Two part (double-blind inclisiran versus placebo [Year 1] followed by open-label inclisiran [Year 2]]) randomized multicenter study to evaluate safety, tolerability and efficacy of inclisiran in children (6 to less than 12 years) with heterozygous familial hypercholesterolemia and elevated LDL-cholesterol (ORION-20)

Heterozygous Familial Hypercholesterolemia

Percentage change in LDL-C from baseline to Day 330 (Year 1)

Time-adjusted percent change in LDL-C from baseline after Day 90 and up to Day 330 (Year 1), Absolute change in LDL-C from baseline to Day 330 (Year 1), Percent change in PCSK9, total cholesterol, Apo B, and non-HDL-C from baseline to Day 330 (Year 1), Percent change and absolute change in LDL-C, PCSK9, total cholesterol, Apo B, non-HDL-C, lipoprotein (a) [Lp(a)], triglycerides, high density lipoprotein cholesterol (HDL-C), very low density lipoprotein cholesterol (VLDL-C), and apolipoprotein A1 (Apo A1) from baseline to each assessment time up to Day 720 (Year 2), Incidence, severity and relationship to study drug of treatment-emergent adverse events (AEs) and serious adverse events (SAEs); vital signs; laboratory parameters; anti-drug antibodies (ADA) measurement; growth (height, weight, body mass index (BMI)); pubertal development (sexual hormones and Tanner staging)

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