NCT06712771进行中（未招募）3 期
A Phase 3 Clinical Trial to Evaluate the Efficacy and Safety of VSA003 Injection in Chinese Adolescents and Adults With Homozygous Familial Hypercholesterolaemia (HoFH)
适应症
干预措施
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 进行中（未招募）
- 入组人数
- 46
- 试验地点
- 1
- 主要终点
- Percent change from baseline in serum LDL-C at month 6
研究概览
简要总结
Significant reduction in ANGPLT3 levels is expected to reduce plasma LDL-C and TG levels and may lead to the reduction of risk of coronary heart disease and cardiovascular events associated with hypertriglyceridemia and LDL-C in subjects with persistent dyslipidemia. In this clinical study, the efficacy and safety of VSA003, an RNAi-based therapeutic targeting ANGPTL3, will be evaluated in subjects with HoFH.
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
- 12 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male or female aged ≥ 12 years and weighing ≥ 40 kg at the time of ICF signature;
- •Patients with HoFH who meet genetic or clinical diagnosis;
- •Willing to follow a daily low-fat diet for the duration of the study;
- •Receiving stable and tolerable lipid-lowering therapy prior to LDL-C testing during the screening period;
- •Fasting LDL-C ≥ 2.6 mmol/L.
排除标准
- •Have received or are receiving targeted ANGPTL3 treatment within 5 months or 5 half-lives, whichever is longer, prior to screening;
- •Fasting TG ≥ 4.5 mmol/L at screening;
- •Presence of uncontrolled endocrine disease affecting lipids or lipoproteins;
- •Weight change of more than 10% in the 4 weeks prior to randomisation;
- •Initiation of a new dietary plan or significant differences from previous dietary structure and habits within 4 weeks prior to screening;
- •Women who are pregnant (including planned pregnancies) or breastfeeding;
- •Refusal to limit alcohol consumption to moderate limits and below during the study period, specifically no more than 14 units per week;
- •Uncontrolled hypertension (blood pressure > 160/100 mmHg at screening);
- •New York Heart Association (NYHA) class IV heart failure or left ventricular ejection fraction <30% within 12 months prior to screening;
- •History of major surgery within 12 weeks prior to baseline, or planning to undergo major surgery during the study;
- •Known allergy to any component of the study drug.
研究组 & 干预措施
VSA003
Experimental
subcutaneous injections
干预措施: VSA003 (Drug)
Placebo
Placebo Comparator
subcutaneous injections
干预措施: Placebo (Drug)
结局指标
主要结局
Percent change from baseline in serum LDL-C at month 6
时间窗: At month 6
次要结局
- Percent change and absolute change in serum ANGPTL3 levels from baseline at month 6(At month 6)
- Percent change and absolute change in serum LDL-C, ApoB, non-HDL-C, VLDL-C, TC and TG levels from baseline(Up to month 6, Up to month 18)
- Percent change and absolute change in serum HDL-C, ApoC-III, ApoC-II, ApoA-I, ApoA-V and Lp[a] levels from baseline(Up to month 6, Up to month 18)
- Percentage of patients achieving ≥30% reduction in serum LDL-C from baseline(At month 6, At month 18)
- Percentage of patients achieving ≥50% reduction in serum LDL-C from baseline(At month 6, At month 18)
- Number of Participants with Adverse Events(Up to month 6, Up to month 18)
- Number of Participants with Anti-Drug Antibodies (ADAs) to VSA003 Over Time(Up to month 6, Up to month 18)
- Correlation between VSA003 serum concentrations and QT intervals(Up to month 6, Up to month 18)
研究者
研究点 (1)
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