NCT03455777撤回2 期
A Phase 2 Open-Label Study to Assess the Pharmacodynamics, Pharmacokinetics, Safety and Tolerability of ISIS 703802 (AKCEA-ANGPTL3-LRx) Administered Subcutaneously to Patients With Homozygous Familial Hypercholesterolemia (HoFH)
适应症
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 撤回
- 试验地点
- 1
- 主要终点
- Reduction of low density lipoprotein cholesterol (LDL-C).
研究概览
简要总结
This is a single center, open-label study to evaluate the efficacy of AKCEA-ANGPTL3- LRX for reduction of low density lipoprotein cholesterol (LDL-C) levels in patients with Homozygous Familial Hypercholesterolemia (HoFH).
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
- •Body mass index (BMI) ≤ 35 kg/m2,
- •Genetic confirmation of two mutant alleles at the LDLR, APOB, PCSK9, or LDLRAP1 gene locus OR an untreated LDL-C > 500 mg/dL (13 mmol/L) or treated LDL-C ≥ 300 mg/dL (2.59 mmol/L) together with either cutaneous or tendon xanthoma before age 10 years OR familial medical history of genetically confirmed heterozygous FH in both parents OR untreated elevated LDL-C and TC > 250 mg/dL consistent with the disease,
- •Patients must be on stable LDL-C lowering agents or on regular apheresis
排除标准
- •Myocardial infarction, percutaneous transluminal coronary intervention, or coronary artery bypass graft surgery within 12 weeks prior to Screening, or cerebrovascular accident within 24 weeks prior to Screening.
- •Diabetes mellitus if newly diagnosed or if HbA1c ≥ 9.0%
研究组 & 干预措施
AKCEA-ANGPTL3-LRX Dose 1
Experimental
干预措施: AKCEA-ANGPTL3-LRX (Drug)
结局指标
主要结局
Reduction of low density lipoprotein cholesterol (LDL-C).
时间窗: 14 weeks
Percent change in LDL-C from Baseline to week 14.
次要结局
- Effect of ISIS 703802 on plasma angiopoietin like 3 (ANGPTL3).(7 and 14 Weeks)
- Effect of ISIS 703802 on lipid parameters.(7 and 14 Weeks)
- Evaluate plasma trough levels of ISIS 703802.(14 Weeks)
- The safety of ISIS 703802 by the incidence of treatment-emergent adverse events(14 Weeks)
研究者
研究点 (1)
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