A Phase 3 Multicenter, Prospective, Open-Label Efficacy and Safety Study of Immune Globulin (Human) 10% Caprylate/Chromatography Purified in Patients with Myasthenia Gravis Exacerbations

Guntis Karelis, Rodica Balasa, Jan L. De Bleecker, Tima Stuchevskaya, Andres Villa, Philip Van Damme, Emmeline Lagrange, Jeannine M. Heckmann, Michael Nicolle, Crisandra Vilciu, Vera Bril, Elsa Mondou, Rhonda Griffin, Junliang Chen, Waleska Henriquez, Beatriz Garcia, Sandra Camprubi, Jaume Ayguasanosa

Research output: Contribution to journalArticlepeer-review

4 Citations (Scopus)

Abstract

Background: Myasthenia gravis (MG) is an autoimmune disorder affecting neuromuscular transmission. Exacerbations may involve increasing bulbar weakness and/or sudden respiratory failure, both of which can be critically disabling. Management of MG exacerbations includes plasma exchange and intravenous immunoglobulin (IVIG); they are equally effective, but patients experience fewer side effects with IVIG. The objective of this study was to assess the efficacy and safety of immune globulin caprylate/chromatography purified (IGIV-C) in subjects with MG exacerbations. Methods: This prospective, open-label, non-controlled 28-day clinical trial was conducted in adults with MG Foundation of America class IVb or V status. Subjects received IGIV-C 2 g/kg over 2 consecutive days (1 g/kg/day) and were assessed for efficacy/safety on Days 7, 14, 21, and 28. The primary efficacy endpoint was the change from Baseline in quantitative MG (QMG) score to Day 14. Secondary endpoints of clinical response, Baseline to Day 14, included at least a 3-point decrease in QMG and MG Composite and a 2-point decrease in MG-activities of daily living (MG-ADL). Results: Forty-nine subjects enrolled. The change in QMG score at Day 14 was significant (p < 0.001) in the Evaluable (-6.4, n = 43) and Safety (-6.7, n = 49) populations. Among evaluable subjects, Day 14 response rates were 77, 86, and 88% for QMG, MG Composite, and MG-ADL, respectively. IGIV-C showed good tolerability with no serious adverse events. Conclusions: The results of this study show that IGIV-C was effective, safe, and well tolerated in the treatment of MG exacerbations.

Original languageEnglish
Pages (from-to)223-230
Number of pages8
JournalEuropean Neurology
Volume81
Issue number5-6
DOIs
Publication statusPublished - 1 Nov 2019

Keywords

  • Exacerbations
  • Immune globulin caprylate/ chromatography purified
  • IV immune globulin
  • Myasthenia gravis

Field of Science

  • 3.2 Clinical medicine

Publication Type

  • 1.1. Scientific article indexed in Web of Science and/or Scopus database

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