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

Academic Article

Abstract

  • © 2012 Movement Disorder Society. Based on the preclinical data and the results of a phase II futility study, creatine was 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.
  • Authors

    Published In

  • Movement Disorders  Journal
  • Digital Object Identifier (doi)

    Author List

  • Elm JJ; Hauser R; Tilley BC; Kieburtz K; Aminoff MJ; Augustine E; Bennett S; Bodis-Wollner IG; Cambi F; Carter JH
  • Start Page

  • 1513
  • End Page

  • 1521
  • Volume

  • 27
  • Issue

  • 12