A Randomized, Double-Blind, Placebo-Controlled, Phase 3 Study of AKCEA-APOCIII-LRx Administered Subcutaneously to Patients With Familial Chylomicronemia Syndrome (FCS)
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 66
- 试验地点
- 47
- 主要终点
- Percent Change From Baseline in Fasting TG at Month 6
研究概览
简要总结
The purpose of the study was to evaluate the efficacy of olezarsen as compared to placebo on the percent change in fasting triglycerides (TG) from baseline.
详细描述
This was a multi-center, double-blind, Phase 3 study in up to 60 patients with FCS. Participants were randomized in a 2:1 ratio to receive Olezarsen or matching placebo in a 53-week treatment period. The length of participation in the study was approximately 74 weeks, which included an up to 8-week screening period, a 53-week treatment period, and a 13-week post-treatment evaluation period. Following the treatment period, eligible patients had the option to enroll in the Open-label Extension (OLE) Study ISIS 678354-CS13 (NCT05130450).
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
- Double (Participant, Investigator)
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
- •A diagnosis of genetically confirmed Familial Chylomicronemia Syndrome (type 1 Hyperlipoproteinemia)
- •Fasting TG ≥ 880 mg/dL (10 millimoles per liter (mmol/L) at Screening
- •History of pancreatitis. Patients without a documented history of pancreatitis are also eligible but their enrollment will be capped at 35%
- •Stable doses of statins, omega-3 fatty acids, fibrates, or other lipid-lowering medications are allowed
排除标准
- •Acute coronary syndrome within 6 months of Screening
- •Major surgery within 3 months of Screening
- •Have any other conditions, which, in the opinion of the Investigator would make the participant unsuitable for inclusion, or could interfere with participating in or completing the study
研究组 & 干预措施
Placebo
Participants received olezarsen-matching placebo, once every 4 weeks by subcutaneous (SC) injection, during Weeks 1 to 49 of the 53-week treatment period.
干预措施: Placebo (Drug)
Olezarsen 50 mg
Participants received olezarsen, 50 milligrams (mg), once every 4 weeks by SC injection, during Weeks 1 to 49 of the 53-week treatment period.
干预措施: Olezarsen (Drug)
Olezarsen 80 mg
Participants received olezarsen 80 mg, once every 4 weeks by SC injection, during Weeks 1 to 49 of the 53-week treatment period.
干预措施: Olezarsen (Drug)
结局指标
主要结局
Percent Change From Baseline in Fasting TG at Month 6
时间窗: Baseline, Month 6
次要结局
- Percent Change From Baseline in Fasting TG at Month 12(Baseline, Month 12)
- Percent Change From Baseline in Fasting Apolipoprotein C-III (apoC-III) at Months 6 and 12(Baseline, Months 6 and 12)
- Percentage of Participants With ≥ 40% Reduction in Fasting TG at Month 6(Month 6)
- Percent Change From Baseline in Fasting Apolipoprotein B-48 (apoB-48) at Months 6 and 12(Baseline, Months 6 and 12)
- Percent Change From Baseline in Fasting Non-High-Density Lipoprotein Cholesterol (Non-HDL-C) at Months 6 and 12(Baseline, Months 6 and 12)
- Adjudicated Acute Pancreatitis Mean Event Rate Per 100 Participant-Years During the Treatment Period (Week 1 Through Week 53) in Participants With Prior History of Pancreatitis(During the treatment period Week 1 through Week 53)
- Adjudicated Acute Pancreatitis Mean Event Rate Per 100 Participant-Years During the Treatment Period (Week 1 Through Week 53)(During the treatment period Week 1 through Week 53)
- Adjudicated Acute Pancreatitis Mean Event Per 100 Participant-Years Rate During Week 13 Through Week 53 in Participants With Prior History of Pancreatitis(Week 13 through Week 53)
- Adjudicated Acute Pancreatitis Mean Event Rate Per 100 Participant-Years During Week 13 to Week 53(Week 13 through Week 53)
- Percentage of Participants With ≥ 70% Reduction in Fasting TG at Month 6(Month 6)
- Percentage of Participants With Fasting TG ≤ 880 mg/dL at Month 6(Month 6)
- Adjudicated Acute Pancreatitis Mean Event Rate Per 100 Participant-Years During Treatment Period in Participants With ≥ 2 Events in 5 Years Prior to Enrollment(During the treatment period Week 1 through Week 53)
- Adjudicated Acute Pancreatitis Mean Event Rate Per 100 Participant-Years From Week 13 to Week 53 in Participants With ≥ 2 Events in 5 Years Prior to Enrollment(Week 13 to Week 53)
- Percentage of Participants With Fasting TG ≤ 500 mg/dL at Month 6(Month 6)