EUCTR2017-002297-39-IT进行中（未招募）1 期
An Open-Label Study to Evaluate the Efficacy and Safety of Alirocumab in Children and Adolescents with Homozygous Familial Hypercholesterolemia
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 18
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional clinical trial of medicinal product
入排标准
- 性别
- All
入选标准
- •-Patients genetically diagnosed with homozygous familial hypercholesterolemia (hoFH).
- •-Patients treated with optimal dose of statin +/- other lipid modifying therapies (LMTs), or non-statin LMTs if statin-intolerant at stable dose(s) for at least 4 weeks prior to screening lipid sample.
- •-A signed informed consent indicating parental permission with or without patient assent.
- •-For patients on apheresis, currently undergoing stable low-density lipoprotein (LDL) apheresis therapy prior to the screening and have initiated apheresis treatment for at least 6 months.
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 50
- •F.1.2 Adults (18-64 years) no
- •F.1.2.1 Number of subjects for this age range
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
排除标准
- •-Patients with low-density lipoprotein - cholesterol (LDL-C) less than 130 mg/dL (3.37 mmol/L) obtained during the screening period after the patient has been on stable apheresis procedure or lipid modifying therapy (LMT) (i.e., stable optimal dose of statin ± other stable LMTs, or stable non statin LMTs in statin-intolerant patients) treatment for at least 4 weeks.
- •-Patients with body weight less than 25 kg.
- •-Patients aged 8 to 9 years not at Tanner Stage 1 and patients aged of 10 to 17 years not at least at Tanner Stage 2 in their development.
- •-Patients with uncontrolled Type 1 or 2 diabetes mellitus.
- •-Patients with known uncontrolled thyroid disease.
- •-Patients with uncontrolled hypertension.
- •-Fasting triglycerides >350 mg/dL.
- •-Severe renal impairment (i.e., estimated glomerular filtration rate [eGFR] <30 mL/min/1.73m^2) at the screening visit.
- •-Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) >2 x upper limit of normal (ULN).
- •-Creatine phosphokinase (CPK) >3 x ULN.
研究者
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