A Randomized, Double-Blind, Placebo-Controlled, Phase 3 Study of ISIS 678354 Administered Subcutaneously to Patients With Severe Hypertriglyceridemia
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 617
- 试验地点
- 401
- 主要终点
- Percent Change from Baseline in Fasting TG Compared to Placebo
研究概览
简要总结
The purpose of the study is 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 is a multi-center, randomized, double-blind, placebo-controlled study in 617 participants. Participants will be randomized to receive olezarsen or placebo in a 53-week treatment period. The length of participation in the study will be approximately 78 weeks, which includes an up to 12-week screening period, a 53-week treatment period, and a 13-week post-treatment evaluation period or transition to open-label extension (OLE) study with up to 1-year treatment.
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
- Triple (Participant, Care Provider, 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
- •Fasting TG ≥ 500 mg/dL (5.65 mmol/L) at Screening and Qualification
- •Patients must be on lipid-lowering therapy that should adhere to standard of care (SOC) per local guidelines. Lipid-lowering medications should be optimized and stabilized for at least 4 weeks prior to Screening to minimize changes in these medications during the study.
排除标准
- •Hemoglobin A1c (HbA1c) ≥ 9.5% at Screening
- •Alanine aminotransferase or aspartate aminotransferase > 3.0 × upper limit of normal
- •Total bilirubin > upper limit of normal unless due to Gilbert's syndrome
- •Estimated GFR < 30 mL/min/1.73 m^2
研究组 & 干预措施
Olezarsen
Olezarsen will be administered once every 4 weeks by subcutaneous (SC) injection from Week 1 through Week 49.
干预措施: Olezarsen (Drug)
Placebo
Olezarsen-matching placebo will be administered once every 4 weeks by SC injection from Week 1 through Week 49.
干预措施: Placebo (Drug)
结局指标
主要结局
Percent Change from Baseline in Fasting TG Compared to Placebo
时间窗: Baseline and Month 6
次要结局
- Percent Change from Baseline in Fasting TG Compared to Placebo(Baseline and Month 12)
- Percent Change from Baseline in Fasting Apolipoprotein C-III (ApoC-III), Remnant Cholesterol and Fasting Non-High-Density Lipoprotein-Cholesterol (Non-HDL-C) Compared to Placebo(Baseline, Month 6 and 12)
- Proportion of Participants Who Achieve Fasting TG < 880 mg/dL (10 mmol/L) Compared to Placebo in the Subgroup of Participants with Baseline TG ≥ 880 mg/dL(Month 12)
- Adjudicated Acute Pancreatitis Event Rate During the Treatment Period Compared to Placebo(Week 1 through Week 53)
- Proportion of Patients Who Achieve Fasting TG <500 mg/dL (5.65 mmol/L) Compared to Placebo(Month 12)
- Absolute Change in Hepatic Fat Fraction (HFF) Between Olezarsen Treatment Group and Pooled Placebo(Baseline through Month 12)