Summary
This is a pivotal phase III study designed to evaluate safety, tolerability, and efficacy of inclisiran in children (aged 2 to <12 years) with homozygous familial hypercholesterolemia (HoFH) and elevated low density lipoprotein cholesterol (LDLC).
Official Title
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 (2 to Less Than 12 Years) With Homozygous Familial Hypercholesterolemia and Elevated LDL-cholesterol
Details
This is a two-part (1 year double-blind inclisiran versus placebo / 1 year open-label inclisiran) multicenter study designed to evaluate safety, tolerability, and efficacy of inclisiran in children (aged 2 to <12 years) with homozygous familial hypercholesterolemia (HoFH) and elevated low density lipoprotein cholesterol (LDL-C) on stable standard of care background lipid-lowering therapy.
Keywords
Familial Hypercholesterolemia - Homozygous, Homozygous familial hypercholesterolemia (HoFH),, LDL-cholesterol (LDL-C), children, pediatric,, small interfering ribonucleic acid (siRNA),, inclisiran,, Familial Hypercholesterolemia,, Homozygous FH,, Hypercholesterolemia,, Lipoprotein(a),, Hyperlipidemia,, Dyslipidemia,, Cardiovascular Diseases,, Heart Failure,, Cholesterol,, Aortic Stenosis, Homozygous Familial Hypercholesterolemia, Hyperlipoproteinemia Type II, Hypercholesterolemia, Hyperlipidemias, Dyslipidemias, Cardiovascular Diseases, Heart Failure, Aortic Valve Stenosis, ALN-PCS, Counterfeit Drugs, Saline Solution, Inclisiran