The next step in translational research: lessons learned from the first preclinical randomized controlled trial
The next step in translational research: lessons learned from the first preclinical randomized controlled trial
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DOI：
10.1111/jnc.13516
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发表时间：
2016-10-01
影响因子：
4.7
通讯作者：
Liesz, Arthur
中科院分区：
文献类型：
--
作者：
Llovera, Gemma;Liesz, Arthur
多年来，临床前试验的低重现性和有希望的临床前治疗向临床的转化较差，对大多数生物医学领域的转化研究构成了重大挑战。为了克服实验和临床研究之间的局限性，国际财团试图建立标准化的研究设计和报告结果数据的指导方针。此外，多中心临床前随机对照试验（pRCT）已被提议作为弥合实验研究与临床试验之间差距的合适工具。我们最近报告了第一个此类pRCT的设计和结果，其中我们证实了与独立跨国研究中心合作使用标准化方案的协调方法的可行性。然而，尽管取得了成功，但第一个pRCT也存在一些困难，特别是在遵循研究设计和分析数据中建立的方案方面。在这里，我们回顾了我们进行研究的经验，并分析和讨论了从执行第一个pRCT中吸取的教训。此外，我们提供了建议，如何克服障碍，以提高性能和未来的pRCT研究的结果。
For years, low reproducibility of preclinical trials and poor translation of promising preclinical therapies to the clinic have posed major challenges to translational research in most biomedical fields. To overcome the limitations that stand between experimental and clinical research, international consortia have attempted to establish standardized guidelines for study design and for reporting the resulting data. In addition, multicenter preclinical randomized controlled trials (pRCTs) have been proposed as a suitable tool for bridging the gap' between experimental research and clinical trials. We recently reported the design and results of the first such pRCT in which we confirmed the feasibility of using a coordinated approach with standardized protocols in collaboration with independent multinational research centers. However, despite its successes, this first pRCT also had several difficulties, particularly with respect to following the protocols established in the study design and analyzing the data. Here, we review our experiences performing the study, and we analyze and discuss the lessons learned from performing the first pRCT. Moreover, we provide suggestions regarding how obstacles can be overcome to improve the performance and outcome of future pRCT studies.