A Randomized, Double-Blind, Placebo-Controlled, Dose Escalation Study to Evaluate the Effect of SRP-1005 in Subjects With Huntington's Disease (INSIGHTT)
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 入组人数
- 32
- 试验地点
- 5
- 主要终点
- Number of Participants with a Treatment-emergent Adverse Event (TEAE), Serious Adverse Event (SAE), and Adverse Event of Special Interest (AESI)
研究概览
简要总结
This is a first-in-human, multi-center trial studying the effects of SRP-1005 in participants with Huntington's disease (HD).
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
- 21 Years 至 70 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Genetically confirmed diagnosis of huntingtin (HTT) cytosine-adenine-guanine repeat length ≥
- •Participant has HD Integrated Staging System (HD-ISS) Stage 2 or Mild Stage
- •At screening, the participant must be classified with the Enroll-HD HD-ISS Modified Calculator as either Stage 2 or Mild Stage
- •Participants who are of childbearing potential, or with partners of childbearing potential, who are sexually active must agree to use a highly effective method of contraception throughout study participation, and for at least 90 days following the end of study.
排除标准
- •Any condition that would compromise the safety or feasibility of lumbar puncture or magnetic resonance imaging (MRI).
- •Presence of other significant neurological or systemic illnesses.
- •Current, chronic or active human immunodeficiency virus, hepatitis B/C.
- •Recent use of investigational agents or HTT-lowering therapies.
- •Uncontrolled psychiatric illness or substance use disorders.
- •Pregnancy or lactation.
- •Note: Other inclusion/exclusion criteria apply.
研究组 & 干预措施
Cohort 2: SRP-1005 Dose 2
Participants will receive SRP-1005 or placebo.
干预措施: SRP-1005 (Drug)
Cohort 2: SRP-1005 Dose 2
Participants will receive SRP-1005 or placebo.
干预措施: Placebo (Drug)
Cohort 3: SRP-1005 Dose 3
Participants will receive SRP-1005 or placebo.
干预措施: SRP-1005 (Drug)
Cohort 1: SRP-1005 Dose 1
Participants will receive SRP-1005 or placebo.
干预措施: SRP-1005 (Drug)
Cohort 1: SRP-1005 Dose 1
Participants will receive SRP-1005 or placebo.
干预措施: Placebo (Drug)
Cohort 3: SRP-1005 Dose 3
Participants will receive SRP-1005 or placebo.
干预措施: Placebo (Drug)
Cohort 4: SRP-1005 Dose 4
Participants will receive SRP-1005 or placebo.
干预措施: SRP-1005 (Drug)
Cohort 4: SRP-1005 Dose 4
Participants will receive SRP-1005 or placebo.
干预措施: Placebo (Drug)
结局指标
主要结局
Number of Participants with a Treatment-emergent Adverse Event (TEAE), Serious Adverse Event (SAE), and Adverse Event of Special Interest (AESI)
时间窗: Baseline through Week 24
次要结局
- Plasma Concentration of SRP-1005(Baseline through Day 17)
- Urine Concentration of SRP-1005(Baseline through Day 17)
- Cerebrospinal Fluid Concentration of SRP-1005(Baseline through Week 24)