SMA valiant trial: A prospective, double-blind, placebo-controlled trial of valproic acid in ambulatory adults with spinal muscular atrophy

John T. Kissel*, Bakri Elsheikh, Wendy M. King, Miriam Freimer, Charles B. Scott, Stephen J. Kolb, Sandra P. Reyna, Thomas O. Crawford, Louise R. Simard, Kristin J. Krosschell, Gyula Acsadi, Mary K. Schroth, Guy D'Anjou, Bernard Lasalle, Thomas W. Prior, Susan Sorenson, Jo Anne Maczulski, Kathryn J. Swoboda

*Corresponding author for this work

Research output: Contribution to journalArticle

43 Scopus citations

Abstract

Introduction: An open-label trial suggested that valproic acid (VPA) improved strength in adults with spinal muscular atrophy (SMA). We report a 12-month, double-blind, cross-over study of VPA in ambulatory SMA adults. Methods: There were 33 subjects, aged 20-55 years, included in this investigation. After baseline assessment, subjects were randomized to receive VPA (10-20 mg/kg/day) or placebo. At 6 months, patients were switched to the other group. Assessments were performed at 3, 6, and 12 months. The primary outcome was the 6-month change in maximum voluntary isometric contraction testing with pulmonary, electrophysiological, and functional secondary outcomes. Results: Thirty subjects completed the study. VPA was well tolerated, and compliance was good. There was no change in primary or secondary outcomes at 6 or 12 months. Conclusions: VPA did not improve strength or function in SMA adults. The outcomes used are feasible and reliable and can be employed in future trials in SMA adults.

Original languageEnglish (US)
Pages (from-to)187-192
Number of pages6
JournalMuscle and Nerve
Volume49
Issue number2
DOIs
StatePublished - Feb 1 2014

Keywords

  • Carnitine
  • Motor neuron disease
  • Spinal muscular atrophy
  • Valproic acid

ASJC Scopus subject areas

  • Physiology
  • Clinical Neurology
  • Cellular and Molecular Neuroscience
  • Physiology (medical)

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