NCT02211209已完成3 期
A Randomized, Double-Blind, Placebo-Controlled, Phase 3 Study of ISIS 304801 Administered Subcutaneously to Patients With Familial Chylomicronemia Syndrome (FCS)
适应症
干预措施
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 67
- 试验地点
- 1
- 主要终点
- Percent Change in Fasting Triglycerides (TG) From Baseline to Month 3
研究概览
简要总结
The purpose of this study is to evaluate the efficacy and safety of volanesorsen given for 52 weeks in participants with Familial Chylomicronemia Syndrome
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
- •History of chylomicronemia
- •A diagnosis of Familial Chylomicronemia Syndrome (Type 1 Hyperlipoproteinemia)
- •Fasting triglycerides (TG) ≥ 750 mg/dL (8.4 mmol/L) at Screening
排除标准
- •Diabetes mellitus if newly diagnosed or if HbA1c ≥ 9.0%
- •Other types of severe hypertriglyceridemia
- •Active pancreatitis within 4 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 IONIS-APOCIIIRx
- •Have any other conditions in the opinion of the investigator which could interfere with the participant participating in or completing the study
研究组 & 干预措施
Placebo
Placebo Comparator
Volanesorsen-matching placebo administered subcutaneously once-weekly for 52 weeks.
干预措施: Placebo (Drug)
Volanesorsen
Experimental
Volanesorsen 300 mg administered subcutaneously once-weekly for 52 weeks.
干预措施: Volanesorsen (Drug)
结局指标
主要结局
Percent Change in Fasting Triglycerides (TG) From Baseline to Month 3
时间窗: Baseline to 3 months
The Month 3 endpoint was defined as the average of Week 12 (Day 78) and Week 13 (Day 85) fasting assessments.
次要结局
- Change From Baseline in Postprandial TG Area Under the Curve (AUC)(0-9h)(Baseline to an on-treatment assessment between Week 13 and Week 19)
- Absolute Change From Baseline in Fasting TG at Month 3(Baseline to 3 months)
- Treatment Response Rate Defined as Participants With Fasting Plasma TG < 750 mg/dL at Month 3(Baseline to 3 months)
- Treatment Response Rate Defined as Participants With Fasting TG ≥ 40% Reduction From Baseline at Month 3(Baseline to 3 months)
- Frequency and Severity of Participant-reported Abdominal Pain During the Treatment Period(Baseline to 12 months)
- Frequency of the Composite of Episodes of Acute Pancreatitis and Participant-reported Moderate/Severe Abdominal Pain During the Treatment Period(12 months)
- Change From Baseline in Hepatosplenomegaly as Assessed by MRI at Week 52(Baseline to Week 52)
研究者
研究点 (1)
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