Parkinson's Disease Neuroprotection Trial

描述(由申请人提供)：Net-PD计划使用了一种合理、科学的方法来选择和评估预防帕金森氏病(PD)进展的潜在疗法。自最初获奖以来，Net-PD计划已经成功完成了两项无效性研究，即无效性研究1(FS-1)和无效性研究2(FS-TOO)，这两项研究评估了四种化合物(肌酸、米诺环素、辅酶Q10(CoQ10)和GPI-1485)的治疗潜力[5，6]。FS-1和FS-TOO的结果为一项大型、简单的临床试验的设计提供了依据，该试验旨在研究肌酸治疗在减缓早期治疗的帕金森氏病(即LS1)参与者临床衰退方面的有效性。LS1是一个多中心。第三阶段，肌酸预防帕金森病进展的双盲临床试验，该试验测试了这样一种假设，即在多巴胺能治疗和最佳帕金森病护理的背景下，每天服用肌酸(10克/天)在减缓帕金森病临床下降方面比安慰剂更有效，从基线到5年的随访。

DESCRIPTION (provided by applicant): The NET-PD program used a rational, scientific approach for the selection and evaluation of potential therapies for the prevention of Parkinson's disease (PD) progression. Since its original award, the NET-PD program has successfully completed two futility studies, known as Futility Study 1 (FS-1) and Futility Study 2 (FS-TOO) which evaluated the therapeutic potential of four compounds (creatine, minocycline, coenzyme Q10 (CoQ10), and GPI-1485) [5, 6]. The results of FS-1 and FS-TOO informed the design of a large, simple clinical trial for efficacy of creatine treatment in slowing clinical decline in participants with early, treated Parkinson's disease, known as LS1. LS1 is a multi-center. Phase 3, double-blind clinical trial of creatine for the prevention of PD progression that tests the hypothesis that daily administration of creatine (10gm/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.