Modifying Orphan Disease Evaluation (MODE) Study: A Multicenter, Open-label Study of the Effects of CER-001 on Plaque Volume in Subjects With Homozygous Familial Hypercholesterolemia (HoFH)
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- 入组人数
- 23
- 试验地点
- 1
- 主要终点
- Percent change from baseline to follow-up in carotid mean vessel wall area
研究概览
简要总结
The available medications used to treat HoFH are targeted at reducing circulating levels of total and LDL-cholesterol. These measures can retard the progression of cardiovascular disease, however, they are unlikely to regress existing disease due to years of cholesterol accumulation in the vessel walls and therefore cannot adequately reduce the existing risk for an ischemic event. HDL has multiple actions that could lead to plaque stabilization and regression, such as rapid removal of large quantities of cholesterol from the vasculature, improvement in endothelial function, protection against oxidative damage and reduction in inflammation. This study will assess the effects of CER-001, a recombinant human Apo-A-1 based HDL mimetic, on indices of atherosclerotic plaque progression and regression as assessed by 3Tesla MRI measurements in patients with HoFH.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 12 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male or female subject 12 years or older
- •Subject presents with Homozygous FH
排除标准
- •Weight >100 kg
- •Subjects with significant health problems in the recent past including blood disorders, cancer, or digestive problems
- •Female subjects of child-bearing potential
- •Known major hematologic, renal , hepatic, metabolic, gastrointestinal or endocrine dysfunction
- •Contraindication to MRI scanning that would preclude the use of contrast-enhanced 3TMRI
研究组 & 干预措施
CER-001
Open label single arm study of CER-001
干预措施: CER-001 (Drug)
结局指标
主要结局
Percent change from baseline to follow-up in carotid mean vessel wall area
时间窗: Baseline then 6 months and/or ~2 weeks post final dose
Percent change from baseline to follow-up in carotid mean vessel wall area
次要结局
- Change in carotid vessel wall volume(Baseline then 6 months and/or ~2 weeks post final dose)