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Clinical Trials/NL-OMON55627
NL-OMON55627CompletedNot Applicable

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

Amgen0 sites25 target enrollmentStarted: TBDLast updated:
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Conditions

Trial Snapshot

Phase
Not Applicable
Status
Completed
Sponsor
Amgen
Enrollment
25
Study OverviewEligibility CriteriaInvestigatorsRecords & IdentifiersSimilar Trials

Study Overview

Brief Summary

No summary available.

Study Design

Study Type
Interventional

Eligibility Criteria

Ages
2 to 17 (—)

Inclusion Criteria

  • •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

Exclusion Criteria

  • •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.

Investigators

Sponsor
Amgen

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