Skip to main content
Journal cover image

Clinical trial of L-Carnitine and valproic acid in spinal muscular atrophy type I.

Publication ,  Journal Article
Krosschell, KJ; Kissel, JT; Townsend, EL; Simeone, SD; Zhang, RZ; Reyna, SP; Crawford, TO; Schroth, MK; Acsadi, G; Kishnani, PS; Hero, B ...
Published in: Muscle Nerve
February 2018

INTRODUCTION: The aim of this study was to determine the safety and therapeutic potential of L-carnitine and valproic acid (VPA) in infants with spinal muscular atrophy (SMA). METHODS: Our investigation was an open-label phase 2 multicenter trial of L-carnitine and VPA in infants with SMA type I with retrospective comparison to an untreated, matched cohort. Primary outcomes were: safety and adverse events; secondary outcomes were survival, time to death/>16 hours/day of ventilator support; motor outcomes; and maximum ulnar compound motor action potential amplitude. RESULTS: A total of 245 AEs were observed in 35 of the 37 treated subjects (95%). Respiratory events accounted for 49% of all adverse events, resulting in 14 deaths. Survival was not significantly different between treated and untreated cohorts. DISCUSSION: This trial provides evidence that, in infants with SMA type I, L-carnitine/VPA is ineffective at altering survival. The substantial proportion of infants reaching end-points within 6 months of enrollment underscores the urgent need for pre-symptomatic treatment in SMA type I. Muscle Nerve 57: 193-199, 2018.

Duke Scholars

Altmetric Attention Stats
Dimensions Citation Stats

Published In

Muscle Nerve

DOI

EISSN

1097-4598

Publication Date

February 2018

Volume

57

Issue

2

Start / End Page

193 / 199

Location

United States

Related Subject Headings

  • Vitamin B Complex
  • Valproic Acid
  • Treatment Outcome
  • Survival Analysis
  • Spinal Muscular Atrophies of Childhood
  • Retrospective Studies
  • Respiration, Artificial
  • Neurology & Neurosurgery
  • Negative Results
  • Male
 

Citation

APA
Chicago
ICMJE
MLA
NLM
Krosschell, K. J., Kissel, J. T., Townsend, E. L., Simeone, S. D., Zhang, R. Z., Reyna, S. P., … Project Cure SMA Investigator’s Network, . (2018). Clinical trial of L-Carnitine and valproic acid in spinal muscular atrophy type I. Muscle Nerve, 57(2), 193–199. https://doi.org/10.1002/mus.25776
Krosschell, Kristin J., John T. Kissel, Elise L. Townsend, Sarah D. Simeone, Ren Zhe Zhang, Sandra P. Reyna, Thomas O. Crawford, et al. “Clinical trial of L-Carnitine and valproic acid in spinal muscular atrophy type I.Muscle Nerve 57, no. 2 (February 2018): 193–99. https://doi.org/10.1002/mus.25776.
Krosschell KJ, Kissel JT, Townsend EL, Simeone SD, Zhang RZ, Reyna SP, et al. Clinical trial of L-Carnitine and valproic acid in spinal muscular atrophy type I. Muscle Nerve. 2018 Feb;57(2):193–9.
Krosschell, Kristin J., et al. “Clinical trial of L-Carnitine and valproic acid in spinal muscular atrophy type I.Muscle Nerve, vol. 57, no. 2, Feb. 2018, pp. 193–99. Pubmed, doi:10.1002/mus.25776.
Krosschell KJ, Kissel JT, Townsend EL, Simeone SD, Zhang RZ, Reyna SP, Crawford TO, Schroth MK, Acsadi G, Kishnani PS, Von Kleist-Retzow J-C, Hero B, D’Anjou G, Smith EC, Elsheikh B, Simard LR, Prior TW, Scott CB, Lasalle B, Sakonju A, Wirth B, Swoboda KJ, Project Cure SMA Investigator’s Network. Clinical trial of L-Carnitine and valproic acid in spinal muscular atrophy type I. Muscle Nerve. 2018 Feb;57(2):193–199.
Journal cover image

Published In

Muscle Nerve

DOI

EISSN

1097-4598

Publication Date

February 2018

Volume

57

Issue

2

Start / End Page

193 / 199

Location

United States

Related Subject Headings

  • Vitamin B Complex
  • Valproic Acid
  • Treatment Outcome
  • Survival Analysis
  • Spinal Muscular Atrophies of Childhood
  • Retrospective Studies
  • Respiration, Artificial
  • Neurology & Neurosurgery
  • Negative Results
  • Male