An Open-Label Safety Study of AKCEA-APOCIII-LRX Administered Subcutaneously to Patients With Familial Chylomicronemia Syndrome (FCS) Previously Treated With Volanesorsen (ISIS 304801)
试验速览
- 阶段
- 3 期
- 状态
- 进行中（未招募）
- 入组人数
- 24
- 试验地点
- 11
- 主要终点
- Proportion of Participants With Decrease in Platelet Count by >30% or >50%, or With Platelet Count Value <50,000/cubic millimeter (mm^3)
研究概览
简要总结
The purpose of the study is to evaluate the safety, tolerability, pharmacokinetic (PK) and pharmacodynamic (PD) effects of olezarsen (formerly known as AKCEA -APOCIII-LRX) in participants with FCS previously treated with volanesorsen.
详细描述
This is a Phase 3, multi-center, open-label safety study in 24 participants with FCS, previously treated with volanesorsen. The study consists of an 8- week screening period, treatment period up to week 209 and a 13-week follow-up period. Participants enrolled will receive olezarsen every 4 weeks during the 209-week Treatment Period.
Treatment period was extended to allow participants to receive olezarsen for an additional 52 weeks for a total of a 209-week treatment period until the drug may be commercially available in the patient's country, or until the Sponsor discontinues the olezarsen development program, whichever occurs earlier.
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
- 未提供
排除标准
- •Treatment with another investigational drug (non-oligonucleotide), biological agent, or device within 4 weeks of Screening, or 5 half-lives of investigational agent, whichever is longer
- •Concomitant medication/procedure restrictions:
- •Systemic corticosteroids or anabolic steroids within 6 weeks prior to Screening and during the study unless approved by the Sponsor Medical Monitor
- •Plasma apheresis within 4 weeks prior to Screening or planned during the study
研究组 & 干预措施
Olezarsen
Olezarsen will be administered once every 4 weeks by subcutaneous (SC) injection for up to 209 weeks.
干预措施: Olezarsen (Drug)
结局指标
主要结局
Proportion of Participants With Decrease in Platelet Count by >30% or >50%, or With Platelet Count Value <50,000/cubic millimeter (mm^3)
时间窗: Baseline to Week 209
Proportion of Participants With Clinical Bleeding Events
时间窗: Baseline to Week 209
Proportion of Participants With Decrease in Estimated Glomerular Filtration Rate (eGFR) by ≥30% or ≥50%
时间窗: Baseline to Week 209
Proportion of Participants With Urine Protein/Creatinine Ratio (UPCR) ≥1000 milligram (mg)/gram (g) or with Urine/Albumin Creatinine Ratio (UACR) ≥500 mg/g
时间窗: Baseline to Week 209
Proportion of Participants With Alanine Aminotransferase (ALT) or Aspartate Aminotransferase (AST) ≥5 x Upper Limit of Normal (ULN)
时间窗: Baseline to Week 209
Proportion of Participants With ALT or AST ≥3 x ULN and Total Bilirubin > 2 x ULN
时间窗: Baseline to Week 209
Proportion of Participants With Total Bilirubin ≥2 mg/deciliter (dL)
时间窗: Baseline to Week 209
次要结局
- Trough (Pre-Dose) Plasma Concentration of Olezarsen(Up to 209 weeks)
- Post-Treatment Plasma Concentration of Olezarsen(Up to 209 weeks)
- Change and Percent Change From Baseline in Fasting Triglycerides (TG)(Baseline to Week 209)
- Change and Percent Change From Baseline in Fasting Apolipoprotein C-III (APOC-III)(Baseline to Week 209)
- Change and Percent Change From Baseline in Fasting Very Low-Density Lipoprotein (VLDL)-C(Baseline to Week 209)
- Change and Percent Change From Baseline in Fasting Chylomicron-TG(Baseline to Week 209)
- Change and Percent Change From Baseline in Fasting Total Cholesterol (TC)(Baseline to Week 209)
- Change and Percent Change From Baseline in Fasting Non-High-Density Lipoprotein (non-HDL)-C(Baseline to Week 209)
- Change and Percent Change From Baseline in Fasting Low-Density Lipoprotein (LDL)-C(Baseline to Week 209)
- Change and Percent Change From Baseline in Fasting Apoprotein B (apoB)(Baseline to Week 209)
- Change and Percent Change From Baseline in Fasting Apoprotein B48 (apoB48)(Baseline to Week 209)
- Change and Percent Change From Baseline in Fasting High-Density Lipoprotein (HDL)-C(Baseline to Week 209)
- Change and Percent Change From Baseline in Fasting Apoprotein A-1 (ApoA-1)(Baseline to Week 209)
- Event Rate of Acute Pancreatitis(Up to 209 weeks)