- 4
- 主要终点
- Parts A, B, C, D: Number of Participants Experiencing Adverse Events (AEs) and Serious Adverse Events (SAEs)
研究概览
简要总结
The primary purpose of this study is to evaluate the safety and tolerability of prosetin in healthy volunteers and participants with ALS.
详细描述
PRO-101 is a four-part study. Parts A and B, which respectively evaluated the safety, tolerability, and PK of single and multiple ascending doses of prosetin in 48 healthy volunteers, have been completed.
Parts C and D, which are ongoing, will evaluate the effects of prosetin on safety, tolerability, PK, and biomarkers in 24 participants with ALS. Part C is a double-blind, placebo-controlled, multiple ascending dose component of the study, and Part D is an optional 52-week open-label extension available to ALS participants who complete 14 days of dosing in Part C.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Sequential
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
盲法说明
Parts A, B, and C of PRO-101 are double-blind, dose escalating portions of the study. Part D is an open-label extension in which no parties are masked and all participants receive the study drug.
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
- 未提供
排除标准
- 未提供
研究组 & 干预措施
Part A - single dose of placebo
Healthy volunteers were administered a single dose of prosetin-matched placebo oral solution.
干预措施: placebo (Drug)
Part A - single ascending doses of prosetin
Healthy volunteers were administered a single dose of prosetin oral solution at 0.03, 0.06, 0.12, or 0.24 mg/kg.
干预措施: prosetin (Drug)
Part B - multiple doses of placebo
Healthy volunteers were administered a once-daily dose of prosetin-matched placebo for 14 days.
干预措施: placebo (Drug)
Part B - multiple ascending doses of prosetin
Healthy volunteers were administered a once-daily dose of prosetin at 0.06 or 0.10 mg/kg for 14 days.
干预措施: prosetin (Drug)
Part C - multiple doses of placebo in participants with ALS
Participants are administered a once-daily dose of prosetin-matched placebo for 14 days.
干预措施: placebo (Drug)
Part C - multiple ascending doses of prosetin in participants with ALS
Participants will be administered a once-daily dose of prosetin at multiple ascending dose levels for 14 days.
干预措施: prosetin (Drug)
Part D - open-label administration of prosetin in participants with ALS
Participants will be administered a once-daily dose of prosetin for up to 52 weeks.
干预措施: prosetin (Drug)
结局指标
主要结局
Parts A, B, C, D: Number of Participants Experiencing Adverse Events (AEs) and Serious Adverse Events (SAEs)
时间窗: Part A: Up to 28 days; Part B: Up to 42 days; Part C: Up to 28 days; Part D: Up to 54 weeks
Parts A, B, C, D: Number of Participants with Clinically Significant Laboratory Test Abnormalities
时间窗: Part A: Up to 28 days; Part B: Up to 42 days; Part C: Up to 28 days; Part D: Up to 54 weeks
Parts A, B, C, D: Number of Participants with Clinically Significant Vital Signs Abnormalities
时间窗: Part A: Up to 28 days; Part B: Up to 42 days; Part C: Up to 28 days; Part D: Up to 54 weeks
Parts A, B, C, and D: Number of Participants with Clinically Significant Electrocardiogram (ECG) Abnormalities
时间窗: Part A: Up to 28 days; Part B: Up to 42 days; Part C: Up to 28 days; Part D: Up to 54 weeks
Parts A, B, C, and D: Number of Participants with Clinically Significant Physical Examination Abnormalities
时间窗: Part A: Up to 28 days; Part B: Up to 42 days; Part C: Up to 28 days; Part D: Up to 54 weeks
Parts A, B, C, and D: Number of Participants with Clinically Significant Neurological Examination Abnormalities
时间窗: Part A: Up to 28 days; Part B: Up to 42 days; Part C: Up to 28 days; Part D: Up to 54 weeks
Parts A, B, C, and D: Number of Participants with Clinically Significant Ophthalmic Examination Abnormalities
时间窗: Part A: Up to 28 days; Part B: Up to 42 days; Part C: Up to 28 days; Part D: Up to 54 weeks
Parts C and D: Number of Participants with Clinically Significant Electroencephalogram (EEG) Abnormalities
时间窗: Part C: Up to 28 days; Part D: Up to 54 weeks
次要结局
- Parts A, B, C, and D: Maximum Observed Concentration (Cmax) of Prosetin in Plasma(Part A: Up to 28 days Part B: Up to 42 days Part C: Up to 28 days Part D: Up to 54 weeks)
- Parts A, B, C, and D: Time to Reach Maximum Observed Concentration (Tmax) of Prosetin in Plasma(Part A: Up to 28 days Part B: Up to 42 days Part C: Up to 28 days Part D: Up to 54 weeks)
- Parts A, B, C, and D: Area Under the Concentration-Time Curve (AUC) of Prosetin in Plasma(Part A: Up to 28 days Part B: Up to 42 days Part C: Up to 28 days Part D: Up to 54 weeks)
- Parts A, B, C, and D: Apparent Terminal Elimination Half-life (t1/2) of Prosetin in Plasma(Part A: Up to 28 days Part B: Up to 42 days Part C: Up to 28 days Part D: Up to 54 weeks)
- Parts A and D: Measure of Concentration of Prosetin in CSF(Part A: Day 1; Part D: Up to 48 weeks)