Limited Competition for Continuation of the NIH Exploratory Trials in PD

描述(申请人提供)：帕金森病(PD)是一种进行性神经退行性疾病，每100名65岁以上的人中有3人会受到影响。目前尚无治愈方法，病因尚不清楚。像左旋多巴这样的药物在几年内都有很好的益处。然而，疾病的发展会导致严重的残疾、生活质量下降和预期寿命缩短。Net-PD是由美国和加拿大的49家中心组成的联盟，由在PD临床试验方面具有专业知识的神经学家和研究协调员组成。它有 自2003年以来一直活跃，目的是开发一种治疗帕金森病的疾病修正疗法。该联盟已经完成了两项无效的试验，从而确定肌酸是有希望的化合物。这导致了一项大型简单试验(LS-1)的启动。主要目的是确定认知、身体和生活质量测量相结合的PD患者的临床衰退是否有减缓的趋势。在多巴胺能治疗和常规医疗的背景下，肌酸的积极治疗被比作安慰剂对照。2007-2010年间有超过1700名患者入选。试验将再持续3年，直到所有患者至少有5年的随访。因此，保留是这项试验最后3年的主要目的。在卡尔加里大学，我们招募了44名患者，保留率为98%。作为一家拥有25年以上临床试验经验的网站，我们有信心能够确保试验成功，并具有出色的保持性。卡尔加里大学的调查人员和协调员是许多委员会的积极成员，包括数据分析、手稿写作和保留战略。这项试验是P的同类试验中规模最大的。如果肌酸被证明具有神经保护作用，这将是第一个被证明减缓神经退行性疾病临床进展的化合物。它将改变我们管理帕金森氏症的方式，并为这种不治之症的患者带来希望。其次，这是关于PD收集的最大数据集，该联盟的所有调查人员都可以获得。它将提供关于PD的广泛信息。

DESCRIPTION (provided by applicant): Parkinson disease (PD) is a progressive neurodegenerative disorder affecting 3/100 people over the age of 65. There is no cure and etiology is unknown. Medications such as levodopa provide good benefit for a number of years. However, disease progression results in significant disability, poor quality of life, and shortened life expectancy. NET-PD is a consortium of 49 Centre's in the US and Canada comprised of neurologists and research coordinators with expertise in carrying out clinical trials in PD. It has been active since 2003 with the aim of developing a disease modifying treatment for PD. The consortium has already completed 2 futility trials, resulting in the identification of creatine as promising compound. This has led to the initiation of a large simple trial (LS-1). The primary objective was to determine if there is a slowing of clinical decline in PD patients defined by a combination of cognitive, physical, and quality of life measures. Active treatment with creatine is being compared to a placebo control against a background of dopaminergic therapy and usual medical care. Over 1700 patients were enrolled between 2007-2010. The trial will continue for another 3 years until all patients have a minimum of 5 years follow up. Thus, retention is a major thrust of the last 3 years of the trial. At the University of Calgary, we have enrolled 44 patients with 98% retention. As a clinical trial site with over 25 years' experience, we feel confident in being able to ensure trial success, with excellent retention. The investigators and coordinators at the University of Calgary are active members in a number of committees, including data analysis, manuscript writing, and retention strategies. This trial is the largest of its kind in P. If creatine is shown to be neuroprotective, this will be the first compound shown to slow clinical progression in a neurodegenerative disorder. It will change the way we manage PD and bring hope to patients with this incurable disease. Secondly, this is the largest data set of information collected on PD, and is available to all investigators in the consortium. It will provide extensive information on PD.