NL-OMON55627已完成不适用
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
适应症
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 发起方
- Amgen
- 入组人数
- 25
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
入排标准
- 年龄范围
- 2 至 17（—）
入选标准
- •All Subjects:
- •Subject has provided written informed consent or subject assent prior to
- •initiation of any study-specific activities/procedures.
- •Subject*s legally acceptable representative has provided informed consent
- •when the subject is legally too young to provide informed consent and the
- •subject has provided written subject assent based on local regulations and/or
- •guidelines prior to any study-specific activities/procedures being initiated.
- •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 (includes the
- •year after the subject completes the 17th year after birth but not the day of
- •completing the 18th year after birth).
- •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.
- •Subject must be on a low-fat diet and receiving background lipid-lowering
- •therapy (such as statins, cholesterol absorption inhibitors, bile acid
- •sequestrants, nicotinic acid, or combinations thereof).
- •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 screening.
- •Fasting LDL-C at screening >= 130 mg/dL (3.4 mmol/L) as determined by central
- •laboratory.
- •Fasting triglycerides <= 400 mg/dL (4.5 mmol/L) by central laboratory at
排除标准
- •All Subjects:
- •Currently receiving treatment in another investigational device or drug
- •study, or less than 30 days since ending treatment on another investigational
- •device or drug study(s). Other investigational procedures or treatments while
- •participating in this study are excluded.
- •Female subject who has experienced menarche and unwilling to use acceptable
- •method(s) of effective birth control during treatment with evolocumab and for
- •an additional 15 weeks after the end of treatment with evolocumab. A female who
- •has experienced menarche is considered of childbearing potential.
- •Female subject is pregnant or breast feeding, or planning to become pregnant
- •or planning to breastfeed during screening, during treatment with evolocumab,
- •and within 15 weeks after the end of treatment with evolocumab.
- •Unreliability as a study participant based on the investigator's (or
- •designee*s) knowledge of the subject (eg, alcohol or other drug abuse in the
- •past year, inability or unwillingness to adhere to the protocol, or psychosis).
- •Subject will not be available for or likely not to comply with
- •protocol-required study visits or procedures, to the best of the subject and
- •investigator*s knowledge (Note: Day 1 and week 80 visits must be scheduled at
- •approximately the same time of day, and should be performed as close as
- •possible to 8 am as the hormones measured have diurnal variation).
- •Known sensitivity to any of the active substances or their excipients to be
- •administered during dosing, eg, carboxymethylcellulose.
- •Subjects with HoFH:
- •Moderate to severe renal dysfunction, defined as an estimated glomerular
- •filtration rate (eGFR) < 30 ml/min/1.73m2 at screening, confirmed by a repeat
- •measurement at least 1 week apart. Note: eGFR will be calculated by the central
- •laboratory and will be provided to the site for eligibility determination.
- •Persistent active liver disease or hepatic dysfunction, defined as aspartate
- •aminotransferase (AST) or alanine aminotransferase (ALT) > 2 times the ULN as
- •determined by central laboratory analysis at screening, confirmed by a repeat
- •measurement at least 1 week apart.
- •CK > 3 times the ULN at screening, confirmed by a repeat measurement at least
- •1 week apart.
- •Known active infection or major hematologic, renal, metabolic,
- •gastrointestinal or endocrine dysfunction in the judgment of the investigator.
- •Subject has taken a cholesterylester transfer protein (CETP) inhibitor such
- •as anacetrapib, dalcetrapib or evacetrapib in the last 12 months, or mipomersen
- •or lomitapide in the last 5 months prior to LDL-C screening.
- •Subject has received evolocumab or any other therapy to inhibit PCSK9 within
- •12 weeks of screening.
- •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, if consulted, would pose a risk to subject
- •safety or interfere with the study evaluation, procedures or completion.
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