A Randomized, Double-blind, Placebo-controlled, Multicenter Trial Assessing the Reduction of the Rate of Lipoprotein Apheresis After Treatment With Pelacarsen (TQJ230) Compared to Placebo in Patients With Hyperlipoproteinemia(a) and Established Cardiovascular Disease Undergoing Weekly Lipoprotein Apheresis in Germany
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 51
- 试验地点
- 14
- 主要终点
- Rate of lipoprotein apheresis sessions performed over 52 weeks normalized to the weekly lipoprotein apheresis schedule
研究概览
简要总结
Phase III study to test the hypothesis that treatment with pelacarsen (TQJ230) 80 mg Q4W compared to placebo significantly reduces the rate of lipoprotein apheresis in patients with hyperlipoproteinemia (a) and established cardiovascular disease currently undergoing lipoprotein apheresis in Germany on a weekly schedule.
详细描述
Lipoprotein apheresis to date is the only approved therapeutic option for cardiovascular (CV) risk reduction in patients with severely elevated Lp(a) levels in Germany. Lipoprotein apheresis is an expensive, burdensome, and time-consuming procedure. The current study (CTQJ230A12302) investigated if treatment with pelacarsen (TQJ230) 80 mg Q4W vs placebo reduces the rate of lipoprotein apheresis in patients with hyperlipoproteinemia(a) and established CV disease
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
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 18 Years 至 80 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients currently undergoing lipoprotein apheresis for isolated Lp(a) on a weekly schedule in Germany for ≥ 12 months prior to screening with at least 40 sessions within the past 52 weeks prior to randomization
- •Lipoprotein(a) (Lp(a))> 60 mg/dL at screening
- •Spontaneous prior myocardial infarction (MI): ≥ 3 months from screening visit to ≤ 10 years prior to the screening visit, and/or
- •Ischemic stroke: ≥ 3 months from screening visit to ≤ 10 years prior to the screening visit, and/or
- •Clinically significant symptomatic peripheral artery disease (PAD)
- •Clinically significant symptomatic coronary artery disease (PAD)
排除标准
- •Uncontrolled hypertension
- •Heart failure New York Heart Association (NYHA) class IV
- •History of malignancy of any organ system
- •History of hemorrhagic stroke or other major bleeding
- •Platelet count <140,000 per mm3 at screening
- •Active liver disease or hepatic dysfunction
- •Significant kidney disease
- •Pregnant or nursing women
研究组 & 干预措施
Pelacarsen (TQJ230)
Pelacarsen (TQJ230) 80 mg s.c. Q4W
干预措施: Pelacarsen (TQJ230) 80 mg s.c. (Drug)
Placebo
Placebo to Pelacarsen s.c. Q4W
干预措施: Corresponding Placebo (Drug)
结局指标
主要结局
Rate of lipoprotein apheresis sessions performed over 52 weeks normalized to the weekly lipoprotein apheresis schedule
时间窗: Over 52 Weeks
Demonstrate superiority of pelacarsen (TQJ230) compared to placebo in reducing the rate of lipoprotein apheresis sessions in patients with hyperlipoproteinemia(a) and established CVD during 52 weeks of treatment
Rate of Lipoprotein Apheresis Sessions Performed Over 52 Weeks Normalized to the Weekly Lipoprotein Apheresis Schedule
时间窗: Up to Week 52
Rate (proportion) of apheresis sessions was calculated as the number of actual lipoprotein apheresis (LA) sessions received, divided by the number of planned LA sessions during the 52-week period, which is 52 for patients who completed all study visits, or pro-rated for those who discontinued early. This rate could range from 0 to 1, with 0 indicating that the patient had skipped all planned LA sessions, and 1 indicating that the patient had received all planned sessions. Multiple imputation for missing Lp(a) data was performed, and missing apheresis data was imputed.
次要结局
- Time to lipoprotein apheresis avoidance (where lipoprotein apheresis avoidance is defined as at least 24 weeks of no lipoprotein apheresis until end of study)(At least 24 weeks up to Week 52)
- Total avoidance of lipoprotein apheresis from week 12 to week 52(Week 12 to Week 52)
- Change from baseline to week 52 in the log-transformed Lp(a) (measured prior to planned lipoprotein apheresis)(52 weeks)
- Time to Lipoprotein Apheresis Avoidance(From randomization up to Week 52)
- Number of Participants With Total Lipoprotein Apheresis Avoidance From Week 12 to Week 52(Week 12 up to Week 52)
- Change From Baseline to Week 52 in the Log-transformed Lp(a) Reported as mg/dL(Baseline, week 52)
- Change From Baseline to Week 52 in the Log-transformed Lp(a) Reported as Nmol/L(Baseline, week 52)