Testing a new cholesterol injection for children with severe inherited high cholesterol

This study is testing a medication called inclisiran to see if it can help children with homozygous familial hypercholesterolemia — a rare genetic condition where the body cannot properly control cholesterol levels — lower their cholesterol safely. For the first year, some children will receive the medication while others receive a placebo (inactive injection), and all children will then receive the active medication in the second year. The goal is to understand whether this treatment works and is safe in young children.

Children with this severe inherited cholesterol condition are at very high risk for heart disease at a young age, even when taking standard cholesterol-lowering medicines. Researchers want to see if this new injectable treatment can provide additional help by working in a different way than current medications.