进展性多发性硬化症临床试验设计。

Clinical trial design for progressive MS trials.

机构信息

Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health, University of Genoa, Genoa, Italy/Policlinic Hospital San Martino-IST, Genoa, Italy.

Department of Biostatistics, University of Alabama at Birmingham, Birmingham, AL, USA.

出版信息

Mult Scler. 2017 Oct;23(12):1642-1648. doi: 10.1177/1352458517729461.

DOI:10.1177/1352458517729461

PMID:29041873

Abstract

The design of clinical trials is a key aspect to maximizing the possibility to detect a treatment effect. This fact is particularly challenging in progressive multiple sclerosis (PMS) studies due to the uncertainty about the right target and/or outcome in phase-2 studies. The aim of this review is to evaluate the current challenges facing the design of clinical trials for PMS. The selection of patients, the instrumental and clinical outcomes that can be used in PMS trials, and issues in their design will be covered in this report.

摘要

临床试验的设计是最大限度地提高检测治疗效果可能性的关键方面。由于在 2 期研究中对于正确的目标和/或结果存在不确定性，因此这一事实在进展性多发性硬化症（PMS）研究中特别具有挑战性。本综述的目的是评估目前 PMS 临床试验设计所面临的挑战。本报告将涵盖 PMS 试验中患者的选择、可使用的仪器和临床结果以及设计中的问题。