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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/study/NCT04652726
Registration number
NCT04652726
Ethics application status
Date submitted
2/12/2020
Date registered
3/12/2020
Date last updated
10/06/2025
Titles & IDs
Public title
Study to Evaluate Efficacy and Safety of Inclisiran in Adolescents With Heterozygous Familial Hypercholesterolemia
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Scientific 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 Adolescents (12 to Less Than 18 Years) With Heterozygous Familial Hypercholesterolemia and Elevated LDL-cholesterol (ORION-16)
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Secondary ID [1]
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2020-002757-18
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Secondary ID [2]
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CKJX839C12301
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Universal Trial Number (UTN)
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Trial acronym
ORION-16
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Familial Hypercholesterolemia - Heterozygous
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Condition category
Condition code
Metabolic and Endocrine
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Other metabolic disorders
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Human Genetics and Inherited Disorders
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Other human genetics and inherited disorders
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Treatment: Drugs - Inclisiran
Treatment: Drugs - Placebo
Experimental: Inclisiran - Year 1 - inclisiran sodium 300 mg subcutaneous injection (given at Days 1, 90, and 270) Day 360 only - placebo subcutaneous injection Year 2 - inclisiran sodium 300 mg subcutaneous injection (given at Days 450 and 630)
Placebo comparator: Placebo - Year 1 - placebo subcutaneous injection (given at Days 1, 90 and 270) Year 2 - inclisiran sodium 300 mg subcutaneous injection (given at Days 360, 450, and 630)
Treatment: Drugs: Inclisiran
Inclisiran sodium 300 mg (equivalent to 284 mg inclisiran) in 1.5 mL solution for subcutaneous injection
Treatment: Drugs: Placebo
Sterile normal saline (0.9% sodium chloride in water for subcutaneous injection)
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Intervention code [1]
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Treatment: Drugs
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Percentage Change in LDL-C From Baseline to Day 330 (Part 1/Year 1)
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Assessment method [1]
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Percentage change in low-density lipoprotein cholesterol (LDL-C) from baseline to Day 330 (Year 1)
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Timepoint [1]
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Baseline and Day 330
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Secondary outcome [1]
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Time-adjusted Percent Change in LDL-C From Baseline After Day 90 and up to Day 330 (Part 1/Year 1)
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Assessment method [1]
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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
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Timepoint [1]
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Baseline, after Day 90 up to Day 330
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Secondary outcome [2]
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Absolute Change in LDL-C From Baseline to up Day 330 (Part 1/Year 1)
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Assessment method [2]
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Absolute change in LDL-C from baseline to Day 330.
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Timepoint [2]
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Baseline and Day 330
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Secondary outcome [3]
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Percent Change in Apo B From Baseline up to Day 330 (Part 1/Year 1)
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Assessment method [3]
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Percentage change in apolipoprotein B (Apo B) from baseline to Day 330.
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Timepoint [3]
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Baseline and Day 330
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Secondary outcome [4]
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Percent Change in Lp(a) From Baseline up to Day 330 (Part 1/Year 1)
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Assessment method [4]
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Percentage change in lipoprotein (a) \[Lp(a)\] from baseline to Day 330.
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Timepoint [4]
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Baseline and Day 330
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Secondary outcome [5]
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Percent Change in Non-HDL-C From Baseline up to Day 330 (Part 1/Year 1)
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Assessment method [5]
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Percentage change in non-high density lipoprotein cholesterol (non-HDL-C) from baseline to Day 330.
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Timepoint [5]
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Baseline and Day 330
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Secondary outcome [6]
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Percent Change in Total Cholesterol From Baseline up to Day 330 (Part 1/Year 1)
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Assessment method [6]
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Percentage change in total cholesterol from baseline to Day 330.
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Timepoint [6]
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Baseline and Day 330
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Secondary outcome [7]
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Percent Change in LDL-C From Baseline up to Day 720
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Assessment method [7]
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Percentage change in LDL-C from baseline to each assessment time up to Day 720.
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Timepoint [7]
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Baseline, up to Day 720
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Secondary outcome [8]
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Absolute Change in LDL-C From Baseline up to Day 720
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Assessment method [8]
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Absolute change in LDL-C from baseline to each assessment time up to Day 720.
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Timepoint [8]
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Baseline, up to Day 720
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Secondary outcome [9]
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Percent Change in Apo B From Baseline up to Day 720
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Assessment method [9]
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Percentage change in apolipoprotein B (Apo B) from baseline to each assessment time up to Day 720.
