title: Efficacy and safety of abobotulinumtoxinA liquid formulation in cervical dystonia: A randomized-controlled trial.
abstract: Approved botulinum toxin A products require reconstitution. AbobotulinumtoxinA solution for injection is a ready-to-use liquid formulation of abobotulinumtoxinA.
  
  
  
  The objective of this study was to demonstrate the superior efficacy of abobotulinumtoxinA solution for injection to placebo and to test the noninferior efficacy of abobotulinumtoxinA solution for injection versus abobotulinumtoxinA (dry formulation) in cervical dystonia.
  
  
  
  This was a phase-3, multicenter, prospective, double-blind, randomized, active, and placebo-controlled study (N = 369). Patients with cervical dystonia were randomized (3:3:1) to abobotulinumtoxinA solution for injection 500 U, abobotulinumtoxinA 500 U, or placebo. Following the double-blind phase, patients received abobotulinumtoxinA solution for injection, open-label, for up to 4 cycles. The primary outcome was change from baseline at week 4 of the Toronto Western Spasmodic Torticollis Rating Scale total score. Secondary measures included change from baseline or cycle baseline in Toronto Western Spasmodic Torticollis Rating Scale scores.
  
  
  
  At week 4, both products were superior to placebo (Toronto Western Spasmodic Torticollis Rating Scale total score least square mean decrease from baseline, abobotulinumtoxinA solution for injection 500 U -12.5, abobotulinumtoxinA 500 U -14.0, placebo -3.9; P < .0001 vs placebo). The noninferiority limit of 3 points in the Toronto Western Spasmodic Torticollis Rating Scale total score at week 4 was not met for abobotulinumtoxinA solution for injection versus abobotulinumtoxinA. Toronto Western Spasmodic Torticollis Rating Scale total score reductions were maintained for up to 4 cycles of abobotulinumtoxinA solution for injection open-label follow-up treatment. Safety profiles of abobotulinumtoxinA solution for injection and abobotulinumtoxinA were similar, with dysphagia and injection-site pain the most frequent drug-related adverse events.
  
  
  
  Although the predefined noninferiority criterion was not met, abobotulinumtoxinA solution for injection was similarly effective to freeze-dried abobotulinumtoxinA in reducing Toronto Western Spasmodic Torticollis Rating Scale total scores with a similar safety profile. AbobotulinumtoxinA solution for injection efficacy was maintained with chronic open-label treatment, and this novel formulation may add convenience as well as dosing accuracy to treatment with abobotulinumtoxinA. © 2016 International Parkinson and Movement Disorder Society.
authors.affiliation: Department of Neurology, Innsbruck Medical University/University Hospital, Innsbruck, Austria.
authors.forename: Werner
authors.initials: W
authors.lastname: Poewe
authors.affiliation: Department of Clinical Neurophysiology, CHU de Bordeaux, Bordeaux, France.|Institut des Maladies Neurodégénératives, Université de Bordeaux, Bordeaux, France.
authors.forename: Pierre
authors.initials: P
authors.lastname: Burbaud
authors.affiliation: Department of Neurology, University Hospital Caremeau, Nimes, France.
authors.forename: Giovanni
authors.initials: G
authors.lastname: Castelnovo
authors.affiliation: Department of Neurology, University of Freiburg, Freiburg, Germany.
authors.forename: Wolfgang H
authors.initials: WH
authors.lastname: Jost
authors.affiliation: Schön Klinik München Schwabing, Department of Neurology, Technische Universität München, München, Germany.
authors.forename: Andres O
authors.initials: AO
authors.lastname: Ceballos-Baumann
authors.affiliation: Department of Neurology, Jagiellonian University Medical College, Kraków, Poland.
authors.forename: Marta
authors.initials: M
authors.lastname: Banach
authors.affiliation: Department of Neurology, Medical University of Warsaw, Warsaw, Poland.
authors.forename: Anna
authors.initials: A
authors.lastname: Potulska-Chromik
authors.affiliation: Clinical Pharmacology Unit, Instituto de Medicina Molecular, Faculty of Medicine, University of Lisbon, Lisbon, Portugal.
authors.forename: Joaquim J
authors.initials: JJ
authors.lastname: Ferreira
authors.affiliation: National Institute of Clinical Neurosciences, Budapest, Hungary.
authors.forename: Katalin
authors.initials: K
authors.lastname: Bihari
authors.affiliation: Department of Neurology, County Hospital Pardubice, Pardubice, Czech Republic.
authors.forename: Edvard
authors.initials: E
authors.lastname: Ehler
authors.affiliation: 1st Department of Neurology, Faculty of Medicine, Masaryk University, Brno, Czech Republic.|St Anne's University Hospital, Brno, Czech Republic.|Department of Neurology, Medical School, University of Minnesota, Minneapolis, Minnesota, USA.
authors.forename: Martin
authors.initials: M
authors.lastname: Bares
authors.affiliation: Dnipropetrovsk State Medical Academy, Dnipropetrovsk Regional Clinical Hospital Mechnykov, Mechnykov, Ukraine.|Department of Neurology, Zhovtneva, Ploshcha, Ukraine.
authors.forename: Lyudmyla A
authors.initials: LA
authors.lastname: Dzyak
authors.affiliation: Research Institute of Traumatology and Orthopaedics, Nizhnyi Novgorod, Russia.
authors.forename: Anna N
authors.initials: AN
authors.lastname: Belova
authors.affiliation: Ipsen Innovation, Les Ulis, France.
authors.forename: Emmanuel
authors.initials: E
authors.lastname: Pham
authors.affiliation: Ipsen Biopharmaceuticals Inc, Basking Ridge, New Jersey, USA.
authors.forename: Wenzhong Jerry
authors.initials: WJ
authors.lastname: Liu
authors.affiliation: Research Institute of Traumatology and Orthopaedics, Nizhnyi Novgorod, Russia.
authors.forename: Philippe
authors.initials: P
authors.lastname: Picaut
mesh_terms: D065087:Acetylcholine Release Inhibitors / Q000008:administration & dosage / Q000009:adverse effects / Q000494:pharmacology*; D000328:Adult; D019274:Botulinum Toxins, Type A / Q000008:administration & dosage / Q000009:adverse effects / Q000494:pharmacology*; D004311:Double-Blind Method; D005260:Female; D006801:Humans; D008297:Male; D008875:Middle Aged; D017063:Outcome Assessment, Health Care / Q000379:methods*; D011446:Prospective Studies; D012720:Severity of Illness Index; D014103:Torticollis / Q000188:drug therapy*
chemical_list: D065087:Acetylcholine Release Inhibitors; D019274:Botulinum Toxins, Type A; C542869:abobotulinumtoxinA
keywords: Toronto Western Spasmodic Torticollis Rating Scale; abobotulinumtoxinA solution for injection; cervical dystonia