Continuation of NET-PD Trials at The Parkinson's Institute

描述(由申请者提供)：帕金森研究所在过去的两个资助期中作为临床中心之一参与了Net-PD计划。目前正在进行的LS-1试验是一项多中心双盲研究的一部分，目的是确定肌酸是否比安慰剂更有效地减缓早期治疗帕金森病(PD)患者的临床下降。帕金森研究所已经招募了41名受试者参加LS-1试验，30名受试者仍在完成随访。主要结果将是一项包含帕金森病所致残疾的多个维度的全球衡量标准。假设是，与安慰剂相比，与安慰剂相比，每天10克肌酸与5年后疾病进展较慢有关。第二个假设是肌酸治疗组将有更好的疗效以及与安慰剂相似的安全性和耐受性。帕金森研究所的具体目标将是 (1)根据研究方案，使用良好的临床实践，继续对所有活动患者进行随访，(2)专注于保留所有受试者，注意随着研究进展患者和护理人员不断变化的需求，以及(3)及时提供高质量的临床数据。这些目标对于准确和有效地分析研究数据、检验研究假设是必不可少的。帕金森研究所在临床研究方面的长期卓越记录将是我们努力的基础。LSI的成功完成将解决帕金森氏病治疗中的一个关键缺口，因为没有任何干预措施被明确证明可以减缓帕金森氏病的进展。随着全世界疾病负担的增加，这成为一个严重的公共卫生问题。

DESCRIPTION (provided by applicant): The Parkinson's Institute has participated as one of the clinical centers in the NET-PD program during the last two funding periods. The current LS-1 trial underway is part of a multi-center, double-blind, study to determine if creatine is more effective than placebo in slowing the clinical decline in Parkinson's disease (PD) patients with early, treated PD. The Parkinson's Institute has enrolled 41 subjects into LS-1 trial and 30 subjects are still completing follow-up visits. The primary outcome will be a global measure incorporating multiple dimensions of disability due to PD. The hypothesis is that creatine 10 grams daily will be associated with less disease progression after 5 years, when compared to placebo. Secondary hypotheses are that the creatine-treated group will have greater efficacy and similar safety and tolerability to placebo. The specific aims of the Parkinson's Institute will be (1) to continue follow-up of all active patients according to the study protocol, using good clinical practices, (2) to focus on retention of all subjects, being mindful of changing needs of patients and caregivers as the study progresses and (3) to provide high quality clinical data in a timely manner. These aims are essential to the accurate and efficient analysis of study data, testing the study hypotheses. The long track record of excellence in clinical research at the Parkinson's Institute will be the foundation of our effort. Successful completion of the LSI will address a critical gap in the treatment of Parkinson's disease, since no intervention has unequivocally been proven to slow progression of PD. As the burden of disease increases worldwide, this becomes a critical public health concern.