NCT03933293已完成2 期
A Phase 2 Study to Evaluate the Safety and Efficacy of PCSK9 Inhibitor AK102 in Patients With Homozygous Familial Hypercholesterolemia (HoFH)
Akeso2 个研究点 分布在 1 个国家目标入组 10 人开始时间: 2019年5月13日最近更新:
适应症
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- 入组人数
- 10
- 试验地点
- 2
- 主要终点
- Incidence of treatment-emergent adverse events as assessed by CTCAE V5.0（only for part 1）
研究概览
简要总结
AK102 is being developed for the treatment of HoFH. The study will be conducted in 2 parts, part 1 is open label, single arm study to evaluate the safety, tolerability and efficacy of PCSK9 inhibitor AK102, and part 2 is double blind, randomized, placebo controlled study to evaluate the efficacy and safety of PCSK9 inhibitor AK102. The treatment period will last 12 week.
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 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Males and females, ≥18 years of age with a diagnosis of homozygous familial hypercholesterolemia by genetic confirmation or a clinical diagnosis based on a history of an untreated low-density lipoprotein cholesterol (LDL-C) concentration >500 mg/deciliter (dL) [13 millimoles/liter (mmol/L)] together with either xanthoma before 10 years of age or evidence of heterozygous familial hypercholesterolemia in both parents
- •Stable on pre-existing, lipid-lowering therapies (statins in combination with ezetimibe) for at least 4 weeks with no planned medication or dose change for the duration of study participation
- •Fasting central lab LDL-C concentration >130 mg/dL (3.4 mmol/L) and triglyceride concentration <400 mg/dL (4.5 mmol/L).
- •Body weight of 40 kilograms (kg) or greater at screening
排除标准
- •Received LDL plasma replacement therapy within 8 weeks before Investigational product administration
- •Received Lomitapide or Mipomersen within 5 months before Investigational product administration
- •Received prior treatment with PCSK9 inhibitors or AK
- •Unexplained creatine kinase (CK) ≥ 5 times the upper limit of normal (ULN)
- •Subjects with untreated chronic hepatitis B or chronic hepatitis B virus (HBV) DNA exceeding 500 IU/ mL or active hepatitis C virus (HCV) should be excluded. Subjects with non-active HBsAg carriers, treated and stable hepatitis B (HBV DNA <500 IU/ mL) , and cured hepatitis C can be enrolled. Subjects with positive HCV antibodies are eligible only if the HCV RNA test results are negative.
- •Known allergic reactions to any ingredients of AK102
- •Any other condition(s) that would compromise the safety of the patient or compromise the quality of the clinical study as judged by the Investigator and/or Medical Monitor.
研究组 & 干预措施
AK102
Experimental
450mg AK102, Q4W, subcutaneous injection
干预措施: Statins (Drug)
AK102
Experimental
450mg AK102, Q4W, subcutaneous injection
干预措施: AK102 (Drug)
AK102
Experimental
450mg AK102, Q4W, subcutaneous injection
干预措施: Ezetimibe (Drug)
placebo
Placebo Comparator
Placebo, Q4W, subcutaneous injection
干预措施: Statins (Drug)
placebo
Placebo Comparator
Placebo, Q4W, subcutaneous injection
干预措施: Ezetimibe (Drug)
结局指标
主要结局
Incidence of treatment-emergent adverse events as assessed by CTCAE V5.0（only for part 1）
时间窗: From baseline through 12 weeks
Percent Change From Baseline in Low-Density Lipoprotein Cholesterol (LDL-C) at Week 12
时间窗: Week 12
次要结局
- Percent Change From Baseline in High-density lipoprotein (HDL) cholesterol(From baseline through 12 weeks)
- Number of subjects who develop detectable anti-drug antibodies (ADAs)(From baseline through 12 weeks)
- Percent Change From Baseline in non High-density lipoprotein (non-HDL) cholesterol(From baseline through 12 weeks)
- Change From Baseline in Proprotein Convertase Subtilisin/Kexin Type 9 (PCSK9)(From baseline through 12 weeks)
- Percent Change From Baseline in Serum Triglyceride (TG)(From baseline through 12 weeks)
- Percent Change From Baseline in Apolipoprotein B (Apo B)(From baseline through 12 weeks)
- Percent Change From Baseline in Apolipoprotein A-I (Apo A-I)(From baseline through 12 weeks)
- Percent Change From Baseline in Total Cholesterol(TC)(From baseline through 12 weeks)
- Concentrations of AK102 in Serum(Part 1: Day 1,Day 2, Day 4, Day 8, Day 15, Day 22, D29, D57. Part 2: Day 1, Day 29, Day 57)
研究者
研究点 (2)
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