Open-label, Phase 1b, Single Ascending Dose Study to Evaluate the Safety of VERVE-102 Administered to Patients With Heterozygous Familial Hypercholesterolemia or Premature Coronary Artery Disease Who Require Additional Lowering of Low-density Lipoprotein Cholesterol
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 入组人数
- 85
- 试验地点
- 28
- 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-10201 is an Open-label, Phase 1b, Single-ascending Dose Study That Will Evaluate the Safety of VERVE-102 Administered to Patients With Heterozygous Familial Hypercholesterolemia (HeFH) or Premature Coronary Artery Disease (CAD) Who Require Additional Lowering of LDL-C. VERVE-102 Uses Base-editing Technology Designed to Disrupt the Expression of the PCSK9 Gene in the Liver and Lower Circulating PCSK9 and LDL-C. This Study is Designed to Determine the Safety and Pharmacodynamic Profile of VERVE-102 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
- •Diagnosis of HeFH or premature CAD
排除标准
- •Homozygous familial hypercholesterolemia
- •Active or history of chronic liver disease
- •Current treatment with PCSK9 inhibitor 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-102.
干预措施: VERVE-102 (Drug)
Cohort 2: Single Ascending Dose Escalation
Participants will receive a single dose of VERVE-102.
干预措施: VERVE-102 (Drug)
Cohort 4: Single Ascending Dose Escalation
Participants will receive a single dose of VERVE-102.
干预措施: VERVE-102 (Drug)
Cohort 3: Single Ascending Dose Escalation
Participants will receive a single dose of VERVE-102.
干预措施: VERVE-102 (Drug)
Cohort 5: Single Ascending Dose Escalation
Participants will receive a single dose of VERVE-102.
干预措施: VERVE-102 (Drug)
Cohort 6: Single Ascending Dose Escalation
Participants will receive a single dose of VERVE-102.
干预措施: VERVE-102 (Drug)
Cohort 9: Single Fixed Dose
Participants will receive a single dose of VERVE-102.
干预措施: VERVE-102 (Drug)
Cohort 7: Single Ascending Dose Escalation
Participants will receive a single dose of VERVE-102.
干预措施: VERVE-102 (Drug)
Cohort 8: Single Ascending Dose Escalation
Participants will receive a single dose of VERVE-102.
干预措施: VERVE-102 (Drug)
Cohort 10: Single Fixed Dose
Participants will receive a single dose of VERVE-102.
干预措施: VERVE-102 (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
- 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 LDL-C(up to Day 365)
- Evaluation of maximum observed concentration (Cmax)(up to Day 365)
- Percent and absolute change from baseline in plasma PCSK9 concentration(up to Day 365)