Enrollment and Retainment Experiences in a Pilot Clinical Trial Testing Deep Brain Stimulation in Early Stage Parkinson's Disease (P06.090)

OBJECTIVE: To determine the factors that influenced patients9 participation in a clinical trial of deep brain stimulation (DBS) in early stage Parkinson9s disease (PD). BACKGROUND: We conducted the only FDA-approved study of DBS for early PD (IDE G050016, IRB 040797). Prior to initiation, ethical concerns centered on exposing subjects to surgical risks when their symptoms were well-controlled with medications. Upon commencement, additional concerns were that subjects would either not enroll in the study or be highly motivated to receive DBS and would, therefore, drop out of the study if randomized to medications. DESIGN/METHODS: 30 subjects (27 men, 60 ± 6.9 mean age) with early stage PD, Hoehn & Yahr ≤ 2 off medication, without motor fluctuations, were enrolled in a prospective, randomized, single-blind clinical trial testing the safety and tolerability of DBS. 15 subjects randomized to DBS were implanted and followed in parallel with subjects randomized to medications. Subjects were evaluated every 6 months for a period of 2 years. At study completion, 27 subjects completed a survey concerning the enrollment and study experience. RESULTS: Upon enrollment, 89% of subjects hoped to be randomized to DBS. Two medication and one DBS subject considered dropping out following randomization. The only subject to drop out cited financial hardship. The most common reasons for remaining in the study were to help future patients with PD through research participation and to honor their commitment. Two subjects in the medication group elected to undergo DBS after study completion and 3 others are considering DBS. CONCLUSIONS: This study demonstrates that patients will enroll and remain in a trial testing DBS in very early stage PD. While most subjects hoped to be randomized to the DBS group upon study entry, dropouts in either study arm were remarkable low. A large scale multicenter study of DBS in very early stage PD is feasible. Supported by: Medtronic, Inc., by Vanderbilt CTSA grant UL1TR000445 from the National Center for Advancing Translational Sciences (NCATS), by NCATS/NIH award UL1TR000011, by NIH R01 EB006136, and by private donations. Medtronic representatives did not take part in data collection, management, analysis, or interpretation of the data or in preparation, review, or approval of this abstract. Disclosure: Dr. Molinari has nothing to disclose. Dr. Currie has nothing to disclose. Dr. Turchan has nothing to disclose. Dr. Sidwell has nothing to disclose. Dr. Charles has received personal compensation for activities with Allergan, Inc., Ipsen, Medtronic, Inc., and the Alliance for Patient Access. Dr. Charles9 institution has received research support from Allergan, Inc., Ipsen, Merz Pharma, Medtronic, Inc., and UCB Pharma.