Alirocumab in patients with heterozygous familial hypercholesterolemia undergoing lipoprotein apheresis: Rationale and design of the ODYSSEY ESCAPE trial Patrick M. Moriarty, MD * ,1 , Klaus G. Parhofer, MD 1 , Stephan P. Babirak, MD, PhD, Emil deGoma, MD, P. Barton Duell, MD, Bernd Hohenstein, MD, Wolfgang Ramlow, MD, Vinaya Simha, MD, Elisabeth Steinhagen-Thiessen, MD, Paul D. Thompson, MD, Anja Vogt, MD, Berndt von Stritzky, MD, Yunling Du, PhD, Garen Manvelian, MD Division of Clinical Pharmacology, Department of Internal Medicine, University of Kansas Medical Center, Kansas City, KS, USA (Dr Moriarty); Medical Department II, Grosshadern, University Munich, Munich, Germany (Dr Parhofer); Metabolic Leader, LLC, Scarborough, PA, USA (Dr Babirak); University of Pennsylvania, Philadelphia, PA, USA (Dr deGoma); Knight Cardiovascular Institute, Oregon Health & Science University, Portland, OR, USA (Dr Duell); Extracorporeal Treatment and Lipoprotein Apheresis Center, Department of Internal Medicine III, University Hospital Carl Gustav Carus, Technische Universitaet Dresden, Dresden, Germany (Dr Hohenstein); Apheresis Centrum Rostock, Rostock, Germany (Dr Ramlow); Mayo Clinic, Rochester, MN, USA (Dr Simha); Charite—Universit€atsmedizin Berlin–Campus Virchow–Klinikum, Berlin, Germany (Dr Steinhagen-Thiessen); Cardiology, Hartford Hospital, Hartford, CT, USA (Dr Thompson); Medizinische Klinik und Poliklinik IV, Klinikum der Universit€at M€unchen, Munich, Germany (Dr Vogt); Medical Department, Sanofi–Aventis Deutschland GmbH, Berlin, Germany (Dr von Stritzky); Regeneron Pharmaceuticals, Inc., Basking Ridge, NJ, USA (Dr Du); and Regeneron Pharmaceuticals, Inc., Tarrytown, NY, USA (Dr Manvelian) KEYWORDS: Alirocumab; Familial hypercholesterolemia; Low-density lipoprotein cholesterol; Monoclonal antibody; Proprotein convertase subtilisin/kexin type 9 BACKGROUND: Many patients with heterozygous familial hypercholesterolemia (HeFH) fail to reach optimal low-density lipoprotein cholesterol (LDL-C) levels with available lipid-lowering medications, including statins, and require treatment using alternative methods such as lipoprotein apheresis. OBJECTIVE: To evaluate the efficacy of alirocumab 150 mg every 2 weeks (Q2W) compared with placebo in reducing the frequency of lipoprotein apheresis treatments in patients with HeFH. METHODS: ODYSSEY ESCAPE is a randomized, double-blind, placebo-controlled, parallel-group, 18-week, phase 3 study being conducted in the United States and Germany. ODYSSEY ESCAPE will evaluate the efficacy and safety of alirocumab in approximately 63 adults with HeFH undergoing ClinicalTrials.gov identifier (clinicaltrials.gov): NCT02326220. 1 These authors contributed equally to this work and are joint first authors. * Corresponding author. University of Kansas Medical Center, 3901 Rainbow Blvd, Kansas City, KS 66160, USA. E-mail address: pmoriart@kumc.edu Submitted October 22, 2015. Accepted for publication February 1, 2016. 1933-2874/Ó 2016 National Lipid Association. All rights reserved. http://dx.doi.org/10.1016/j.jacl.2016.02.003 Journal of Clinical Lipidology (2016) 10, 627–634