title: Alirocumab in patients with heterozygous familial hypercholesterolemia undergoing lipoprotein apheresis: Rationale and design of the ODYSSEY ESCAPE trial.
abstract: 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.
  
  
  
  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.
  
  
  
  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 regular weekly (QW; for ≥4 weeks) or Q2W (for ≥8 weeks) lipoprotein apheresis. Patients will be randomly assigned (2:1, respectively) to receive alirocumab 150 mg subcutaneously Q2W or placebo subcutaneously Q2W (both in 1-mL injections) for 18 weeks. From day 1 to week 6, the apheresis frequency will be fixed to the individual patient's established schedule (QW or Q2W); thereafter, apheresis will be performed according to the LDL-C value at that visit: apheresis will not be performed when the LDL-C value is ≥30% lower than the baseline pre-apheresis LDL-C value. The primary end point is the frequency of apheresis treatments over a 12-week period starting at week 7.
  
  
  
  The ODYSSEY ESCAPE trial will determine whether alirocumab reduces the frequency of lipoprotein apheresis in patients with HeFH.
authors.affiliation: Division of Clinical Pharmacology, Department of Internal Medicine, University of Kansas Medical Center, Kansas City, KS, USA. Electronic address: pmoriart@kumc.edu.
authors.forename: Patrick M
authors.initials: PM
authors.lastname: Moriarty
authors.affiliation: Medical Department II, Grosshadern, University Munich, Munich, Germany.
authors.forename: Klaus G
authors.initials: KG
authors.lastname: Parhofer
authors.affiliation: Metabolic Leader, LLC, Scarborough, PA, USA.
authors.forename: Stephan P
authors.initials: SP
authors.lastname: Babirak
authors.affiliation: University of Pennsylvania, Philadelphia, PA, USA.
authors.forename: Emil
authors.initials: E
authors.lastname: deGoma
authors.affiliation: Knight Cardiovascular Institute, Oregon Health & Science University, Portland, OR, USA.
authors.forename: P Barton
authors.initials: PB
authors.lastname: Duell
authors.affiliation: Extracorporeal Treatment and Lipoprotein Apheresis Center, Department of Internal Medicine III, University Hospital Carl Gustav Carus, Technische Universitaet Dresden, Dresden, Germany.
authors.forename: Bernd
authors.initials: B
authors.lastname: Hohenstein
authors.affiliation: Apheresis Centrum Rostock, Rostock, Germany.
authors.forename: Wolfgang
authors.initials: W
authors.lastname: Ramlow
authors.affiliation: Mayo Clinic, Rochester, MN, USA.
authors.forename: Vinaya
authors.initials: V
authors.lastname: Simha
authors.affiliation: Charite-Universitätsmedizin Berlin-Campus Virchow-Klinikum, Berlin, Germany.
authors.forename: Elisabeth
authors.initials: E
authors.lastname: Steinhagen-Thiessen
authors.affiliation: Cardiology, Hartford Hospital, Hartford, CT, USA.
authors.forename: Paul D
authors.initials: PD
authors.lastname: Thompson
authors.affiliation: Medizinische Klinik und Poliklinik IV, Klinikum der Universität München, Munich, Germany.
authors.forename: Anja
authors.initials: A
authors.lastname: Vogt
authors.affiliation: Medical Department, Sanofi-Aventis Deutschland GmbH, Berlin, Germany.
authors.forename: Berndt
authors.initials: B
authors.lastname: von Stritzky
authors.affiliation: Regeneron Pharmaceuticals, Inc., Basking Ridge, NJ, USA.
authors.forename: Yunling
authors.initials: Y
authors.lastname: Du
authors.affiliation: Regeneron Pharmaceuticals, Inc., Tarrytown, NY, USA.
authors.forename: Garen
authors.initials: G
authors.lastname: Manvelian
mesh_terms: D000911:Antibodies, Monoclonal / Q000009:adverse effects / Q000627:therapeutic use*; D061067:Antibodies, Monoclonal, Humanized; D001781:Blood Component Removal / Q000009:adverse effects; D004305:Dose-Response Relationship, Drug; D004311:Double-Blind Method; D006801:Humans; D006938:Hyperlipoproteinemia Type II / Q000097:blood / Q000188:drug therapy / Q000628:therapy*; D008074:Lipoproteins / Q000097:blood*; D012449:Safety; D016896:Treatment Outcome
chemical_list: D000911:Antibodies, Monoclonal; D061067:Antibodies, Monoclonal, Humanized; D008074:Lipoproteins; C571059:alirocumab
keywords: Alirocumab; Familial hypercholesterolemia; Low-density lipoprotein cholesterol; Monoclonal antibody; Proprotein convertase subtilisin/kexin type 9