A Phase 1b Single Ascending Dose Study to Evaluate the Safety of VERVE-201 in Patients With Refractory Hyperlipidemia
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 入组人数
- 36
- 试验地点
- 17
- 主要终点
- Incidence of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs)
研究概览
简要总结
VT-20101 is an Open-label, Phase 1b, Single-ascending dose study that will evaluate the safety of VERVE-201 administered to patients with Refractory Hyperlipidemia.
VERVE-201 uses base-editing technology designed to inactivate the expression of the ANGPTL3 gene in the liver and lower circulating low-density lipoprotein cholesterol (LDL-C) and triglycerides. This study is designed to determine the safety and pharmacodynamic profile of VERVE-201 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 70 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Refractory hypercholesterolemia
- •Refractory hypertriglyceridemia
排除标准
- •Active or history of chronic liver disease
- •Current treatment with monoclonal antibody targeting ANGPTL3 or prior treatment within specified timeframe
- •Clinically significant or abnormal laboratory values as defined by the protocol
研究组 & 干预措施
Cohort 1: Single Ascending Dose Escalation
Participants will receive a single dose of VERVE-201.
干预措施: VERVE-201 (Drug)
Cohort 2: Single Ascending Dose Escalation
Participants will receive a single dose of VERVE-201.
干预措施: VERVE-201 (Drug)
Cohort 3: Single Ascending Dose Escalation
Participants will receive a single dose of VERVE-201.
干预措施: VERVE-201 (Drug)
Cohort 5: Single Ascending Dose Escalation
Participants will receive a single dose of VERVE-201.
干预措施: VERVE-201 (Drug)
Cohort 6: Single Ascending Dose Escalation
Participants will receive a single dose of VERVE-201.
干预措施: VERVE-201 (Drug)
Cohort 4: Single Ascending Dose Escalation
Participants will receive a single dose of VERVE-201.
干预措施: VERVE-201 (Drug)
结局指标
主要结局
Incidence of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs)
时间窗: Up to Day 365
次要结局
- Percent and absolute change from baseline in serum triglycerides(Up to Day 365)
- Percent and absolute change from baseline in serum triglycerides(Up to Day 365)
- Percent and absolute change from baseline in serum LDL-C(Up to Day 365)
- 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)
- Percent and absolute change from baseline in serum ANGPTL3 concentration(Up to Day 365)