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

doi:10.1002/mus.23904

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

School of Medicine

Research output: Contribution to journal › Article › peer-review

45 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 language	English (US)
Pages (from-to)	187-192
Number of pages	6
Journal	Muscle and Nerve
Volume	49
Issue number	2
DOIs	https://doi.org/10.1002/mus.23904
State	Published - Feb 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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10.1002/mus.23904

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Kissel, J. T., Elsheikh, B., King, W. M., Freimer, M., Scott, C. B., Kolb, S. J., Reyna, S. P., Crawford, T. O., Simard, L. R., Krosschell, K. J., Acsadi, G., Schroth, M. K., D'Anjou, G., Lasalle, B., Prior, T. W., Sorenson, S., Maczulski, J. A., & Swoboda, K. J. (2014). SMA valiant trial: A prospective, double-blind, placebo-controlled trial of valproic acid in ambulatory adults with spinal muscular atrophy. Muscle and Nerve, 49(2), 187-192. https://doi.org/10.1002/mus.23904

Kissel, JT, Elsheikh, B, King, WM, Freimer, M, Scott, CB, Kolb, SJ, Reyna, SP, Crawford, TO, Simard, LR, Krosschell, KJ, Acsadi, G, Schroth, MK, D'Anjou, G, Lasalle, B, Prior, TW, Sorenson, S, Maczulski, JA & Swoboda, KJ 2014, 'SMA valiant trial: A prospective, double-blind, placebo-controlled trial of valproic acid in ambulatory adults with spinal muscular atrophy', Muscle and Nerve, vol. 49, no. 2, pp. 187-192. https://doi.org/10.1002/mus.23904

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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.",

keywords = "Carnitine, Motor neuron disease, Spinal muscular atrophy, Valproic acid",

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

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doi = "10.1002/mus.23904",

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AU - Elsheikh, Bakri

AU - King, Wendy M.

AU - Freimer, Miriam

AU - Scott, Charles B.

AU - Kolb, Stephen J.

AU - Reyna, Sandra P.

AU - Crawford, Thomas O.

AU - Simard, Louise R.

AU - Krosschell, Kristin J.

AU - Acsadi, Gyula

AU - Schroth, Mary K.

AU - D'Anjou, Guy

AU - Lasalle, Bernard

AU - Prior, Thomas W.

AU - Sorenson, Susan

AU - Maczulski, Jo Anne

AU - Swoboda, Kathryn J.

PY - 2014/2

Y1 - 2014/2

N2 - 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.

AB - 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.

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KW - Motor neuron disease

KW - Spinal muscular atrophy

KW - Valproic acid

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

Abstract

Keywords

ASJC Scopus subject areas

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