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Timepoint [9]
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Baseline, up to Day 720
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Secondary outcome [10]
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Absolute Change in Apo B From Baseline up to Day 720
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Assessment method [10]
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Absolute change in apolipoprotein B (Apo B) from baseline to each assessment time up to Day 720.
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Timepoint [10]
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Baseline, up to Day 720
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Secondary outcome [11]
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Percent Change in Lp(a) From Baseline up to Day 720
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Assessment method [11]
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Percentage change in lipoprotein (a) \[Lp(a)\] from baseline to each assessment time up to Day 720.
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Timepoint [11]
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Baseline, up to Day 720
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Secondary outcome [12]
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Absolute Change in Lp(a) From Baseline up to Day 720
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Assessment method [12]
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Absolute change in lipoprotein (a) \[Lp(a)\] from baseline to each assessment time up to Day 720.
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Timepoint [12]
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Baseline, up to Day 720
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Secondary outcome [13]
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Percent Change in Non-HDL-C From Baseline up to Day 720
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Assessment method [13]
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Percentage change in non-high density lipoprotein cholesterol (non-HDL-C) from baseline to each assessment time up to Day 720.
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Timepoint [13]
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Baseline, up to Day 720
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Secondary outcome [14]
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Absolute Change in Non-HDL-C From Baseline up to Day 720
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Assessment method [14]
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Absolute change in non-high density lipoprotein cholesterol (non-HDL-C) from baseline to each assessment time up to Day 720.
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Timepoint [14]
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Baseline, up to Day 720
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Secondary outcome [15]
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Percent Change in Total Cholesterol From Baseline up to Day 720
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Assessment method [15]
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Percentage change in total cholesterol from baseline to each assessment time up to Day 720.
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Timepoint [15]
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Baseline, up to Day 720
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Secondary outcome [16]
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Absolute Change in Total Cholesterol From Baseline up to Day 720
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Assessment method [16]
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Absolute change in total cholesterol from baseline to each assessment time up to Day 720.
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Timepoint [16]
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Baseline, up to Day 720
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Secondary outcome [17]
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Percent Change in Triglycerides From Baseline up to Day 720
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Assessment method [17]
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Percentage change in triglycerides from baseline to each assessment time up to Day 720.
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Timepoint [17]
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Baseline, up to Day 720
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Secondary outcome [18]
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Absolute Change in Triglycerides From Baseline up to Day 720
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Assessment method [18]
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Absolute change in triglycerides from baseline to each assessment time up to Day 720.
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Timepoint [18]
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Baseline, up to Day 720
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Secondary outcome [19]
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Percent Change in HDL-C From Baseline up to Day 720
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Assessment method [19]
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Percentage change in high density lipoprotein cholesterol (HDL-C) from baseline to each assessment time up to Day 720.
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Timepoint [19]
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Baseline, up to Day 720
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Secondary outcome [20]
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Absolute Change in HDL-C From Baseline up to Day 720
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Assessment method [20]
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Absolute change in high density lipoprotein cholesterol (HDL-C) from baseline to each assessment time up to Day 720.
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Timepoint [20]
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Baseline, up to Day 720
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Secondary outcome [21]
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Percent Change in VLDL-C From Baseline up to Day 720
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Assessment method [21]
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Percentage change in very low density lipoprotein cholesterol (VLDL-C) from baseline to each assessment time up to Day 720.
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Timepoint [21]
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Baseline, up to Day 720
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Secondary outcome [22]
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Absolut Change in VLDL-C From Baseline up to Day 720
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Assessment method [22]
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Absolute change in very low density lipoprotein cholesterol (VLDL-C) from baseline to each assessment time up to Day 720.
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Timepoint [22]
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Baseline, up to Day 720
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Secondary outcome [23]
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Percent Change in Apo A1 From Baseline up to Day 720
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Assessment method [23]
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Percentage change in apolipoprotein A1 (Apo A1) from baseline to each assessment time up to Day 720.
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Timepoint [23]
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Baseline, up to Day 720
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Secondary outcome [24]
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Absolute Change in Apo A1 From Baseline up to Day 720
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Assessment method [24]
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Absolute change in apolipoprotein A1 (Apo A1) from baseline to each assessment time up to Day 720.
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Timepoint [24]
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Baseline, up to Day 720
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Secondary outcome [25]
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Percent Change in PCSK9 From Baseline up to Day 720
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Assessment method [25]
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Percentage change in proprotein convertase subtilisin/kexin type 9 (PCSK9) from baseline to each assessment time up to Day 720.
