The primary objective of the study is to assess the pharmacodynamic effect of REGN727 on serum low-density lipoprotein cholesterol during 14 weeks of subcutaneously administered REGN727 in patients with autosomal dominant hypercholesterolemia and gain-of-function mutations (GOFm) in 1 or both alleles of the proprotein convertase subtilisin/kexin type 9 (PCSK9) gene.
The secondary objectives of the study are to assess in patients with PCSK9 GOFm:
1. The safety and tolerability of SC administered REGN727
2. The PD effect of REGN727 on other serum lipids/Apolipoproteins including: total cholesterol, HDL-C, non-HDL-C, very low-density lipoprotein cholesterol (VLDL-C), TG, ApoB100, ApoA1, and Lp(a)
3. The PK profile of multiple SC doses of REGN727
4. The immunogenicity profile in patients after 14 weeks of dosing with REGN727.
5. Patients will be required to provide a blood sample for genetic testing. Genes are made up of DNA, which is the substance in cells that carries genetic (inherited) information. The genes contain key instructions for cell function and help determine the characteristics of each individual. Patients are required to provide one DNA sample for the main study. This sample will be used only to test the genes PCSK9, LDLR, and APOB. You already had the DNA for PCSK9 tested. They play an important role in how much cholesterol circulates in your blood. We will test these genes again to confirm the results of the testing that was performed on them in the past.
6. After completing the first 15 visits (blinded portion), participants will be invited to continue the study drug as part of an open-label treatment study for approximately 3 years or until approval of the study drug. Participants will continue to receive 150 mg REGN727 to assess the long-term safety and durability of the effect of REGN727 on lipids and lipoproteins.
Principle Investigator: Paul Hopkins
Principle Department: Cardiovascular Genetics
Phone: (801) 581-3810