This was a pivotal phase III study designed to evaluate safety, tolerability, and efficacy of inclisiran in adolescents with heterozygous familial hypercholesterolemia (HeFH) and elevated low density lipoprotein cholesterol (LDL-C).
This was 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 adolescents with heterozygous familial hypercholesterolemia (HeFH) and elevated low density lipoprotein cholesterol (LDL-C) on stable standard of care background lipid-lowering therapy. The primary objective was to demonstrate superiority of inclisiran compared to placebo in reducing LDL-C (percent change) at Day 330.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
DOUBLE
Enrollment
141
Inclisiran sodium 300 mg (equivalent to 284 mg inclisiran) in 1.5 mL solution for subcutaneous injection
Sterile normal saline (0.9% sodium chloride in water for subcutaneous injection)
Percentage Change in LDL-C From Baseline to Day 330 (Part 1/Year 1)
Percentage change in low-density lipoprotein cholesterol (LDL-C) from baseline to Day 330 (Year 1)
Time frame: Baseline and Day 330
Time-adjusted Percent Change in LDL-C From Baseline After Day 90 and up to Day 330 (Part 1/Year 1)
Time-adjusted percent change in LDL-C (after Day 90 and up to Day 330), calculated as the average of percent changes from baseline to Days 150, 270 and 330
Time frame: Baseline, after Day 90 up to Day 330
Absolute Change in LDL-C From Baseline to up Day 330 (Part 1/Year 1)
Absolute change in LDL-C from baseline to Day 330.
Time frame: Baseline and Day 330
Percent Change in Apo B From Baseline up to Day 330 (Part 1/Year 1)
Percentage change in apolipoprotein B (Apo B) from baseline to Day 330.
Time frame: Baseline and Day 330
Percent Change in Lp(a) From Baseline up to Day 330 (Part 1/Year 1)
Percentage change in lipoprotein (a) \[Lp(a)\] from baseline to Day 330.
Time frame: Baseline and Day 330
Percent Change in Non-HDL-C From Baseline up to Day 330 (Part 1/Year 1)
Percentage change in non-high density lipoprotein cholesterol (non-HDL-C) from baseline to Day 330.
Time frame: Baseline and Day 330
Percent Change in Total Cholesterol From Baseline up to Day 330 (Part 1/Year 1)
Percentage change in total cholesterol from baseline to Day 330.
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Tucson Medical Center
Tucson, Arizona, United States
Icahn School of Medicine at Mount Sinai
New York, New York, United States
Wake Forest U of Health Sciences
Winston-Salem, North Carolina, United States
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States
Childrens Hospital Pittsburgh of UPMC
Pittsburgh, Pennsylvania, United States
Primary Children's Medical Center
Salt Lake City, Utah, United States
Novartis Investigative Site
Formosa, Formosa Province, Argentina
Unidade de pesquisa clinica - Hospital Universitario Walter Cantidio
Fortaleza, Ceará, Brazil
Nucleo de Pesquisa Clinica do Rio Grande do Sul
Porto Alegre, Rio Grande do Sul, Brazil
Setor de Lípides, Aterosclerose e Biologia
São Paulo, São Paulo, Brazil
...and 49 more locations
Time frame: Baseline and Day 330
Percent Change in LDL-C From Baseline up to Day 720
Percentage change in LDL-C from baseline to each assessment time up to Day 720.
Time frame: Baseline, up to Day 720
Absolute Change in LDL-C From Baseline up to Day 720
Absolute change in LDL-C from baseline to each assessment time up to Day 720.
Time frame: Baseline, up to Day 720
Percent Change in Apo B From Baseline up to Day 720
Percentage change in apolipoprotein B (Apo B) from baseline to each assessment time up to Day 720.
Time frame: Baseline, up to Day 720
Absolute Change in Apo B From Baseline up to Day 720
Absolute change in apolipoprotein B (Apo B) from baseline to each assessment time up to Day 720.
Time frame: Baseline, up to Day 720
Percent Change in Lp(a) From Baseline up to Day 720
Percentage change in lipoprotein (a) \[Lp(a)\] from baseline to each assessment time up to Day 720.
Time frame: Baseline, up to Day 720
Absolute Change in Lp(a) From Baseline up to Day 720
Absolute change in lipoprotein (a) \[Lp(a)\] from baseline to each assessment time up to Day 720.
Time frame: Baseline, up to Day 720
Percent Change in Non-HDL-C From Baseline up to Day 720
Percentage change in non-high density lipoprotein cholesterol (non-HDL-C) from baseline to each assessment time up to Day 720.
Time frame: Baseline, up to Day 720
Absolute Change in Non-HDL-C From Baseline up to Day 720
Absolute change in non-high density lipoprotein cholesterol (non-HDL-C) from baseline to each assessment time up to Day 720.
Time frame: Baseline, up to Day 720
Percent Change in Total Cholesterol From Baseline up to Day 720
Percentage change in total cholesterol from baseline to each assessment time up to Day 720.
Time frame: Baseline, up to Day 720
Absolute Change in Total Cholesterol From Baseline up to Day 720
Absolute change in total cholesterol from baseline to each assessment time up to Day 720.
Time frame: Baseline, up to Day 720
Percent Change in Triglycerides From Baseline up to Day 720
Percentage change in triglycerides from baseline to each assessment time up to Day 720.
Time frame: Baseline, up to Day 720
Absolute Change in Triglycerides From Baseline up to Day 720
Absolute change in triglycerides from baseline to each assessment time up to Day 720.
Time frame: Baseline, up to Day 720
Percent Change in HDL-C From Baseline up to Day 720
Percentage change in high density lipoprotein cholesterol (HDL-C) from baseline to each assessment time up to Day 720.
Time frame: Baseline, up to Day 720
Absolute Change in HDL-C From Baseline up to Day 720
Absolute change in high density lipoprotein cholesterol (HDL-C) from baseline to each assessment time up to Day 720.
Time frame: Baseline, up to Day 720
Percent Change in VLDL-C From Baseline up to Day 720
Percentage change in very low density lipoprotein cholesterol (VLDL-C) from baseline to each assessment time up to Day 720.
Time frame: Baseline, up to Day 720
Absolut Change in VLDL-C From Baseline up to Day 720
Absolute change in very low density lipoprotein cholesterol (VLDL-C) from baseline to each assessment time up to Day 720.
Time frame: Baseline, up to Day 720
Percent Change in Apo A1 From Baseline up to Day 720
Percentage change in apolipoprotein A1 (Apo A1) from baseline to each assessment time up to Day 720.
Time frame: Baseline, up to Day 720
Absolute Change in Apo A1 From Baseline up to Day 720
Absolute change in apolipoprotein A1 (Apo A1) from baseline to each assessment time up to Day 720.
Time frame: Baseline, up to Day 720
Percent Change in PCSK9 From Baseline up to Day 720
Percentage change in proprotein convertase subtilisin/kexin type 9 (PCSK9) from baseline to each assessment time up to Day 720.
Time frame: Baseline, up to Day 720
Absolut Change in PCSK9 From Baseline up to Day 720
Absolute change in proprotein convertase subtilisin/kexin type 9 (PCSK9) from baseline to each assessment time up to Day 720.
Time frame: Baseline, up to Day 720