An Open-Label Extension Study of Olezarsen (ISIS 678354) Administered Subcutaneously to Patients with Severe Hypertriglyceridemia (SHTG)
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 入组人数
- 603
- 试验地点
- 137
- 主要终点
- Proportion of Patients With Change in Laboratory Values From Baseline to Week 53, and from Baseline to Week 105, and from Baseline to Week 157. Values include platelet count value <50,000 mm^3, Clinical bleeding events, decrease in eGFR by 30%, decrease in eGFR by ≥ 50%, urine protein/creatinine ratio (UPCR) ≥1000 mg/g, urine/albumin creatinine ratio (UACR) ≥500 mg/g, ALT or AST ≥5 × upper limit of normal (ULN), total bilirubin ≥2.0 mg/dL, and ALT or AST ≥3 × ULN and total bilirubin ≥2 × ULN.
研究概览
简要总结
To evaluate the safety and tolerability of olezarsen
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
- •Satisfactory completion of either ISIS 678354-CS5 or ISIS 678354-CS6 with an acceptable safety profile, per Investigator judgment. Satisfactory completion requires that the patient did not terminate early from treatment.
- •Participants must be on a stable regimen of lipid-lowering therapy that should adhere to standard of care (SOC) per local guidelines. A patient may be eligible if their regimen of lipid-lowering therapy has been changed recently, provided that lipid lowering medications are optimized and stabilized for at least 4 weeks prior to the end of the Qualification Period of this OLE study; ideally, such changes are initiated after Week 53 of the index study (ISIS 678354-CS5 or -CS6).
- •A patient may re-enroll in the study after having completed the study when the Treatment Period was 53 weeks, if the patient satisfactorily completed the study with an acceptable safety profile, per Investigator judgment. For a given patient to re-enroll, there is no maximum time between their last dose of olezarsen when initially enrolled and their first dose of olezarsen when re-enrolled (i.e., not exceeding 17 weeks between doses does not apply when re-enrolling).
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 participant participating in or completing the study, including need for treatment with disallowed medications, or need to change the required stable regimen as per either ISIS 678354-CS5 or ISIS 678354-CS6 study entry criteria. Although, it is acceptable to adjust a regimen of lipid-lowering therapy as described in Inclusion Criterion 3, and it is acceptable to adjust or stop lipid-lowering therapy at or after Week 13 per Investigator judgment.
结局指标
主要结局
Proportion of Patients With Change in Laboratory Values From Baseline to Week 53, and from Baseline to Week 105, and from Baseline to Week 157. Values include platelet count value <50,000 mm^3, Clinical bleeding events, decrease in eGFR by 30%, decrease in eGFR by ≥ 50%, urine protein/creatinine ratio (UPCR) ≥1000 mg/g, urine/albumin creatinine ratio (UACR) ≥500 mg/g, ALT or AST ≥5 × upper limit of normal (ULN), total bilirubin ≥2.0 mg/dL, and ALT or AST ≥3 × ULN and total bilirubin ≥2 × ULN.
Proportion of Patients With Change in Laboratory Values From Baseline to Week 53, and from Baseline to Week 105, and from Baseline to Week 157. Values include platelet count value <50,000 mm^3, Clinical bleeding events, decrease in eGFR by 30%, decrease in eGFR by ≥ 50%, urine protein/creatinine ratio (UPCR) ≥1000 mg/g, urine/albumin creatinine ratio (UACR) ≥500 mg/g, ALT or AST ≥5 × upper limit of normal (ULN), total bilirubin ≥2.0 mg/dL, and ALT or AST ≥3 × ULN and total bilirubin ≥2 × ULN.
Proportion of Participants Who Experience Adverse Events (AEs) [Time Frame: Up to 170 weeks]
Proportion of Participants Who Experience Adverse Events (AEs) [Time Frame: Up to 170 weeks]
Proportion of Participants Who Use Concomitant Medications [Time Frame: Up to 170 weeks]
Proportion of Participants Who Use Concomitant Medications [Time Frame: Up to 170 weeks]
次要结局
未报告次要终点
研究者
Global Regulatory Affairs
Scientific
Ionis Pharmaceuticals Inc.