NCT02173158已完成3 期
A Phase 3, Single-Arm, Open-Label, Multicenter Study to Evaluate the Efficacy and Safety of Lomitapide in Japanese Patients With Homozygous Familial Hypercholesterolemia (HoFH) on Concurrent Lipid-Lowering Therapy
适应症
干预措施
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 9
- 主要终点
- Percent Change in LDL-C
研究概览
简要总结
Study to Evaluate the Efficacy and Safety of Lomitapide in Japanese Patients with Homozygous Familial Hypercholesterolemia (HoFH) on Concurrent Lipid-Lowering Therapy.
详细描述
This is a Phase 3, single-arm, open-label, multicenter clinical trial to evaluate both the efficacy and long-term safety of lomitapide in Japanese patients with HoFH receiving maximally-tolerated, stable lipid-lowering therapy. This study is comprised of a run-in period, a primary 26-week Efficacy Phase, and a 30-week Safety Phase.
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
- •Japanese male and female patients aged ≥ 18 years of age who are receiving maximally tolerated, stable, lipid-lowering therapy
- •Diagnosis of functional HoFH
- •Body weight ≥ 40 kg and < 136 kg
- •Negative pregnancy test at screening
排除标准
- •Uncontrolled hypertension
- •History of chronic renal insufficiency
- •History of biopsy proven cirrhosis or abnormal liver function tests (LFTs) at screening
- •Any major surgical procedure occurring < 3 months prior to the screening visit
- •Cardiac insufficiency
- •Previous organ transplantation
- •History of a non-skin malignancy within the previous 3 years
- •Patients who are not able to limit their alcohol intake
- •Participation in an investigational drug study within 6 weeks prior to the screening visit
- •Known significant gastrointestinal bowel disease
- •Nursing mothers
- •Serious or unstable medical or psychological conditions
- •Requirement for certain prohibited medications known to be potentially hepatotoxic
- •Use of strong or moderate inhibitors of CYP3A4
- •Use of simvastatin at doses >10 mg per day
- •Documented diagnosis of any liver disease
研究组 & 干预措施
lomitapide
Experimental
Maximum tolerated dose of lomitapide (up to 60mg/day) in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis.
干预措施: lomitapide (Drug)
结局指标
主要结局
Percent Change in LDL-C
时间窗: Baseline to Week 26
Mean percent change from baseline
次要结局
- Change in HDL-C(Baseline to Week 56)
- Change in Apo AI(Baseline to Week 56)
- Change in Apo B(Baseline to Week 56)
- Change in Triglycerides(Baseline to Week 56)
- Change in Non-HDL-C(Baseline to Week 56)
- Change in Total Cholesterol(Baseline to Week 56)
- Change in VLDL-C(Baseline to Week 56)
- Change in Lp(a)(Baseline to Week 56)
- Change in LDL-C(Baseline to Week 56)
研究者
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