A Randomized, Double-Blind, Placebo-Controlled, Parallel Group Study to Evaluate the Efficacy and Safety of SAR236553/REGN727 in Patients With Heterozygous Familial Hypercholesterolemia and LDL-C higher or equal to 160mg/dL with Their Lipid-Modifying Therapy (Protocol EFC12732). Heterozygous Familial hypercholesterolemia (heFH) is an inherited disorder of lipid (fat) metabolism that predisposes a person to premature severe cardiovascular disease (CVD). Current Low Density Lipoprotein Cholesterol (LDL-C)-lowering medications include statins, cholesterol absorption inhibitors (ie, ezetimibe), fibrates, niacin, and bile acid sequestrants. Statins are the most commonly prescribed, as they have shown a greater ability to lower LDL-C and reduce CHD events. LDL-C collects in the walls of blood vessels, causing the blockages. Higher LDL levels put you at greater risk for a heart attack from a sudden blood clot in an artery. However, despite these available treatments that can be also used in combination, many high-risk patients fail to reach the guideline target level. Preliminary studies of the drug SAR236553/REGN727 have shown reductions in LDL-C by as much as 60% in this patient population. The objective of the present study is to assess the efficacy and safety (does it work and is it safe?) of SAR236553/REGN727 in patients with heFH and who need an improvement in their medication management of their high lipids. Two groups of patients will use either the experimental drug or placebo in addition to their normal lipid lowering medications. Subjects will be on study for approximately 18 months. Over this time they will have 13-14 study visits. Study visits will consist of an ECG, a physical exam, review of any side effects of the drug, health questionnaires, and a blood draw. Blood will be processed and sent to a central lab for analysis.
A Randomized, Double-Blind, Placebo-Controlled, Parallel Group Study to Evaluate the Efficacy and Safety of SAR236553/REGN727 in Patients With Heterozygous Familial Hypercholesterolemia…
095-12Principal Investigator: Conducted at:
Long Beach MemorialCurrently enrolling additional patients: