NCT05130450进行中（未招募）3 期
An Open-Label Extension Study of AKCEA-APOCIII-LRx 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
- 进行中（未招募）
- 入组人数
- 60
- 试验地点
- 27
- 主要终点
- Percent Change From Baseline in Fasting TG at 6 Months (Average of Weeks 23, 25, and 27) Compared to Baseline
研究概览
简要总结
The purpose of this study is to evaluate the effect of olezarsen (formerly known as AKCEA-APOCIII-LRx) on the percent change in fasting triglycerides (TG) from baseline.
详细描述
This is a multi-center, open-label extension (OLE) study of up to 60 participants with FCS rolling-over from Study ISIS 678354-CS3 (NCT04568434). Participants will receive olezarsen during a 157-week treatment period, followed by a 13-week post-treatment follow-up period. The length of participation in this study is approximately 201 weeks, which includes an up to 31-day qualification period, a 157-week treatment period, and a 13-week post-treatment evaluation period.
Treatment has been extended to obtain additional safety assessments and efficacy data and to provide patients with continued access to ISIS 678354 until the drug may be available commercially.
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
- •Satisfactory completion of treatment with olezarsen in the index study (ISIS 678354-CS3, last dose as scheduled at Week 49) with an acceptable safety profile, per Investigator judgement.
排除标准
- •Have any new condition or worsening of existing condition which in the opinion of the Investigator would make the participant unsuitable for enrollment, or could interfere with the patient participating in or completing the study, including need for treatment with medications disallowed in the index study (ISIS 678354-CS3).
研究组 & 干预措施
Olezarsen
Experimental
Olezarsen will be administered once every 4 weeks by subcutaneous (SC) injection from Week 1 through Week 153.
干预措施: Olezarsen (Drug)
结局指标
主要结局
Percent Change From Baseline in Fasting TG at 6 Months (Average of Weeks 23, 25, and 27) Compared to Baseline
时间窗: Baseline and 6 months
次要结局
- Adjudicated Acute Pancreatitis Event Rate During the Treatment Period(Week 1 through Weeks 53, 105, or 157)
- Percent Change in Fasting non-HDL-C from Months 12, 24, 36(At 12, 24, 36 months)
- Adjudicated Acute Pancreatitis Event Rate During the Treatment Period in Participants With ≥ 2 Events of Adjudicated Acute Pancreatitis in 5 Years Prior to Treatment With Study Drug in the Index Study(Week 1 through Week 157)
- Percent Change From Baseline in Fasting Apolipoprotein 48 (apoB48) at 6 Months(At 6 months)
- Percentage of Participants Who Achieve Fasting TG ≤ 880 mg/dL at 12, 24, 36 months(At 12, 24, 36 months)
- Percentage of Participants Who Achieve ≥ 70% Reduction in Fasting TG From Baseline at 6 Months(At 6 months)
- Adjudicated Acute Pancreatitis Event Rate During the Treatment Period in Patients with a Prior History of Pancreatitis within 10 Years Prior to Screening in the Index Study(Week 1 through Weeks 53, 105, or 157)
- Percentage of Participants Who Achieve Fasting TG ≤ 500 mg/dL at 12, 24, 36 Months(At 12, 24, 36 months)
- Percent Change in Fasting apoC-III from Baseline at Month 6(Baseline and 6 months)
- Percentage of Participants Who Achieve Fasting TG ≤ 880 milligrams per deciliter (mg/dL) at 6 Months(At 6 months)
- Percent Change From Baseline in Fasting apoB48 at 12, 24, 36 Months(At 12, 24, 36 months)
- Percentage of Participants Who Achieve ≥ 70% Reduction in Fasting TG From Baseline at 12, 24, 36 Months(At 12, 24, 36 months)
- Percentage of Participants Who Achieve Fasting TG ≤ 500 mg/dL at 6 Months(At 6 months)
- Percent Change From Baseline in Fasting TG at 12 Months (Average of Weeks 51 and 53) at 24 Months (Average of Week 103 and Week 105) and 36 Months (Average of Week 155 and Week 157)(Baseline and 36 months)
- Percentage of Participants Who Achieve ≥ 40% Reduction in Fasting TG From Baseline at 6 Months(At 6 months)
- Percentage of Participants Who Achieve ≥ 40% Reduction in Fasting TG From Baseline at 12, 24, 36 Months(At 12, 24, 36 months)
- Percent Change in Fasting apoC-III from Month 12, 24, 36(At 12, 24, 36 months)
- Percent Change in Fasting non-HDL-C from Baseline at Month 6(Baseline and 6 months)
研究者
研究点 (27)
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