Open Label Evolocumab Study in Patients Aged 10-17yrs with HeFH & HoFH
Research type
Research Study
Full title
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)
IRAS ID
195314
Contact name
Saikat Santra
Contact email
Sponsor organisation
Amgen Ltd
Eudract number
2015-002276-25
Clinicaltrials.gov Identifier
Duration of Study in the UK
2 years, 9 months, 29 days
Research summary
Heterozygous Familial Hypercholesterolaemia (HeFH) and Homozygous familial hypercholesterolemia (HoFH) are inherited disorders 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 or HoFH. Lowering the LDL-c level in the blood can reduce the risk of heart disease in later life. There are a number of medications already licensed for children with HeFH or HoFH to lower LDL-c levels, the most commonly used being statins.
However it is known that some children with HeFH or HoFH 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 approved by agencies in multiple countries to lower LDL-c levels in certain patients when used with diet and statins. Evolocumab is approved for use in patients with HoFH; however, the safety and effectiveness of evolocumab have not been established in paediatric patients with HoFH who are younger than 13 years old.
This study is designed to see if evolocumab is safe, effective and well tolerated by children and young adults with HeFH or HoFH, aged 10-17 years whose LDL-c levels are still high despite treatment with an adequate dose of a statin. This is an open label, single-arm study run across multiple centres and seeks to recruit about 115 children and young people who will receive injections under the skin of evolocumab every month for about 18.5 months whilst continuing their existing low fat diet and statin treatment.REC name
South Central - Hampshire A Research Ethics Committee
REC reference
17/SC/0136
Date of REC Opinion
9 May 2017
REC opinion
Further Information Favourable Opinion