This study will evaluate the efficacy and safety of zodasiran subcutaneous (sc) injection in participants 12 to \<18 years of age with genetically or clinically diagnosed homozygous familial hypercholesterolemia (HoFH) and low-density lipoprotein cholesterol (LDL-C) ≥116 milligrams per deciliter (mg/dL) on maximally tolerated lipid-lowering therapy.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
12
By sc injection
Percent Change from Baseline to Month 12 in Fasting LDL-C
Time frame: Baseline, Month 12
Percent Change from Baseline to Month 12 in Fasting Apolipoprotein B (ApoB)
Time frame: Baseline, Month 12
Change from Baseline to Month 12 in Fasting ApoB
Time frame: Baseline, Month 12
Percent Change from Baseline to Month 12 in Fasting Non-High Density Lipoprotein Cholesterol (non-HDL-C)
Time frame: Baseline, Month 12
Change from Baseline to Month 12 in Fasting Non-HDL-C
Time frame: Baseline, Month 12
Change from Baseline to Month 12 in Fasting LDL-C
Time frame: Baseline, Month 12
Area Under the Plasma Concentration Versus the Time Curve (AUC) from Baseline to Month 12 for Fasting LDL-C
Time frame: Baseline, Month 12
Percent Change from Baseline to Month 12 in Fasting Triglycerides (TGs)
Time frame: Baseline, Month 12
Change from Baseline to Month 12 in Fasting TGs
Time frame: Baseline, Month 12
Percent Change from Baseline to Month 12 in Fasting Angiopoietin-like Protein 3 (ANGPTL3)
Time frame: Baseline, Month 12
Change from Baseline to Month 12 in Fasting ANGPTL3
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Time frame: Baseline, Month 12
Percent Change from Baseline to Month 12 in Fasting Total Cholesterol
Time frame: Baseline, Month 12
Change from Baseline to Month 12 in Fasting Total Cholesterol
Time frame: Baseline, Month 12
Percent Change from Baseline to Month 12 in Fasting HDL-C
Time frame: Baseline, Month 12
Change from Baseline to Month 12 in Fasting HDL-C
Time frame: Baseline, Month 12
Number of Participants who Meet European Union (EU) LDL-C Apheresis Eligibility Criteria (per German Apheresis Working Group) at Month 12
Time frame: Month 12
Number of Participants who Meet United States (US) Apheresis Eligibility Criteria (per National Lipid Association) at Month 12
Time frame: Month 12
Number of Participants with Fasting LDL-C <100 mg/dL at Month 12
Time frame: Month 12
Percent Change from Baseline in Fasting LDL-C Over Time
Time frame: Baseline, up to Month 12
Change from Baseline in Fasting LDL-C Over Time
Time frame: Baseline, up to Month 12
Number of Participants with Treatment-Emergent Adverse Events (TEAEs)
Time frame: Up to 27.5 months