Safety, Tolerability and Efficacy of Evolocumab (AMG 145) in Children With Inherited Elevated Low-density Lipoprotein Cholesterol (Familial Hypercholesterolemia) (HAUSER-OLE)
Status and phase
Conditions
Treatments
Study type
Funder types
Identifiers
Details and patient eligibility
About
The main purpose of this study is to describe the safety and tolerability of 80 weeks of subcutaneous (SC) evolocumab when added to standard of care in children 10 to 17 years of age with familial hypercholesterolemia.
Enrollment
Sex
Ages
Volunteers
Inclusion criteria
Heterozygous Familial Hypercholesterolemia (HeFH):
-Completed Study 20120123 (NCT02392559) while still on assigned investigational product and did not experience a treatment-related serious adverse event
Homozygous Familial Hypercholesterolemia (HoFH):
- Male or female, ≥ 10 to ≤ 17 years of age at time of enrollment
- Diagnosis of HoFH
- On a low-fat diet and receiving background lipid-lowering therapy
- Lipid-lowering therapy 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)
- Fasting triglycerides ≤ 400 mg/dL (4.5 mmol/L)
Exclusion criteria
-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); except Study 20120123
HoFH:
- Moderate to severe renal dysfunction
- Active liver disease or hepatic dysfunction,
- Creatine kinase > 3 times the upper limit of normal (ULN) at screening
Trial design
Primary purpose
Allocation
Interventional model
Masking
163 participants in 1 patient group
Trial contacts and locations
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Data sourced from clinicaltrials.gov
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