Study to evaluate efficacy and safety of inclisiran in children aged 6 to under 12 years of age with heterozygous familial hypercholesterolemia.

Prior treatment

• Previous treatment (within 90 days of screening) with monoclonal antibodies directed towards PCSK9

Other illness

• Secondary hypercholesterolemia, e.g. hypothyroidism or nephrotic syndrome

Lab values

• Active liver disease defined as any known current infectious, neoplastic, or metabolic pathology of the liver or unexplained alanine aminotransferase (ALT), aspartate aminotransferase (AST) elevation >3x ULN, or total bilirubin elevation >2x ULN (except patients with Gilbert’s syndrome)

Other

• Homozygous familial hypercholesterolemia (HoFH)
• Body weight <16 kg at the screening and/or randomization (Day 1) visit
• Pregnant or nursing females
• Recent and/or planned use of other investigational medicinal products or devices

• Male or female participants, 6 to <12 years of age at screening
• HeFH diagnosed either by genetic testing or on phenotypic criteria
• Fasting LDL-C >130 mg/dL (3.4 mmol/L) at screening
• For participants 8 to <12 years, on an optimal dose of statin (investigator’s discretion) unless statin intolerant, with or without other lipid-lowering therapy (e.g. ezetimibe). For participants <8 years, the use of background lipid-lowering treatment is based on investigator’s discretion.
• Participants on lipid-lowering therapies (such as statin and/or e.g. ezetimibe) must be on a stable dose for ≥30 days before screening with no planned medication or dose changes during study participation.