A Phase 2 Open-Label Study to Assess the Pharmacodynamics, Pharmacokinetics, Safety and Tolerability of AKCEA-ANGPTL3-LRx (ISIS 703802) Administered Subcutaneously to Patients With Familial Chylomicronemia Syndrome (FCS)
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 3
- 试验地点
- 1
- 主要终点
- Absolute Change From Baseline to Month 3 in Fasting Triglycerides (TG)
研究概览
简要总结
This is a single center, open-label study to evaluate the efficacy of AKCEA-ANGPTL3-LRx for reduction of triglyceride (TG) levels in participants with FCS.
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
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Genetically confirmed chylomicronemia syndrome.
- •Fasting triglycerides greater than or equal to (>=) 750 milligrams per deciliter (mg/dL) [8.4 millimoles per liter (mmol/L)] at Screening.
排除标准
- •Diabetes mellitus if newly diagnosed or if glycated hemoglobin (HbA1c) >= 9.0%.
- •Active pancreatitis within 2 weeks of screening.
- •Acute coronary syndrome within 6 months of screening.
- •Major surgery within 3 months of screening.
- •Treatment with Glybera therapy within 2 years of screening.
- •Previous treatment with AKCEA-ANGPTL3-LRx.
- •Have any other conditions in the opinion of the investigator which could interfere with the patient participating in or completing the study.
研究组 & 干预措施
AKCEA-ANGPTL3-LRx 20 mg
Participants received a subcutaneous (SC) injection of AKCEA-ANGPTL3-LRx, 20 milligrams (mg), weekly (QW) for 13-weeks of treatment period. Participants were followed up to Week 26.
干预措施: AKCEA-ANGPTL3-LRx (Drug)
结局指标
主要结局
Absolute Change From Baseline to Month 3 in Fasting Triglycerides (TG)
时间窗: Baseline to Month 3
Baseline was defined as the average of Day 1 predose fasting assessment and the last fasting measurement prior to Day 1 pre-dose fasting assessment. Month 3 was defined as the average of Week 13 and Week 14 fasting assessments.
Percent Change From Baseline to Month 3 in Fasting Triglycerides (TG)
时间窗: Baseline to Month 3
Baseline was defined as the average of Day 1 predose fasting assessment and the last fasting measurement prior to Day 1 pre-dose fasting assessment. Month 3 was defined as the average of Week 13 and Week 14 fasting assessments.
次要结局
- Absolute Change From Baseline to Month 3 in Fasting Angiopoietin-Like 3 (ANGPTL3)(Baseline to Month 3)
- Percent Change From Baseline to Month 3 in Fasting Angiopoietin-like 3 (ANGPTL3)(Baseline to Month 3)
- Fasting Lipid and Lipoprotein Measurements at Month 3(Month 3)
- Absolute Change From Baseline to Month 3 in Other Fasting Lipid Parameters(Baseline to Month 3)
- Percent (%) Change From Baseline to Month 3 in Other Fasting Lipid Parameters(Baseline to Month 3)
- Change From Baseline to Day 92 in Maximum Postprandial Triglycerides (TG)(Baseline to Day 92)
- Number of Participants Who Experienced Abdominal Pain During the Treatment Period(Days 1, 29, 57 and 92)
- Number of Participants With Treatment Emergent Adverse Events (TEAEs)(From time of informed consent to end of follow-up period (Up to Week 26))