Effects of Coenzyme Q10 in Parkinson Disease-Phase 3

描述（由申请人提供）：帕金森病（PD）是一种进行性神经退行性疾病，影响超过1，000，000名美国人。在NIH支持的II期试验中，我们的研究小组证明，用高剂量辅酶Q10（300，600和1200 mg/d）治疗早期未经治疗的PD患者是安全的，耐受性良好，并且通过统一帕金森病评定量表（UNIPES）测量，治疗有减缓进行性损害的积极趋势。在预先指定的次要分析中，我们注意到辅酶Q10的最高剂量是最有效的剂量，并且用辅酶Q10治疗有助于PD患者保持独立性，如Schwab和England量表所测量的。在拟议的研究中，我们将进行一项前瞻性、随机、安慰剂对照、双盲的辅酶Q10临床试验，以确认和扩展我们II期研究的结果。我们计划招募600例早期PD且尚不需要多巴胺能药物治疗的受试者。受试者将被随机分配接受安慰剂、辅酶Q10 1200 mg/d或2400 mg/d，在筛选、基线、第1、4、8、12和16个月访视时进行评估，并使用CAFRS进行评估。研究者将确定受试者是否达到需要多巴胺能药物治疗的残疾。我们的主要分析将通过分析受试者达到需要多巴胺能药物治疗的残疾时或16个月访视时的总BPRS评分变化来确认我们II期研究中发现的获益。我们还将分析Schwab和England量表以及PD生活质量量表数据，这些数据收集到受试者达到需要多巴胺能药物治疗的残疾或16个月访视时。我们将确定基线和第1、8和16个月访视时的平均血浆辅酶Q10水平，并确定平均值是否与总APORS评分恶化的减少相关。

DESCRIPTION (provided by applicant): Parkinson's Disease (PD) is a progressive, neurodegenerative disease, which affects over 1,000,000 Americans. In an NIH-supported phase II trial, our group demonstrated that treatment of patients with early, untreated PD with high dosages of coenzyme Q10 (300, 600 and 1200 mg/d) was safe and well tolerated and that there was a positive trend for treatment to slow the progressive impairment as measured by the Unified Parkinson Disease Rating Scale (UPDRS). In pre-specified secondary analyses, we noted that the highest dosage of coenzyme Q10 was the most effective dosage and that treatment with coenzyme Q10 helped the PD patients maintain independence, as measured by Schwab and England Scale. In the proposed study, we will conduct a prospective, randomized, and placebo-controlled, double-blind phase clinical trial of coenzyme Q10 to attempt to confirm and extend the results of our phase II study. We propose to enroll 600 subjects who have early PD and do not yet require treatment with dopaminergic agents. Subjects will be randomly assigned to receive placebo, coenzyme Q10 1200 mg/d or 2400 mg/d, evaluated at screening, baseline, months 1, 4, 8, 12, and 16 visits and assessed with the UPDRS. The investigator will determine whether the subject has reached disability requiring treatment with a dopaminergic agent. Our primary analysis will undertake to confirm the benefit found in our phase II study by analyzing the change in total UPDRS score to the point that the subjects reach disability requiring treatment with a dopaminergic agent or to the 16 month visit. We will also analyze the Schwab and England Scale and the PD Quality of Life Scale data collected to the point that the subjects reach disability requiring treatment with a dopaminergic agent or to the 16 month visit. We will determine the mean plasma coenzyme Q10 levels at baseline and at visits 1, 8 and 16 months, and determine whether the mean correlates with reduction in worsening of the total UPDRS score.