Hypercholestrolemia Clinical Trials

Cedars-Sinai Heart Institute is currently conducting the following hypercholestrolemia clinical trial:

• Treatment of Familial Hypercholesterolemia with Microsomal Triglyceride Transfer Protein Inhibitor AEGR-733


Treatment of Familial Hypercholesterolemia with Microsomal Triglyceride Transfer Protein Inhibitor AEGR-733

Principal Investigator: P. K. Shah, MD

IRB Approval Number: 15211

Contact: Colleen Sam (310) 248-7651

Status: Enrolling

Summary: This is a phase III open label clinical trial to evaluate both the efficacy and long term
safety of AEGR-733 at the maximum tolerated dose. Twenty-five (25) subjects with already diagnosed familial high cholesterolemia and who test positive for homozygous familial hypercholesterolemia (hoHF) on a one time genetic screening test will be dosed with AEGR-733 for 26 weeks (efficacy phase) and then an additional 52 weeks (safety phase) at the maximum tolerated dose determined during the efficacy phase.  Study drug is in addition to their current lipid lowering therapy (medications and, in most cases, LDL-apheresis).  AEGR-733 is a potent inhibitor of human MTP (microsomal transfer protein). In vitro, AEGR-733 inhibits lipid transfer by directly binding to MTP. In cell culture studies, the IC50 for inhibition of apoB secretion by AEGR-733 was much lower than that for apo AI secretion (0.8 nanoM vs 6.5 μM), indicating that the compound is a highly selective inhibitor of apo B secretion