Randomized, Open-Label, Cross-Over, Phase 3 Study to Evaluate the Efficacy and Safety of LIB003 With Evolocumab in Homozygous Familial Hypercholesterolemia Patients on Stable Lipid-Lowering Therapy
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 发起方
- 入组人数
- 65
- 试验地点
- 12
- 主要终点
- Percent reduction in Low Density Lipoprotein Cholesterol (LDL-C) at week 24
研究概览
简要总结
To compare the safety, tolerability and LDL-C response after 24 Weeks of monthly (every 4 weeks [Q4W]) subcutaneous (SC) dosing of LIB003 300 mg with monthly (Q4W) SC dosing of 420 mg evolocumab (Repatha®) in patients with HoFH on stable diet and oral LDL-C-lowering drug therapy
详细描述
Patients with verified HoFH on stable and continuing doses of oral lipid lowering therapy will be randomized to either evolocumab 420 mg Q4W or LIB003 300 mg Q4W for 24 weeks (Period A). At Week 24, subjects will be crossed over to LIB003 if they were on evolocumab and vice versa for the next 24 weeks (Period B).
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Crossover
- 主要目的
- Treatment
- 盲法
- None
盲法说明
treatment is open label but lipid results are masked to participant, investigator and sponsor
入排标准
- 年龄范围
- 10 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •HoFH diagnosed clinically and confirmed by genotyping
- •Weight of >30 kg and body mass index (BMI) >17 and <40 kg/m2
- •stable diet and lipid-lowering oral therapies for at least 4 weeks
排除标准
- •mipomersen within 6 months of screening;
- •LDL or plasma apheresis <2 months prior to randomization
- •history of non-response to PCSK9 mAb or presence of receptor negative/null LDLR activity expected to result in non-response to PCSK9 inhibition
- •prior or active clinical condition or acute and/or unstable systemic disease compromising subject inclusion
研究组 & 干预措施
LIB003 (lerodalcibep)
300 mg SC Q4W
干预措施: lerodalcibep (Drug)
evolocumab
420 mg SC Q4W
干预措施: evolocumab (Drug)
结局指标
主要结局
Percent reduction in Low Density Lipoprotein Cholesterol (LDL-C) at week 24
时间窗: baseline to 24 weeks on each treatment
Change in serum LDL-C from baseline after 24 weeks
次要结局
- The incidence and severity of treatment emergent adverse events (TEAEs)(baseline to 24 weeks on each treatment)