Open Label Study to Evaluate Safety, Tolerability and Efficacy of Evolocumab (AMG 145) in Pediatric Subjects (10 to 17 Years of Age) With Heterozygous Familial Hypercholesterolemia (HeFH) or Homozygous Familial Hypercholesterolemia (HoFH).



Status:Recruiting
Conditions:High Cholesterol
Therapuetic Areas:Cardiology / Vascular Diseases
Healthy:No
Age Range:10 - 17
Updated:2/10/2019
Start Date:September 10, 2016
End Date:June 29, 2021
Contact:Amgen Call Center
Email:medinfo@amgen.com
Phone:866-572-6436

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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)

To describe the safety and tolerability of 80 weeks of subcutaneous (SC) evolocumab (AMG 145)
when added to standard of care in pediatric subjects 10 to 17 years of age with HeFH or HoFH.

An open label, single arm study to evaluate the safety and tolerability of 80 weeks of SC
evolocumab when added to standard of care in pediatric subjects 10 to 17 years of age with
HeFH or HoFH.

Inclusion Criteria:

Subjects with HeFH:

-Completed Study 20120123 while still on assigned investigational product and did not
experience a treatment‑related serious adverse event

Subjects with 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:

All Subjects

-Currently receiving treatment in another investigational device or drug study, or less
than 30 days since ending treament on another investigational device or drug study(s);
except Study 20120123

Subjects with HoFH:

- Moderate to severe renal dysfunction

- Active liver disease or hepatic dysfunction,

- CK > 3 times the ULN at screening
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