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Timepoint [25]
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Baseline, up to Day 720
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Secondary outcome [26]
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Absolut Change in PCSK9 From Baseline up to Day 720
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Assessment method [26]
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Absolute change in proprotein convertase subtilisin/kexin type 9 (PCSK9) from baseline to each assessment time up to Day 720.
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Timepoint [26]
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Baseline, up to Day 720
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Eligibility
Key inclusion criteria
* Heterozygous Familial Hypercholesterolemia (HeFH) diagnosed either by genetic testing or on phenotypic criteria
* Fasting LDL-C >130 mg/dL (3.4 mmol/L) at screening
* Fasting triglycerides <400 mg/dL (4.5 mmol/L) at screening
* On maximally tolerated dose of statin (investigator's discretion) with or without other lipid-lowering therapy; stable for = 30 days before screening
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Minimum age
12
Years
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Maximum age
17
Years
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Sex
Both males and females
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Can healthy volunteers participate?
No
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Key exclusion criteria
* Homozygous familial hypercholesterolemia (HoFH)
* Active liver disease
* Secondary hypercholesterolemia, e.g. hypothyroidism or nephrotic syndrome
* Previous treatment with monoclonal antibodies directed towards PCSK9 (within 90 days of screening)
* Recent and/or planned use of other investigational medicinal products or devices
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Study design
Purpose of the study
Treatment
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Allocation to intervention
Randomised controlled trial
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Blinded (masking used)
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Who is / are masked / blinded?
The people receiving the treatment/s
The people analysing the results/data
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Intervention assignment
Parallel
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Other design features
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Phase
Phase 3
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Completed
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
27/01/2021
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
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Actual
27/11/2024
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Sample size
Target
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Accrual to date
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Final
141
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Recruitment in Australia
Recruitment state(s)
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Recruitment outside Australia
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United States of America
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New York
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North Carolina
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Ohio
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Pennsylvania
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Utah
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Argentina
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Formosa
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Brazil
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Ceara
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Brazil
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RS
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Brazil
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Czechia
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Praha 5
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Praha
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France
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France
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Baden-Wuerttemberg
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Germany
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Frankfurt
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Germany
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Freiburg
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Greece
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Greece
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Athens
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Hungary
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Israel
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Jerusalem
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Israel
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Ramat Gan
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Italy
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MI
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Jordan
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Irbid
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Oslo
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Lodz
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Kemerovo
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Moscow
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Russian Federation
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Novosibirsk
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Poprad
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New Taipei
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United Kingdom
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Middlesex
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Funding & Sponsors
Primary sponsor type
Commercial sector/industry
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Name
Novartis Pharmaceuticals
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Address
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Ethics approval
Ethics application status
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Summary
Brief summary
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).
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Trial website
https://clinicaltrials.gov/study/NCT04652726
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Trial related presentations / publications
Reijman MD, Schweizer A, Peterson ALH, Bruckert E, Stratz C, Defesche JC, Hegele RA, Wiegman A. Rationale and design of two trials assessing the efficacy, safety, and tolerability of inclisiran in adolescents with homozygous and heterozygous familial hypercholesterolaemia. Eur J Prev Cardiol. 2022 Jul 20;29(9):1361-1368. doi: 10.1093/eurjpc/zwac025. Warden BA, Duell PB. Inclisiran: A Novel Agent for Lowering Apolipoprotein B-containing Lipoproteins. J Cardiovasc Pharmacol. 2021 Aug 1;78(2):e157-e174. doi: 10.1097/FJC.0000000000001053.
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Public notes
This record is viewable in the ANZCTR as it had previously listed Australia and/or New Zealand as a recruitment site, however these sites have since been removed
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Contacts
Principal investigator
Name
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Novartis Pharmaceuticals
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Novartis Pharmaceuticals
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Contact person for scientific queries
Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Yes
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What data in particular will be shared?
Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations.
This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com.
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When will data be available (start and end dates)?
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Available to whom?
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Available for what types of analyses?
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How or where can data be obtained?
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What supporting documents are/will be available?
No Supporting Document Provided
Type
Other Details
Attachment
Study protocol
https://cdn.clinicaltrials.gov/large-docs/26/NCT04652726/Prot_000.pdf
Statistical analysis plan
https://cdn.clinicaltrials.gov/large-docs/26/NCT04652726/SAP_001.pdf
Results publications and other study-related documents
No documents have been uploaded by study researchers.
Results are available at
https://clinicaltrials.gov/study/NCT04652726
Download to PDF