A First in Human Phase 1 Study in Healthy Volunteers and in Participants With Frontotemporal Dementia (FTD) With Granulin Mutation
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 发起方
- Alector Inc.
- 入组人数
- 64
- 试验地点
- 8
- 主要终点
- Evaluation of safety and tolerability of AL001 measured by number of subjects with adverse events and Dose Limiting Adverse Event (DLAEs)
研究概览
简要总结
A first in human phase 1 study in healthy volunteers and participants with Granulin (GRN) mutation causative of frontotemporal dementia (FTD) to assess AL001 safety, tolerability, pharmacokinetics, and pharmacodynamics
详细描述
This is a first-in-human Phase 1 study designed to investigate the safety, tolerability, PK and PD of intravenously administered AL001 in Healthy Volunteers and FTD-GRN mutation carriers.
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 80 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 是
入选标准
- •BMI 18.0-35.0 kg/m2
- •45-120 kg, inclusive
- •At screening, females must be non-pregnant and non-lactating, or of nonchildbearing potential (either surgically sterilized or physiologically incapable of becoming pregnant, or at least 1-year postmenopausal (amenorrhoea duration of 12 consecutive months); nonpregnancy will be confirmed for all females by a pregnancy test conducted at screening, (each) admission, and at follow-up.
- •Female participants of child-bearing potential must agree to use adequate contraception from screening until 90 days after the follow-up visit.
- •In good physical health on the basis of no clinically significant findings from medical history, physical examination (PE), laboratory tests, 12 lead ECG, and vital signs, as judged by the Investigator.
- •Willingness and able to comply with the study protocol, in the investigator's judgement.
排除标准
- •Known history of severe allergic, anaphylactic, or other hypersensitivity reactions to chimeric, human, or humanized antibodies or fusion proteins.
- •Positive drug or alcohol at screening and prior to first dose
- •History of alcohol abuse or substance abuse
研究组 & 干预措施
Saline Solution
Saline solution will be administered as a single infusion for each cohort in a ratio of 6 active and 2 placebo subjects
干预措施: Placebo (Other)
AL001
Up to six single ascending doses of AL001
干预措施: AL001 (Biological)
结局指标
主要结局
Evaluation of safety and tolerability of AL001 measured by number of subjects with adverse events and Dose Limiting Adverse Event (DLAEs)
时间窗: 85 days
Incidence of adverse events and dose limiting Adverse Events during the DLAE observation period and/or study treatment periods.
次要结局
- Pharmacokinetics (PK) of AL001(85 days)
- Maximum plasma concentration (Cmax) for AL001(85 days)
- Area under the curve concentration (AUC) for AL001(85 days)