Evaluation of the Effect of Lomitapide Treatment on Major Adverse Cardiovascular Events (MACE) in Patients With Homozygous Familial Hypercholesterolemia: A Multicenter, Retrospective and Prospective Observational Study
试验速览
- 阶段
- 不适用
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 73
- 试验地点
- 30
- 主要终点
- Incidence of Major Adverse Cardiovascular Events (MACE) Before and After Lomitapide Treatment
研究概览
简要总结
This observational, multicenter, retrospective and prospective study aims to evaluate the effect of lomitapide treatment on Major Adverse Cardiovascular Events (MACE) in patients with Homozygous Familial Hypercholesterolemia (HoFH).
HoFH is a rare genetic disorder characterized by extremely high levels of LDL cholesterol (LDL-C), leading to an increased risk of early cardiovascular diseases. Lomitapide is an approved medication that lowers LDL-C levels by inhibiting microsomal triglyceride transfer protein (MTP).
The study will collect data from patients who have been treated with lomitapide for at least 12 months and will compare the incidence of MACE during the first three years of treatment with the three years before treatment initiation. The study includes data collection from multiple lipid centers across Europe.
The primary objective is to assess the impact of lomitapide on MACE, while secondary objectives include evaluating changes in lipid profiles, liver function tests, and lipid-lowering treatments.
详细描述
This is a multicenter, international, long-term observational study investigating the real-world impact of lomitapide on Major Adverse Cardiovascular Events (MACE) in patients with Homozygous Familial Hypercholesterolemia (HoFH).
Study Design:
Observational, open-label, retrospective and prospective study Data will be collected from 30 lipid centers across Europe Patients will serve as their own control, with comparisons between pre-treatment (3 years before lomitapide) and post-treatment (first 3 years of lomitapide therapy) periods
Study Population:
Approximately 72 adult patients (≥18 years) diagnosed with HoFH Patients must have received lomitapide for at least 12 months Availability of 3 years of pre-treatment clinical records
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Other
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
- •Adult patients (age ≥18 years)
- •Clinical or genetic diagnosis of HoFH
- •Treated with lomitapide at any dosage
- •On treatment with lomitapide for at least 12 months at the time of enrollment
- •Availability of 3 years of medical records prior to lomitapide treatment to confirm MACE
- •Giving written informed consent
排除标准
- •Patients who were prescribed lomitapide outside of the marketing authorization or in contraindicated patients
- •Patients receiving lomitapide in clinical trials
- •Patients receiving an investigational agent, defined as any drug or biologic agent other than lomitapide that has not received Market Authorization in the country of participation, at the time of enrolment
研究组 & 干预措施
HoFH Patients Treated with Lomitapide
This cohort consists of adult patients (≥18 years) diagnosed with Homozygous Familial Hypercholesterolemia (HoFH) who have been treated with lomitapide for at least 12 months. The study will retrospectively analyze the incidence of Major Adverse Cardiovascular Events (MACE) in the three years before lomitapide treatment, and prospectively track MACE incidence during the first three years of lomitapide therapy.
结局指标
主要结局
Incidence of Major Adverse Cardiovascular Events (MACE) Before and After Lomitapide Treatment
时间窗: 3 years before treatment vs. 3 years during treatment
This measure will assess the incidence of MACE (including myocardial infarction, stroke, cardiovascular death, and hospitalization due to unstable angina) during the first three years of lomitapide treatment compared to the three years prior to treatment initiation. Unit of Measure: Number of events per 100 patient-years.
次要结局
- Change in LDL-Cholesterol Levels(Baseline, 1 year, 2 years, 3 years)
- Change in Total Cholesterol, Triglycerides, and HDL-Cholesterol(Baseline, 1 year, 2 years, 3 years)
- Change in Triglyceride Levels(Baseline, 1 year, 2 years, 3 years.)
- Change in HDL-Cholesterol Levels(Baseline, 1 year, 2 years, 3 years)
- Liver Safety Profile - ALT Levels(Baseline, 1 year, 2 years, 3 years.)
- Liver Safety Profile - AST Levels(Baseline, 1 year, 2 years, 3 years.)
- Liver Safety Profile - GGT Levels(Baseline, 1 year, 2 years, 3 years.)
- Lipid-Lowering Therapy Modifications(Baseline, 1 year, 2 years, 3 years.)