A placebo-controlled trial of gabapentin in spinal muscular atrophy.

To evaluate the efficacy of gabapentin in increasing muscle strength of patients with spinal muscular atrophy (SMA). Preclinical data in experimental models of motor neuron disease suggest a neuroprotective effect of gabapentin. 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). 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. This study demonstrates the feasibility of this trial design and provides data for the design of future clinical trials in SMA.