Phase 3, Multicenter, Randomized, 48 Week, Double Blind, Parallel Group, Placebo Controlled Study to Evaluate Efficacy and Safety of CER-001 on Vessel Wall Area in Patients With Genetically Defined Familial Primary Hypoalphalipoproteinemia
试验速览
- 阶段
- 3 期
- 状态
- 终止
- 发起方
- 入组人数
- 30
- 试验地点
- 1
- 主要终点
- Change in Mean Vessel Wall Area (MVWA) of the Carotid Artery
研究概览
简要总结
The purpose of this study is to assess the impact of 29 intravenous infusions of CER-001 vs. placebo, given at weekly (9 infusions) and biweekly (20 infusions) intervals on carotid vessel wall area as measured by 3TMRI, when administered to patients with familial primary hypoalphalipoproteinemia with proven CVD and appropriate background lipid-lowering therapy.
详细描述
Subjects will be required to have acceptable baseline 3TMRI imaging of carotid arteries. Subjects meeting all eligibility criteria will be randomized to receive CER-001 or placebo (2:1 randomization scheme). Randomized subjects will return weekly for a total of 9 infusions and then biweekly for an additional 20 infusions. 3TMRI imaging of the carotid and femoral arteries will be performed at Week 8, Week 24 (primary endpoint) and Week 48. The total study duration from randomization can range from 50 to 54 weeks for patients completing the study as designed.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male and female patients, aged 18 and above.
- •ApoA-I < 70 mg/dL
- •Symptomatic or asymptomatic cardiovascular disease
- •Diagnosis of genetically confirmed HDL-c deficiency due to defects in genes coding for ABCA1 and/or ApoA-1
- •Stable doses of lipid lowering therapies for at least 6 weeks prior to baseline procedures
排除标准
- •Females of childbearing potential
- •Patients with LCAT mutations
- •Patients who experienced recent cardiovascular or cerebrovascular events
- •Hypertriglyceridemia (>500 mg/dL)
- •Severe anemia (Hgb < 10 g/dL)
- •Uncontrolled diabetes (HbA1c >10%)
- •Congestive heart failure (NYHA class II or higher)
- •Contraindication for MRI scanning (e.g., implanted metal objects, claustrophobia)
研究组 & 干预措施
CER-001
CER-001 infusion; 9 weekly infusions followed by 20 biweekly infusions
干预措施: CER-001 (Drug)
Placebo
Saline infusion; 9 weekly infusions followed by 20 biweekly infusions
干预措施: Placebo (Drug)
结局指标
主要结局
Change in Mean Vessel Wall Area (MVWA) of the Carotid Artery
时间窗: Baseline to Week 24
Change from baseline to Week 24 carotid MVWA; CER-001 versus placebo; measured by 3TMRI
次要结局
- Change in Mean Vessel Wall Area (MVWA) of the Carotid Artery(Baseline to Week 48)
- Change in Target to Background Ratio (TBR) of the Carotid Artery(Baseline to Week 24)