Open-label, Single-arm, Multicenter Study to Evaluate the Safety, Tolerability and Efficacy of Evolocumab for LDL-C Reduction, as Add-on to Diet and Lipid-lowering Therapy, in Pediatric Subjects From 10 to 17 Years of Age With Heterozygous Familial Hypercholesterolemia (HeFH) or Homozygous Familial Hypercholesterolemia (HoFH) - HAUSER-OLE

Amgen Inc0 个研究点目标入组 163 人开始时间: 2016年5月16日最近更新: 2021年12月6日

适应症

试验速览

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•Subjects with HeFH:
•-Completed Study 20120123 while still on assigned investigational product.
•Subjects with HoFH:
•-Male or female, = 10 to = 17 years of age at time of enrollment
•-Diagnosis of HoFH by genetic confirmation or a clinical diagnosis based on a
•history of an untreated LDL cholesterol concentration > 500 mg/dL (13 mmol/L)
•together with either xanthoma before 10 years of age or evidence of
•heterozygous familial hypercholesterolemia in both parents.
•All subjects:
•- Subject must be on a low-fat diet and receiving background lipid-lowering therapy
•-Lipid-lowering therapy, including statin dose, must be unchanged for = 4 weeks
•prior to LDL-C screening; fibrates must be stable for at least 6 weeks prior to
•Are the trial subjects under 18? yes
•Number of subjects for this age range: 115
•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

•Subjects with HoFH:
•- estimated glomerular filtration rate (eGFR) < 30 ml/min/1.73m2; CK > 3x ULN; AST or ALT > 3x ULN; (all screening by central laboratory);
•- known active infection or major hematologic, renal, metabolic, gastrointestinal or endocrine dysfunction;
•- subject has taken a cholesterylester transfer protein (CETP) inhibitor in the last 12 months, or mipomersen or lomitapide in the last 5 months prior to LDL-C screening, or has received any therapy to inhibit proprotein convertase subtilisin/kexin type 9 (PCSK9) within 12 weeks prior to screening;
•- subject has a history or evidence of any other clinically significant disorder, condition or disease, or planned or expected procedure that, in the opinion of the Investigator or Amgen physician, would pose a risk to subject safety or interfere with the study evaluation, procedures or completion.
•The following are major exclusion criteria for all subjects:
•- subjects cannot be receiving treatment in another investigational device or drug study, or less than 30 days since ending treatment on another investigational device or drug study;
•- female subjects of childbearing potential cannot be pregnant or breast feeding or planning to become pregnant or planning to breast feed and must be willing to use acceptable method(s) of effective birth control (may include true sexual abstinence) during treatment with evolocumab and for an additional 15 weeks after the end of treatment with evolocumab.

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