Why the study?
Severely elevated LDL-C in HoFH starts in utero and accelerates atherosclerosis, creating an unmet need for aggressive LDLR-independent lipid-lowering therapies in pediatric patients.
Does intravenous evinacumab reduce LDL-C in pediatric patients with homozygous familial hypercholesterolemia inadequately controlled on optimized lipid-lowering therapy?
Population
14 patients 5 to 11 years of age with HoFH and LDL-C >130 mg/dL
Comparison
Intravenous evinacumab 15 mg/kg every 4 weeks
Design
Phase 3 open-label study
Follow-up
24 weeks
Authors
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May support LDL-C lowering with evinacumab in young HoFH patients; leaves open need for randomized outcome trials.
Does intravenous evinacumab reduce LDL-C in pediatric patients with homozygous familial hypercholesterolemia inadequately controlled on optimized lipid-lowering therapy?
Evinacumab safely and effectively reduces LDL-C by nearly half in pediatric patients aged 5 to 11 years with homozygous familial hypercholesterolemia who are inadequately controlled on optimized lipid-lowering therapy.
Wiegman et al. (2023) studied this question.