A Multi-Modular Trial to Evaluate VGA039 in Healthy Volunteers and Patients With Von Willebrand Disease and Other Bleeding Disorders (VIVID)
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 入组人数
- 116
- 试验地点
- 25
- 主要终点
- Incidence of Treatment-Emergent Adverse Events [Safety and tolerability]
研究概览
简要总结
The VIVID study is structured in a master protocol format comprised of multiple parts that evaluate intravenous (IV) and subcutaneous (SC) VGA039 in healthy volunteers and subjects with von Willebrand Disease (VWD) and other bleeding disorders.
详细描述
This first-in-human study consists of 5 parts based on the subject population. Part 1 is a randomized, double-blind, placebo-controlled, single-ascending dose (SAD) evaluation of IV or SC VGA039 or placebo in up to 8 cohorts in healthy volunteers. Part 2 is an open-label, SAD of SC or IV VGA039 in up to 8 cohorts in subjects diagnosed with VWD. All participants will be enrolled, treated, and followed up for 15 weeks (IV SAD) or 8 weeks(SC SAD). Part 3 is an open-label, Phase 1b study of SC multiple doses (MD) of VGA039 in up to 4 cohorts. Part 4 is an open-label, Phase 2 study of SC single, surgical prophylaxis (SP) doses of VGA039 administered prior to a minor surgical procedure in subjects diagnosed with VWD in up to 2 cohorts. Part 5 is an open-label extension (OLE) study of SC MD of VGA039 in eligible subjects diagnosed with VWD who have previously participated in a VGA039 interventional trial.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Sequential
- 主要目的
- Treatment
- 盲法
- Double (Participant, Investigator)
入排标准
- 年龄范围
- 12 Years 至 60 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 是
入选标准
- 未提供
排除标准
- 未提供
研究组 & 干预措施
Part 4
Cohorts of VGA039 single dose for surgical prophylaxis
干预措施: VGA039 (Drug)
Part 3
Cohorts MD-1 to MD-4, SC VGA039 multiple doses, dose to be determined
干预措施: VGA039 (Drug)
Part 1
Cohorts 1-8 IV or SC VGA039 or Placebo dose to be determined
干预措施: VGA039 (Drug)
Part 1
Cohorts 1-8 IV or SC VGA039 or Placebo dose to be determined
干预措施: Placebo (Other)
Part 2
Cohorts A-H IV or SC VGA039 dose to be determined
干预措施: VGA039 (Drug)
结局指标
主要结局
Incidence of Treatment-Emergent Adverse Events [Safety and tolerability]
时间窗: From start of study drug administration until 15 or 8 weeks after IV or SC study drug administration, respectively
Incidence, nature and severity of adverse events (AEs) and serious adverse events (SAEs), including dose-limiting toxicities (DLTs).
次要结局
- Plasma Concentrations of single IV and SC doses of VGA039(From baseline until 15 or 8 weeks after IV or SC study drug administration, respectively)
- Pharmacodynamics of single IV and SC doses of VGA039(From baseline until 15 or 8 weeks after IV or SC study drug administration, respectively)
- Incidence of Anti-drug antibodies to VGA039(From baseline until 15 or 8 weeks after IV or SC study drug administration, respectively)