Evolocumab in patients with HeFH aged 10-17
Research type
Research Study
Full title
Double-blind, Randomized, Multicenter, Placebo-controlled, Parallel Group Study to Characterize the Efficacy, Safety, and Tolerability of 24 Weeks of Evolocumab for Low Density Lipoprotein-cholesterol (LDL-C) Reduction, as Add-on to Diet and Lipid-lowering Therapy, in Pediatric Subjects 10 to 17 Years of Age With Heterozygous Familial Hypercholesterolemia (HeFH)
IRAS ID
195284
Contact name
Saikat Santra
Contact email
Sponsor organisation
Amgen Inc
Eudract number
2014-002277-11
Clinicaltrials.gov Identifier
Duration of Study in the UK
1 years, 8 months, 1 days
Research summary
Heterozygous Familial Hypercholesterolaemia (HeFH) is an inherited disorder caused by mutations in one of several genes involved in clearing low density lipoprotein cholesterol (LDL-c) from the bloodstream. Children with this disease have higher levels of LDL-c in their blood than other children and as a result have a higher risk of developing heart disease at an earlier age in adulthood than people who don’t have HeFH. Lowering the LDL-c level in the blood can reduce the risk of heart disease in later life and it is currently recommended (in NICE Guideline CG71) that children with HeFH start treatment to lower their LDL-c level from the age of 10 years. There are a number of medications already licensed for children with HeFH to lower LDL-c levels, the most commonly used being statins. However it is known that some children with HeFH still run high levels of LDL-c even when treated with statins and so may still have a higher risk of heart disease in adulthood.
Evolocumab is a new drug being developed to lower LDL-c levels in people with high cholesterol levels. It is licensed for use in adults in many countries.
This study is designed to see if evolocumab is safe, effective and well tolerated by children and young adults with HeFH aged 10-17 years whose LDL-c levels are still high despite treatment with an adequate dose of a statin. This is a randomised, placebo-controlled, double-blind study run across multiple centres and seeks to recruit about 150 children and young people who will be randomised to receive injections under the skin of evolocumab or placebo in a 2:1 ratio every month for about 6 months whilst continuing their existing low fat diet and statin treatment.
REC name
South Central - Hampshire A Research Ethics Committee
REC reference
16/SC/0378
Date of REC Opinion
15 Sep 2016
REC opinion
Further Information Favourable Opinion