This is a pivotal phase III study designed to evaluate safety, tolerability, and efficacy of inclisiran in children (aged 6 to \<12 years) with heterozygous familial hypercholesterolemia (HeFH) and elevated low density lipoprotein cholesterol (LDLC).
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 6 to \<12 years) with heterozygous familial hypercholesterolemia (HeFH) and elevated low density lipoprotein cholesterol (LDL-C) on stable standard of care background lipid-lowering therapy.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
DOUBLE
Enrollment
51
Inclisiran (inclisiran sodium 300 mg subcutaneous (s.c.) for participants with body weight ≥23 kg and inclisiran sodium 180 mg s.c. for participants with body weight \<23 kg. The dose level is based on the participant's body weight on Day 1 (for Part 1) and Day 360 (for Part 2), respectively.
Sterile normal saline (0.9% sodium chloride in water for subcutaneous injection)
Percentage change in LDL-C from baseline to Day 330 (Year 1)
Demonstrate superiority of inclisiran compared to placebo in reducing LDL-C \[percent change\] at Day 330
Time frame: Baseline and Day 330
Time-adjusted percent change in LDL-C from baseline after Day 90 and up to Day 330 (Year 1)
Demonstrate superiority of inclisiran compared to placebo in reducing LDL-C \[time-adjusted percent change\] over Year 1
Time frame: Baseline, after Day 90 up to Day 330
Absolute change in LDL-C from baseline to Day 330 (Year 1)
Demonstrate superiority of inclisiran compared to placebo in reducing LDL-C \[absolute change\] at Day 330 (Year 1)
Time frame: Baseline and Day 330
Percent change in PCSK9 from baseline to Day 330 (Year 1)
Demonstrate superiority of inclisiran compared to placebo in reducing PCSK9, total cholesterol, Apo B, and non-HDL-C \[percent change\] at Day 330 (Year 1)
Time frame: Baseline and Day 330
Percent change in total cholesterol, non-HDL-C from baseline to Day 330 (Year 1)
Demonstrate superiority of inclisiran compared to placebo in reducing PCSK9, total cholesterol, Apo B, and non-HDL-C \[percent change\] at Day 330 (Year 1)
Time frame: Baseline and Day 330
Percent change in Apo B from baseline to Day 330 (Year 1)
Demonstrate superiority of inclisiran compared to placebo in reducing PCSK9, total cholesterol, Apo B, and non-HDL-C \[percent change\] at Day 330 (Year 1)
Time frame: Baseline and Day 330
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UC San Francisco Medical Center
San Francisco, California, United States
RECRUITINGUC San Francisco Medical Center
San Francisco, California, United States
RECRUITINGChildren's National Hospital
Washington D.C., District of Columbia, United States
NOT_YET_RECRUITINGChildrens National Hospital
Washington D.C., District of Columbia, United States
RECRUITINGExcel Medical Clinical Trials LLC
Boca Raton, Florida, United States
RECRUITINGIcahn School of Med at Mt Sinai
New York, New York, United States
RECRUITINGPrimary Childrens Medical Center
Salt Lake City, Utah, United States
NOT_YET_RECRUITINGPrimary Childrens Medical Center
Salt Lake City, Utah, United States
RECRUITINGVirginia Commonwealth University
Richmond, Virginia, United States
RECRUITINGWest Virginia Childrens Hospital
Morgantown, West Virginia, United States
RECRUITING...and 56 more locations
Percent change in LDLC, total cholesterol, non-HDLC, triglycerides, HDL-C, VLDL-C from baseline to each assessment time up to Day 720 (Year 2)
Evaluate the effect of inclisiran, compared to placebo (for Year 1) and long-term (up to Day 720), on lowering LDL-C, other lipoprotein and lipid parameters, and PCSK9 over time
Time frame: Baseline, up to Day 720
Percent change in PCSK9 from baseline to each assessment time up to Day 720 (Year 2)
Evaluate the effect of inclisiran, compared to placebo (for Year 1) and long-term (up to Day 720), on lowering LDL-C, other lipoprotein and lipid parameters, and PCSK9 over time
Time frame: Baseline, up to Day 720
Percent change in Apo B, Apo A1 from baseline to each assessment time up to Day 720 (Year 2)
Evaluate the effect of inclisiran, compared to placebo (for Year 1) and long-term (up to Day 720), on lowering LDL-C, other lipoprotein and lipid parameters, and PCSK9 over time
Time frame: Baseline, up to Day 720
Absolute change in LDLC, total cholesterol, non-HDLC, triglycerides, HDL-C, VLDL-C from baseline to each assessment time up to Day 720 (Year 2)
Evaluate the effect of inclisiran, compared to placebo (for Year 1) and long-term (up to Day 720), on lowering LDL-C, other lipoprotein and lipid parameters, and PCSK9 over time
Time frame: Baseline, up to Day 720
Absolute change in PCSK9 from baseline to each assessment time up to Day 720 (Year 2)
Evaluate the effect of inclisiran, compared to placebo (for Year 1) and long-term (up to Day 720), on lowering LDL-C, other lipoprotein and lipid parameters, and PCSK9 over time
Time frame: Baseline, up to Day 720
Absolute change in Apo B, Apo A1 from baseline to each assessment time up to Day 720 (Year 2)
Evaluate the effect of inclisiran, compared to placebo (for Year 1) and long-term (up to Day 720), on lowering LDL-C, other lipoprotein and lipid parameters, and PCSK9 over time
Time frame: Baseline, up to Day 720
Percent change in Lp(a) from baseline to each assessment time up to Day 720 (Year 2)
Evaluate the effect of inclisiran, compared to placebo (for Year 1) and long-term (up to Day 720), on lowering LDL-C, other lipoprotein and lipid parameters, and PCSK9 over time
Time frame: Baseline, up to Day 720
Absolute change in Lp(a) from baseline to each assessment time up to Day 720 (Year 2)
Evaluate the effect of inclisiran, compared to placebo (for Year 1) and long-term (up to Day 720), on lowering LDL-C, other lipoprotein and lipid parameters, and PCSK9 over time
Time frame: Baseline, up to Day 720