ISIS 678354-CS7: An Open-Label 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
- 招募中
- 入组人数
- 1
- 试验地点
- 1
- 主要终点
- 1. Safety endpoints include a proportion of patients who show the following changes from Baseline to Week 53, Week 105, Week 157 and Week 209 :-Decrease in platelet (PLT) count by>30% -Decrease in PLT count by>50% - PLT count value<50,000mm3 -Major bleeding events -Clinically relevant non-major bleeding events -Decrease in eGFR by>30% -Decrease in eGFR by>50% - UPCR≥1000mg/g -UACR≥500mg/g- ALT or AST>5×ULN- Total bilirubin>2.0mg/dL- ALT or AST>3×ULN and total bilirubin≥2xULN
研究概览
简要总结
- To evaluate the safety of ISIS 678354 in patients with FCS previously treated with volanesorsen
- To evaluate the tolerability of ISIS 678354 in patients with FCS previously treated with volanesorsen
入排标准
- 年龄范围
- 18 years 至 65+ years（65+ Years, 18-64 Years）
- 接受健康志愿者
- 是
入选标准
- •Patients with FCS (clinical or genetic diagnosis) currently on or previously treated with volanesorsen (ISIS 304801) Study participants in countries where Waylivra is commercially approved and available for patients should not be deprived of the treatment option with Waylivra. Participation in this study for such patients will only be allowed when Waylivra was discontinued due to AEs
- •Other protocol-defined inclusion criteria apply.
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. Have any other conditions including significant medical history which, in the opinion of the Investigator would make the patient unsuitable for inclusion, or could interfere with the patient participating in or completing the Study
结局指标
主要结局
1. Safety endpoints include a proportion of patients who show the following changes from Baseline to Week 53, Week 105, Week 157 and Week 209 :-Decrease in platelet (PLT) count by>30% -Decrease in PLT count by>50% - PLT count value<50,000mm3 -Major bleeding events -Clinically relevant non-major bleeding events -Decrease in eGFR by>30% -Decrease in eGFR by>50% - UPCR≥1000mg/g -UACR≥500mg/g- ALT or AST>5×ULN- Total bilirubin>2.0mg/dL- ALT or AST>3×ULN and total bilirubin≥2xULN
1. Safety endpoints include a proportion of patients who show the following changes from Baseline to Week 53, Week 105, Week 157 and Week 209 :-Decrease in platelet (PLT) count by>30% -Decrease in PLT count by>50% - PLT count value<50,000mm3 -Major bleeding events -Clinically relevant non-major bleeding events -Decrease in eGFR by>30% -Decrease in eGFR by>50% - UPCR≥1000mg/g -UACR≥500mg/g- ALT or AST>5×ULN- Total bilirubin>2.0mg/dL- ALT or AST>3×ULN and total bilirubin≥2xULN
2. Tolerability assessments will include adverse events (AEs), clinical laboratory test, and use of concomitant medications
2. Tolerability assessments will include adverse events (AEs), clinical laboratory test, and use of concomitant medications
次要结局
- 1. PK assessments include determination of trough (pre-dose) and post-treatment plasma ISIS 678354 concentrations
- 2. PD endpoints include: 1. Change and percent change from Baseline to Week 53, Week 105, Week 157 and Week 209 in fasting: - Triglycerides (TG) - APOC-III, very low-density lipoprotein (VLDL)-C, chylomicron-TG, total cholesterol (TC), non-HDL-C, low-density lipoprotein (LDL)-C, apoB, apoB48, HDL-C, ApoA-1 2. Event rate of acute pancreatitis (Week 1 through Weeks: 53, 105, and 157)
研究者
Global Regulatory Affairs
Scientific
Ionis Pharmaceuticals Inc.