Electrophysiologic endpoint measures in a multicenter ALS drug trial.
We report the analysis of a battery of secondary electrophysiologic measurements to assess the progression of amyotrophic lateral sclerosis (ALS) in a two center, six month, double-blind, three arm trial comparing branched chain amino acids to L-threonine with pyridoxal 5-phosphate to placebo. The endpoint measurements were chosen to separately assess the effects of lower motor neuron loss and collateral reinnervation. For tests of inter-center reliability, we found no differences that could not be readily explained by variations in electrophysiologic testing techniques. Since the drug study was negative for the primary endpoint measure (muscle strength), we combined data from both centers and the three treatment arms. For measures of progression, all measures changed in the expected direction during the 6 months of the trial. We conclude that a battery of electrophysiologic measures can be used in a multicenter ALS drug trial to provide information on changes in lower motor neuron numbers and the effects of collateral reinnervation.
Department of Neurology, The University of Utah, Salt Lake City, UT, USA. mbromberg@hsc.utah.edu
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Bromberg
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Fries
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Forshew
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Tandan
D000597:Amino Acids, Branched-Chain / Q000627:therapeutic use; D000690:Amyotrophic Lateral Sclerosis / Q000188:drug therapy* / Q000473:pathology; D018450:Disease Progression; D004311:Double-Blind Method; D004594:Electrophysiology; D023381:Endpoint Determination; D006801:Humans; D009046:Motor Neurons / Q000502:physiology; D015203:Reproducibility of Results
D000597:Amino Acids, Branched-Chain