Harefield Hospital recruits first UK patient to international paediatric drug trial

Researchers at Harefield Hospital have successfully recruited the first UK patient for a study looking into the effects of a cholesterol-lowering drug in children and young people with heterozygous familial hypercholesterolemia (HeFH).

HeFH is a hereditary disease that affects the body’s ability to clear low density lipoprotein (LDL) cholesterol (“bad” cholesterol) from the blood, leading to an increased risk of early heart disease.

Led by Professor Mahmoud Barbir, consultant cardiologist, the ORION-16 trial will study the effect of the drug inclisiran, in children and young people with HeFH, to determine the safety of the drug and whether it will lower LDL-cholesterol.

Sponsored by Novartis, the trial is being conducted in 26 countries. In the UK there are two centres participating, but it is at Harefield Hospital where the first UK participant has been recruited and successfully randomised.

During the first year of the trial, participants will be randomly assigned to receive either the study drug or a placebo in addition to their current lipid-lowering treatment. In the second year, all participants will be given the opportunity to receive the study drug.

Professor Barbir highlights the importance of this trial for patients with HeFH:

“Hypercholesterolaemia is a leading cause of cardiovascular morbidity and mortality.  Accordingly, efforts to lower Apolipoprotein B containing lipoprotein in plasma are the centre piece of strategies for cardiovascular prevention and treatment in primary and secondary management.

“Many children with heterozygous FH do not achieve appropriate low density lipoprotein (LDL) cholesterol goals in spite of treatment with statins. The study drug is a novel agent for lowering the Apolipoprotein B - containing lipoproteins. This has the potential to further advance the lipid lowering treatment options for paediatric patients with familial hypercholesterolaemia and aims to prevent atherosclerotic cardiovascular disease in children with HeFH.”

The study also marks an important milestone for paediatric research at our hospitals, with this being the first paediatric drug study to be conducted at Harefield Hospital.

The project would not have been possible without the support of the Familial Hypercholesterolaemia paediatric service, established in 2011 with support from Jane Breen, nurse specialist, and paediatric consultants Dr Rodney Franklin, Dr Zdenek Slavik and Dr Anke Furck. The service is currently run by Professor Barbir, Dr Tina Khan, and clinical nurse specialists Emma Neves and Lorraine Priestley-Barnham.

Professor Barbir also highlighted the importance of the dedicated research team, led by research nurse Catherine Hugget, in making it possible, not only for setting up the first paediatric drug study at Harefield Hospital but also working hard to recruit and randomise the first UK patient onto the trial.  

Laura Barker, one of the research nurses working on the trial, said:

“We were incredibly excited about this clinical trial opening at Harefield Hospital and our new collaborations with the Lipid Management Team and the Paediatric Seedlings Clinic Team.

“The hard work we all put in was worth it to see our first patient enter the trial (with mum present) and we know that this may positively change the landscape for the future of children born with HeFH. I personally cannot thank the children that enter this trial enough.”

Once the study is completed, a summary of the results will be publicly available here and here.

To find out more about this trial or any of our other research projects, please contact us.