Treatment Efficacy of 1-Octanol Compared to Placebo in Adults With Essential Tremor

Essential tremor is a very common movement disorder affecting approximately 1.4% of the population. Response to medications such as beta blockers and mysoline may be only partial or be accompanied by intolerable side effects. Roughly 80% of patients have significant tremor reduction from? ethanol although daily use of this as a treatment has potentially serious social and legal consequences. The leading hypothesis for the pathophysiology of essential tremor is the unmasking of spontaneous oscillation of neurons in the inferior olive. Both ethanol and 1-octanol have been shown to reduce these spontaneous oscillations in a animal model of essential tremor; however, 1-octanol does this at a dose much lower than an intoxicating dose suggesting that it may be useful in the treatment of essential tremor. Two pilot studies with 1-octanol in patients with essential tremor suggested it was both efficacious and safe. Objectives: The aim of the present study is to further assess efficacy of 1-octanol in essential tremor in a double-blinded, placebo-controlled study. Study population: Patients with alcohol-responsive essential tremor. Design: Double-blind, placebo-controlled two-period cross-over design. Outcome parameters: Primary outcome parameter is tremor as assessed by accelerometry, secondary outcome measures are spiral drawing, subjective and clinical scoring. Estimate of the outcome: 1-octanol at the dose of 32mg/kg three times a day will significantly improve tremor in patients with alcohol-responsive essential tremor. Anticipated risks and benefits: Besides the unusual taste, there are no anticipated risks associated with 1-octanol ingestion. Patients may experience an improvement of their tremor. Potential meaning for the field: Better treatment of essential tremor.