Open Label Phase 3b Study to Evaluate the Long-Term Efficacy and Safety of Lerodalcibep in Children and Adolescents, 6 to 17 Years of Age, With Heterozygous Familial Hypercholesterolemia (LIBerate-Kids OLE)
试验速览
- 阶段
- 3 期
- 状态
- 尚未招募
- 发起方
- 入组人数
- 150
- 试验地点
- 5
- 主要终点
- Change in LDL cholesterol at Week 156 from baseline
研究概览
简要总结
The goal is to assess the long term efficacy (LDL cholesterol reduction) and safety over 3 years of lerodalcibep (Lerochol) SC 300 mg QM administered by auto-injector (AI)/pre-filled pen (PFP) in male and female pediatric patients 6 to 17 years of age, with inherited high cholesterol (HeFH) on a stable diet and maximally tolerated oral LDL C lowering drug therapy such as statins who completed the 24 week placebo controlled base trial.
The main question[s] it aims to answer are:
How effective is Lerochol in maintaining LDL cholesterol reductions over years? How well is it tolerated and are there any safety concerns?
Participants will visit the clinic every month for 3 months and then home dosed with clinic visits every 3 months. They will undergo periodic physical exams, height and weight measurements, answer questions, have blood drawn from a vein in their arm, have blood pressure measurements, EKC heart tests, and receive monthly injections lasting about 5 seconds in their arms or abdomen with an autoinjector.
详细描述
STUDY DESIGN AND DURATION:
This is an open label Phase 3b study to evaluate the long-term efficacy, safety, and tolerability of lerodalcibep 300 mg administered SC QM (<31 days) over a 156-week Treatment Period. Approximately 150 males and females 6 to 17 years of age at time of entry in the base LIB003 008 study, with HeFH and who completed the LIB003-008 study at approximately 30 sites in the United States, Canada, Central and South America, Europe, South Africa, Asia, Australasia, and the Middle East may be eligible to continue into this 156-week long term open-label extension with lerodalcibep after signing an independent informed consent form.
Patients maintaining their statin with or without ezetimibe and other allowed oral LDL-C-lowering agents will be eligible.
Study Procedures Patients who meet all eligibility criteria for the LIB003-016 study may proceed directly to the first dose of lerodalcibep at the final visit of the LIB003-008 study. Patients will receive SC doses, via auto-injector (AI)/prefilled pen (PFP) of lerodalcibep 300 mg QM (31 days) until Week 152. Following administration of the last dose at Week 152, the patients will return 4 weeks later for follow-up at Week 156.
Patients may begin the OLE on the final visit of their prior study and/or have up to 28 days to return for Day 1 of the OLE. The period between the base study and LIB003-008 may be extended upon review and approval.
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
- 6 Years 至 17 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Provision of written and signed informed consent/assent of the LIB003-016 trial prior to any study-specific procedure;
- •Completion of the LIB003-008 study having received the last dose of study drug at Week 20 and completed the Week 24 visit;
- •Male or female, 6 to 17 years of age (defined as from 6 to less than 18 years of age), at the first Screening Visit of the LIB003-008 study;
- •Weight of >18 kg (40 lbs) and BMI <17 and >42 kg/m2;
- •On stable diet and lipid-lowering oral drug therapy (statins, ezetimibe, bile-acid sequestrants) or combinations thereof (excluded oral lipid-lowering agents are include mipomersen, lomitapide, and gemfibrozil);
排除标准
- •History of any prior or active clinical condition or acute and/or unstable systemic disease compromising patient inclusion, at the discretion of the Investigator, which in the Investigator's opinion, would not be suitable for the study from a patient safety consideration or could interfere with the results of the study;
- •Females of childbearing potential who are sexually active, not using or unwilling to use a highly effective form of contraception during the study and until 60 days after last dose of study drug, pregnant or breastfeeding, or who have a positive urine pregnancy test at the last Screening Visit;
- •Patients who cannot be available for Protocol-required study visits or procedures, to the best of the patient's and Investigator's knowledge;
- •A history, during the LIB003-008 study of prescription drug abuse, illicit drug use, or alcohol abuse according to medical history;
- •Have any other finding which, in the opinion of the Investigator, would compromise the patient's safety or participation in the study;
研究组 & 干预措施
Lerodalcibep
lerodalcibep 300 mg monthly
干预措施: Lerodalcibep (Drug)
结局指标
主要结局
Change in LDL cholesterol at Week 156 from baseline
时间窗: 156 weeks
The primary objective of this study is to assess the change in LDL-C (by Friedewald Formula) at Week 156 with monthly lerodalcibep 300 mg QM
次要结局
未报告次要终点