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

描述（由申请人提供）： 在过去的两个资助期内，帕金森研究所作为临床中心之一参加了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，该研究旨在确定肌酸是否比安慰剂更有效地减缓帕金森病（PD）患者早期治疗PD的临床下降。帕金森研究所已入组41例受试者进入LS-1试验，30例受试者仍在完成随访访视。主要结局将是一项纳入PD所致残疾多个维度的全球指标。假设是，与安慰剂相比，肌酸10克每天将与5年后较少的疾病进展相关。次要假设是，肌酐治疗组将具有更大的疗效和类似的安全性和耐受性安慰剂。帕金森研究所的具体目标将 （1）根据研究方案，使用药物临床试验质量管理规范，继续对所有活动患者进行随访，（2）关注所有受试者的保留，注意随着研究进展患者和护理人员需求的变化，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，检验研究假设至关重要。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病治疗中的一个关键空白，因为没有任何干预措施被明确证明可以减缓PD的进展。随着世界范围内疾病负担的增加，这成为一个关键的公共卫生问题。

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.