Mehta L R, Schwid S R, Arnold D L, Cutter G R, Aradhye S, Balcer L J, Calabresi P A, Cohen J A, Cole P E, Glanzman R, Goelz S, Inglese M, Kapoor R, Kappos L, Kreitman R, Lublin F D, Mann A, Marrie R A, O'Looney P, Polman C H, Ravina B M, Reingold S C, Richert J R, Sandrock A W, Waubant E
University of Rochester Department of Neurology, Rochester, NY, USA.
Mult Scler. 2009 May;15(5):542-6. doi: 10.1177/1352458508101939.
There is considerable interest in tissue-protective treatments for multiple sclerosis (MS).
We convened a group of MS clinical trialists and related researchers to discuss designs for proof of concept studies utilizing currently available data and assessment methods.
Our favored design was a randomized, double-blind, parallel-group study of active treatment versus placebo focusing on changes in brain volume from a post-baseline scan (3-6 months after starting treatment) to the final visit 1 year later. Study designs aimed at reducing residual deficits following acute exacerbations are less straightforward, depending greatly on the anticipated rapidity of treatment effect onset.
The next step would be to perform one or more studies of potential tissue-protective agents with these designs in mind, creating the longitudinal data necessary to refine endpoint selection, eligibility criteria, and sample size estimates for future trials.
对于多发性硬化症（MS）的组织保护治疗有相当大的兴趣。
我们召集了一组MS临床试验人员和相关研究人员，讨论利用现有数据和评估方法进行概念验证研究的设计。
我们青睐的设计是一项活性治疗与安慰剂的随机、双盲、平行组研究，重点关注从基线扫描后（开始治疗3 - 6个月）到1年后最后一次访视时脑容量的变化。旨在减少急性加重后残留缺陷的研究设计不太直接，很大程度上取决于预期的治疗效果起效速度。
下一步将是考虑这些设计对一种或多种潜在组织保护剂进行一项或多项研究，生成必要的纵向数据，以完善未来试验的终点选择、入选标准和样本量估计。
