Clinical Center for Neuroprotection in Parkinson Disease

描述(申请人提供)：帕金森氏病是一种原因不明的不可抗拒的进行性疾病，黑质神经元进行性退化，导致大脑多巴胺缺乏的程度越来越严重。虽然已经开发了一些改善神经化学缺陷的治疗方法，但还没有任何治疗方法在人类身上证明可以延缓疾病的进展(与掩饰症状区分开来)。这样的效果是非常可取的，因为如果能够实现这一点，就可以显著推迟患者的临床恶化。这项竞争性延续提案旨在继续将德克萨斯大学西南医学中心作为正在进行的Net-PD临床试验的临床地点。其中包括正在进行的LS-1研究和FS-ZONE研究。LSI是一项为期5年的双盲、安慰剂对照研究，目的是将肌酸作为治疗帕金森病的可能的疾病改良剂。FS-ZONE是一项为期44周的无效性试验，评估吡格列酮作为帕金森病可能的疾病改良剂。 德克萨斯州达拉斯的德克萨斯大学西南医学中心非常适合继续其作为Net-PD网络的临床站点的工作。它是北得克萨斯州的主要学术转介中心，其四名神经病学家在帕金森氏症临床试验的设计、实施和报告方面经验丰富。如果获得资金，该网站将继续积极跟踪目前在NET-PD LS1试验中登记的49名受试者，并将继续招募和跟踪正在进行的FS-ZONE研究的受试者。虽然尚不清楚正在研究的两种药物是否会减缓帕金森病的进展，但这个独特的临床研究中心网络非常适合进行大规模、持续时间长、科学合理的临床试验，这些试验最终有望发现一种或多种可以减缓帕金森病退变过程的药物。

DESCRIPTION (provided by applicant): Parkinson's disease is an inexorably progressive disorder of unknown cause in which neurons of the substantia nigra progressively degenerate resulting in ever greater degrees of brain dopamine deficiency. While a number of treatments have been developed that improve the neurochemical deficit, no treatment has been demonstrated in humans that delays the progression of the disease (as distinguished from masking symptoms). Such an effect is highly desirable because if this could be achieved, a significant delay in clinical deterioration of patients could be realized. This competitive continuation proposal aims to continue UT Southwestern Medical Center as a Clinical Site for ongoing conduct of the NET-PD clinical trials. These include the ongoing LS-1 study and FS-ZONE study. LSI is a 5 year double-blind, placebo controlled investigation of creatine as a possible disease-modifying agent for PD. FS-ZONE is a 44 week futility trial assessing pioglitazone as a possible disease-modifying agent for PD. UT Southwestern Medical Center in Dallas, TX is well suited to continuing its work as a Clinical Site of the NET-PD network. It functions as the primary academic referral center for North Texas, and its four staff neurologists are experienced in the design, conduct, and reporting of clinical trials in Parkinson's disease. If funded, the site will continue its active follow-up of te 49 subjects currently enrolled in the NET-PD LS1 trial and will continue to recruit and follow subjects for the ongoing FS-ZONE study. While it is unclear if either agent under study will slow the progression of PD, this unique network of clinical research centers is ideally suited to perform large scale, long duration, scientifically sound clinical trials that have great promise fo ultimately discovering a drug or drugs that can slow the degenerative process of PD.