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Design innovations and baseline findings in a long-term Parkinson's trial: The national institute of neurological disorders and stroke exploratory trials in Parkinson's Disease Long-Term Study–1§

Authors

  • Jordan J. Elm,

    Corresponding author
    1. Division of Biostatistics and Epidemiology, Medical University of South Carolina, 135 Cannon Street, Suite 303, P.O. Box 250835, Charleston, SC 29425, USA
    • Division of Biostatistics and Epidemiology, Medical University of South Carolina, 135 Cannon Street, Suite 303, P.O. Box 250835, Charleston, SC 29425, USA
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  • The NINDS NET-PD Investigators


  • Funding agencies: This study was sponsored by the National Institutes of Health, National Institute of Neurological Disorders and Stroke.

  • Relevant conflicts of interest/financial disclosure: Nothing to report.

  • §

    Full financial disclosures and author roles may be found in the online version of this article.

Abstract

Based on the preclinical data and the results of a phase II futility study, creatine was selected for an efficacy trial in Parkinson's disease (PD). We present the design rationale and a description of the study cohort at baseline. A randomized, multicenter, double-blind, parallel-group, placebo-controlled phase III study of creatine (10 g daily) in participants with early, treated PD, the Long-term Study–1 (LS-1), is being conducted by the National Institute of Neurological Disorders and Stroke Exploratory Trials in Parkinson's Disease network. The study utilizes a global statistical test (GST) encompassing five clinical rating scales to provide a multidimensional assessment of disease progression. A total of 1,741 PD participants from 45 sites in the United States and Canada were randomized 1:1 to either 10 g of creatine/day or matching placebo. Participants are being evaluated for a minimum of 5 years. The LS-1 baseline cohort includes participants treated with dopaminergic therapy and generally mild PD. LS-1 represents the largest cohort of patients with early treated PD ever enrolled in a clinical trial. The GST approach should provide high power to test the hypothesis that daily administration of creatine (10 g/day) is more effective than placebo in slowing clinical decline in PD between baseline and the 5-year follow-up visit against the background of dopaminergic therapy and best PD care. © 2012 Movement Disorder Society

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