An Open-label, Single Arm, Multicenter Extension Study to Evaluate Long-term Safety and Tolerability of Inclisiran in Participants With Heterozygous or Homozygous Familial Hypercholesterolemia Who Have Completed the Pediatric ORION-16, ORION-13, ORION-20 or ORION-19 Studies (VICTORION-PEDS-OLE)
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 入组人数
- 195
- 试验地点
- 58
- 主要终点
- Number of participants with treatment-emergent adverse events (TEAEs) and serious adverse events (TESAEs)
研究概览
简要总结
The purpose of this open-label, single arm, multicenter extension study is to evaluate the long-term safety and tolerability of inclisiran in participants with HeFH or HoFH who have completed the ORION-16 (CKJX839C12301), ORION-13 (CKJX839C12302), ORION-20 (CKJX839C12303) or ORION-19 (CKJX839C12304) studies.
详细描述
This is an open-label, single arm, multicenter study designed to evaluate long-term safety and tolerability of inclisiran. In addition, the study will provide participants the opportunity to have continued access to treatment with inclisiran.
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
- 12 Years 至 100 Years（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Key inclusion:
- •Male and female participants with a diagnosis of HeFH or HoFH who completed the ORION-16, ORION-13, ORION-20 or ORION-19 studies
- •Per investigator's clinical judgment, participant derived benefit from treatment with inclisiran in the ORION-16, ORION-13, ORION-20 or ORION-19 studies
排除标准
- •Participants who in the feeder ORION-16, ORION-13, ORION-20, or ORION-19 studies either screen failed or permanently discontinued from the treatment/study for any reason or had serious safety or tolerability issues related to inclisiran treatment
- •Any uncontrolled or serious disease, or any medical, physical, or surgical condition, that may either interfere with participation in the clinical study or interpretation of clinical study results, and/or put the participant at significant risk
研究组 & 干预措施
Inclisiran
Participants who have completed ORION-16 or ORION-13 receive inclisiran sodium 300mg (equivalent to 284mg inclisiran*) in 1.5mL solution.
Participants who have completed ORION-19 and ORION-20 receive Inclisiran dose levels based on weight.
干预措施: Inclisiran (Drug)
结局指标
主要结局
Number of participants with treatment-emergent adverse events (TEAEs) and serious adverse events (TESAEs)
时间窗: From Day 1 in the study up to the end of study visit; up to 1080 days
Safety and tolerability: TEAEs, TESAEs (incidence, severity, relationship to study drug and discontinuation due to TEAEs)
次要结局
- Percentage and absolute change in LDL-C from baseline in the feeder study to end of study(Baseline (of feeder study) and Day 1080)