Affiliations: Department of Neurology, Vanderbilt University Medical Center, Nashville, TN, USA | Stritch School of Medicine, Loyola University Chicago, Maywood, IL, USA | Center for Biomedical Ethics, Vanderbilt University Medical Center, Nashville, TN, USA | Department of Psychology, Vanderbilt University Medical Center, Nashville, TN, USA | Department of Neurosurgery, Vanderbilt University Medical Center, Nashville, TN, USA | Department of Biostatistics, Vanderbilt University Medical Center, Nashville, TN, USA
Note:  Correspondence to: David Charles, MD, Professor and Vice-Chairman, Department of Neurology, Suite A-1106 Medical Center North, 1161 21st Avenue South, Nashville, TN 37232-2551, USA. Tel.: +1 615 936 2025; Fax: +1 615 936 2675; E-mail: email@example.com
Abstract: Background: Deep brain stimulation provides significant symptomatic benefit for people with advanced Parkinson's disease whose symptoms are no longer adequately controlled with medication. Preliminary evidence suggests that subthalamic nucleus stimulation may also be efficacious in early Parkinson's disease, and results of animal studies suggest that it may spare dopaminergic neurons in the substantia nigra. Objective: We report the methodology and design of a novel Phase I clinical trial testing the safety and tolerability of deep brain stimulation in early Parkinson's disease and discuss previous failed attempts at neuroprotection. Methods: We recently conducted a prospective, randomized, parallel-group, single-blind pilot clinical trial of deep brain stimulation in early Parkinson's disease. Subjects were randomized to receive either optimal drug therapy or deep brain stimulation plus optimal drug therapy. Follow-up visits occurred every six months for a period of two years and included week-long therapy washouts. Results: Thirty subjects with Hoehn & Yahr Stage II idiopathic Parkinson's disease were enrolled over a period of 32 months. Twenty-nine subjects completed all follow-up visits; one patient in the optimal drug therapy group withdrew from the study after baseline. Baseline characteristics for all thirty patients were not significantly different. Conclusions: This study demonstrates that it is possible to recruit and retain subjects in a clinical trial testing deep brain stimulation in early Parkinson's disease. The results of this trial will be used to support the design of a Phase III, multicenter trial investigating the efficacy of deep brain stimulation in early Parkinson's disease.
Keywords: Parkinson's disease, deep brain stimulation, subthalamic nucleus, research design