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AN OPEN-LABEL, SINGLE-ARM, PROOF-OF-CONCEPT STUDY TO EVALUATE THE SAFETY AND EFFICACY OF SINGLE AND MULTIPLE DOSES OF REGN1500 IN PATIENTS WITH HOMOZYGOUS FAMILIAL HYPERCHOLESTEROLEMIA

Please refer to protocol, section 1.2 "Rationale"

A Three-Part, Single-Arm, Open-Label Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of Evinacumab in Pediatric Patients with Homozygous Familial Hypercholesterolemia

PrimaryThe primary objective for Part A of the study is to assess the pharmacokinetics (PK) of evinacumab in pediatric patients with homozygous familial hypercholesterolemia (HoFH).The primary objective for Part B of the study is to demonstrate a…

An open-label study to evaluate the long-term safety and efficacy of evinacumab in patients with homozygous familial hypercholesterolemia

The primary objectives of the study are- To evaluate the long-term safety and tolerability of evinacumab 15 mg/kg intravenous (IV) administered every 4 weeks (Q4W) in patients with homozygous familial hypercholesterolemia (HoFH).- To evaluate the…