A placebo-controlled trial of gabapentin in spinal muscular atrophy

R. G. Miller, D. H. Moore, V. Dronsky, W. Bradley, R. Barohn, W. Bryan, T. W. Prior, D. F. Gelinas, S. Iannaccone, J. Kissel, R. Leshner, J. Mendell, M. Mendoza, B. Russman, F. Samaha, S. Smith

Research output: Contribution to journalArticlepeer-review

77 Scopus citations


Objective: To evaluate the efficacy of gabapentin in increasing muscle strength of patients with spinal muscular atrophy (SMA). Background: Preclinical data in experimental models of motor neuron disease suggest a neuroprotective effect of gabapentin. Methods: Gabapentin (1200 mg), or placebo, was administered three times daily in a randomized, double-blind trial for 12 months. The primary outcome measure was the average percent change from baseline, based on the measurement of strength in four muscles (elbow flexion and hand grip bilaterally) for each patient. Drug efficacy was examined by comparing the percent change in strength for patients on drug vs. placebo. Secondary efficacy variables included: forced vital capacity (FVC), SMA functional rating scale (SMAFRS), and mini-Sickness Impact Profile (SIP). Results: Eighty-four patients, with type II or III SMA, were enrolled at eight sites across the United States. There were no differences in baseline features. There was no difference between the placebo and drug groups in any outcome measure. Conclusions: This study demonstrates the feasibility of this trial design and provides data for the design of future clinical trials in SMA.

Original languageEnglish (US)
Pages (from-to)127-131
Number of pages5
JournalJournal of the Neurological Sciences
Issue number1-2
StatePublished - Oct 15 2001


  • Clinical trial
  • Gabapentin
  • Placebo-controlled trial
  • Randomized trial
  • Spinal muscular atrophy

ASJC Scopus subject areas

  • Neurology
  • Clinical Neurology


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