University of Rochester Medical Center, 1351 Mt. Hope Ave., Ste. 223, Rochester, NY 14620, USA.
Neurology. 2007 Jan 2;68(1):20-8. doi: 10.1212/01.wnl.0000250355.28474.8e.
To determine if future studies of coenzyme Q(10) and GPI-1485 in Parkinson disease (PD) may be warranted.
We conducted a randomized, double-blind, calibrated futility clinical trial of coenzyme Q10 and GPI-1485 in early untreated PD using placebo data from the DATATOP study to establish the futility threshold.
The primary outcome measure (change in total Unified Parkinson's Disease Rating Scale scores over 1 year) did not meet the prespecified criteria for futility for either agent. Secondary analyses using calibration controls and other more recent placebo data question the appropriateness of the predetermined definition of futility, and suggest that a more restrictive threshold may be needed.
Coenzyme Q(10) and GPI-1485 may warrant further study in Parkinson disease, although the data are inconsistent. Additional factors (cost, availability of other agents, more recent data on placebo outcomes, other ongoing trials) should also be considered in the selection of agents for Phase III studies.
确定辅酶Q(10)和GPI-1485在帕金森病（PD）中的后续研究是否有必要。
我们使用DATATOP研究中的安慰剂数据进行了一项关于辅酶Q10和GPI-1485在早期未经治疗的PD中的随机、双盲、校准无效性临床试验，以确定无效性阈值。
主要结局指标（1年内帕金森病统一评分量表总分的变化）对任何一种药物均未达到预先设定的无效性标准。使用校准对照和其他更新的安慰剂数据进行的二次分析对预先确定的无效性定义的适当性提出了质疑，并表明可能需要更严格的阈值。
辅酶Q(10)和GPI-1485在帕金森病中可能值得进一步研究，尽管数据并不一致。在选择用于III期研究的药物时，还应考虑其他因素（成本、其他药物的可获得性、关于安慰剂结局的更新数据、其他正在进行的试验）。
