Open-label, Phase 1b, Single-ascending Dose and Optional re Dosing Study to Evaluate the Safety of VERVE-101 Administered to Patients With Heterozygous Familial Hypercholesterolemia, Atherosclerotic Cardiovascular Disease, and Uncontrolled Hypercholesterolemia
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 入组人数
- 13
- 试验地点
- 1
- 主要终点
- Incidence of treatment-emergent adverse events (TEAEs), serious adverse events (SAEs), and adverse events of special interest (AESIs).
研究概览
简要总结
VT-1001 is an open-label, phase 1b, single-ascending dose study that will evaluate the safety of VERVE-101 administered to patients with heterozygous familial hypercholesterolemia (HeFH), atherosclerotic cardiovascular disease (ASCVD), and uncontrolled hypercholesterolemia. VERVE-101 uses base-editing technology designed to disrupt the expression of the PCSK9 gene in the liver and lower circulating PCSK9 and LDL-C in patients with established ASCVD due to HeFH. This study is designed to determine the safety and pharmacodynamic profile of VERVE-101 in this patient population.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Sequential
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 75 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male and/or female participants 18 up to 75 years at time of signing of informed consent
- •Female participants not of child-bearing potential
- •Diagnosis of HeFH
- •Established ASCVD
排除标准
- •Active or history of chronic liver disease
- •Current treatment with PCSK9 monoclonal antibody therapy
- •Current or past treatment with inclisiran
- •Clinically significant or abnormal laboratory values as defined by the protocol
研究组 & 干预措施
Part A: Single Ascending Dose Escalation/Adaptive Design
Participants will receive a single dose of VERVE-101 in multiple dose-escalation cohorts.
干预措施: VERVE-101 (Drug)
Part B: Single Dose Expansion
Participants will receive a single dose of VERVE-101 selected based on the doses studied in Part A.
干预措施: VERVE-101 (Drug)
结局指标
主要结局
Incidence of treatment-emergent adverse events (TEAEs), serious adverse events (SAEs), and adverse events of special interest (AESIs).
时间窗: up to Day 365
次要结局
- Evaluation of maximum observed concentration (Cmax)(up to Day 365)
- Evaluation of time to maximum observed concentration (tmax)(up to Day 365)
- Evaluation of terminal elimination half-life (t1/2)(up to Day 365)