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

2013; Wiley; Volume: 49; Issue: 2 Linguagem: Inglês

10.1002/mus.23904

1097-4598

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

Neurogenetic and Muscular Disorders Research

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.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.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.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.