A 4-Week Randomized, Double-Blind, Placebo-Controlled, Phase 2a Safety and PK Study of Oral ELND005 in Young Adults With Down Syndrome Without Dementia
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 23
- 试验地点
- 3
- 主要终点
- Incidence of Adverse Events (TEAEs)
研究概览
简要总结
This is a prospective, randomized, double-blind, placebo-controlled, parallel-group, three-arm, multicenter study of the safety and PK of ELND005 administered orally for 4 weeks. This study will enroll Down Syndrome patients 18 to 45 years of age (inclusive) without dementia.
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
- Triple (Participant, Care Provider, Investigator)
入排标准
- 年龄范围
- 18 Years 至 45 Years（Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •18 - 45 years of age
- •Has an IQ of > 40 (K-BIT)
- •Able and willing to have a brain MRI
排除标准
- •Symptoms of dementia or worsening cognition over the past year.
- •Has a history of hepatitis B, hepatitis C, or HIV
研究组 & 干预措施
ELND005 BID
ELND005 250 mg BID
干预措施: ELND005 (Drug)
ELND005 QD
ELND005 250 mg QD
干预措施: ELND005 (Drug)
Placebo
Placebo BID
干预措施: Placebo (Drug)
结局指标
主要结局
Incidence of Adverse Events (TEAEs)
时间窗: 4 weeks
For all AE summaries, if a patient had more than one AE within a preferred term, the patient was counted only once, at the maximum severity and with the closest relationship to study drug. If a patient had more than one AE within a SOC, the subject was similarly counted only once when reporting results for that SOC.
次要结局
未报告次要终点