A Phase 2 Pilot Study to Assess the Safety and Efficacy of CAT-2003 in Patients With Chylomicronemia
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- 入组人数
- 13
- 主要终点
- Percent change from baseline in fasting triglycerides in patients with chylomicronemia
研究概览
简要总结
The purpose of this study is to assess safety and efficacy of CAT-2003 in patients with chylomicronemia. The study will evaluate the effects of CAT-2003 on fasting total and chylomicron triglyceride levels, as well as postprandial total and chylomicron triglyceride clearance.
This is a single-blind study. All patients will receive placebo for 1 week, and CAT-2003 for 12 weeks during the 13 week treatment period.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- Single (Participant)
入排标准
- 年龄范围
- 18 Years 至 74 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Familial Chylomicronemia (Fasting triglycerides ≥ 880 mg/dL at Screening and documented history of plasma post-heparin LDL activity < 20% of normal or genetic confirmation of homozygosity or compound heterozygosity for loss-of-function mutations in familial chylomicronemia-causing genes) OR
- •Non-familial Chylomicronemia (Fasting triglycerides ≥ 440 mg/dL at Screening and documented history of fasting triglycerides ≥ 880 mg/dL)
排除标准
- •History of any major cardiovascular event within 6 months of Screening
- •Type I diabetes mellitus or use of insulin
- •History of pancreatitis within 3 month of Screening
研究组 & 干预措施
CAT-2003 or Placebo
All patients will receive placebo for 1 week, and CAT-2003 for 12 weeks during the 13 week treatment period.
干预措施: CAT-2003 (Drug)
CAT-2003 or Placebo
All patients will receive placebo for 1 week, and CAT-2003 for 12 weeks during the 13 week treatment period.
干预措施: Placebo (Drug)
结局指标
主要结局
Percent change from baseline in fasting triglycerides in patients with chylomicronemia
时间窗: 12 Weeks
次要结局
- Absolute and percent change from baseline on chylomicron triglyceride clearance(12 Weeks)
- Absolute and percent change from baseline on postprandial total and chylomicron triglyceride levels(12 Weeks)
- Absolute and percent change from baseline in plasma non-HDL-C(12 Weeks)
- Frequency of adverse events(13 weeks)