A Randomized, Double-Blind, Placebo-Controlled Study Followed by an Open Label Treatment Period to Evaluate the Efficacy and Safety of Alirocumab in Children and Adolescents with Heterozygous Familial Hypercholesterolemia

Sanofi-aventis recherche & développement0 个研究点目标入组 500 人开始时间: 2019年1月8日最近更新: 2021年4月5日

适应症

试验速览

暂无简介。

•-Children and adolescent male and female patients aged 8 to 17 years at the time of signed informed consent.
•-Patients with diagnosis of heterozygous familial hypercholesterolemia (heFH) through genotyping or clinical criteria.
•-Patients treated with optimal dose of statin +/- other LMT(s) or non-statin LMT(s) if statin intolerant at stable dose for at least 4 weeks prior to screening lipid sampling.
•-Patients with calculated LDL-C greater than or equal to 130 mg/dL (=3.37 mmol/L) at the screening visit except for patients who have previously participated in the DFI14223 study.
•-A signed informed consent indicating parental permission with or without patient assent.
•Are the trial subjects under 18? yes
•Number of subjects for this age range: 500
•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

•-Patient with body weight less than 25 kg.
•-Patients aged of 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 secondary hyperlipidemia.
•-Diagnosis of homozygous familial hypercholesterolemia.
•-Patient who has received lipid apheresis treatment within 2 months prior to the screening period, or has plans to receive it during the study.
•-Patients with uncontrolled type 1 or type 2 diabetes mellitus.
•-Patients with known uncontrolled thyroid disease.
•-Patients with uncontrolled hypertension.
•-Fasting triglycerides >350 mg/dL (3.95 mmol/L).
•-Severe renal impairment (ie, estimated glomerular filtration rate [eGFR] <30 mL/min/1.73 m^2.
•-Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) >2 x upper limit of normal (ULN).
•-Creatinine phosphokinase (CPK) >3 x ULN.

发起方