NCT02765841撤回3 期
A Phase 3, Single-arm, Open-label, International, Multi-center Study to Evaluate the Efficacy and Safety of Lomitapide in Pediatric Patients With Homozygous Familial Hypercholesterolemia on Stable Lipid-lowering Therapy
适应症
干预措施
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 撤回
- 主要终点
- Percent change in LDL-C
研究概览
简要总结
This is a Phase 3 single-arm, open-label, international, multi-center clinical trial to evaluate the efficacy and safety of lomitapide in pediatric patients with HoFH who are receiving stable lipid-lowering therapy, including LDL apheresis. The study is comprised of a 12-week Run-in Period, a primary 24-week Efficacy Phase, followed by an 80-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
- 5 Years 至 17 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male or female aged ≥5 and <18 years with diagnosed functional HoFH
- •Patient must weigh at least 15 kg and be at or above the 10th percentile in BMI and at least 10th percentile in height for age and gender based on CDC growth charts
- •Negative pregnancy test at Screening and during the study for females of child bearing age
- •Potentially sexually active female patients who are of child-bearing age must either be sexually abstinent or follow two acceptable methods of contraception
排除标准
- •Other forms of primary hyperlipoproteinemia and secondary causes of hypercholesterolemia (e.g., nephrotic syndrome, hypothyroidism).
- •Abnormal liver function test at Screening
- •Moderate or severe hepatic impairment or active liver disease
- •Serum creatine phosphokinase (CPK) level >2 × ULN.
- •Chronic renal insufficiency
- •History of drug abuse within the last 3 years or habitual alcohol consumption
- •New York Heart Association (NYHA) Class III or IV congestive heart failure.
- •Uncontrolled hypertension
- •In the judgment of the PI, precocious or delayed puberty or endocrine disorder that would affect growth
- •History of non-skin malignancy or other cancers occurring within the past 3 years
- •History of inflammatory bowel disease or other malabsorption syndrome or a history of bowel resection, gastric bypass, or other weight loss surgical procedure.
- •Use of mipomersen within 6 months of Screening.
- •Any medical condition for which the life expectancy is predicted to be less than 5 years.
- •Any patient who is unable to avoid treatment with strong or moderate cytochrome P450 3A4 (CYP3A4) inhibitors, or other drugs contraindicated for use with lomitapide during the study.
- •Participation in an interventional clinical study within 6 weeks for a statin therapy or within 6 months for any other unapproved therapy.
研究组 & 干预措施
Lomitapide
Experimental
干预措施: Lomitapide (Drug)
结局指标
主要结局
Percent change in LDL-C
时间窗: Baseline, Week 24
次要结局
- Percent change in non-HDL-C(Baseline, Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52, 56, 68, 80, 92, 104)
- Percent Change in TC(Baseline, Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52, 56, 68, 80, 92, 104)
- Percent change in TG(Baseline, Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52, 56, 68, 80, 92, 104)
- Percent change in HDL-C(Baseline, Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52, 56, 68, 80, 92, 104)
- Changes in lipid-lowering therapy(Week 24 through Week 104)
- Percent of patients achieving goal LDL-C of <70 mg/dL [1.8 mmol/L]) for patients with documented CVD at Baseline.(Week 24 and through Week 108)
- Changes in laboratory parameters (including hepatic and renal function)(Baseline through Year 2)
- Percent change in VLDL-C(Baseline, Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52, 56, 68, 80, 92, 104)
- Percent change in Lp(a)(Baseline, Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52, 56, 68, 80, 92, 104)
- Percent change in LDL-C(Baseline, Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52, 56, 68, 80, 92, 104)
- Electrocardiogram (ECG) changes(Baseline through Year 2)
- Pulmonary function tests (PFTs)(Baseline through Year 2)
- Percent change in apo B(Baseline, Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52, 56, 68, 80, 92, 104)
- Percent change in apo A-1(Baseline, Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52, 56, 68, 80, 92, 104)
- Changes in LDL apheresis(Week 24 through Week 104)
- Percent of patients achieving goal (LDL-C of <100 mg/dL [2.6 mmol/L] for patients without documented cardiovascular disease [CVD] at Baseline(Week 24 and through Week 108)
- Reported Adverse Events(Baseline through Year 2)
- Height Measurement(Baseline through Year 2)
- Weight Measurement(Baseline through Year 2)
- Body Mass Measurement(Baseline through Year 2)
- Tanner Staging(Baseline through Year 2)
- Percent change in hepatic fat(Baseline through Year 2)
- Blood Pressure(Baseline through Year 2)
- Heart Rate(Baseline through Year 2)
- Temperature(Baseline through Year 2)
- Respiration (breaths/min)(Baseline through Year 2)
- Bone health/age (x-ray of the wrist)(Baseline through Year 2)
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