Progressive Staging of Pilot Studies to Improve Phase III Trials for Motor Interventions.
Progressive Staging of Pilot Studies to Improve Phase III Trials for Motor Interventions.
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DOI：
10.1177/1545968309331863
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发表时间：
2009-03
影响因子：
4.2
通讯作者：
Dobkin BH
中科院分区：
文献类型：
--
作者：
Dobkin BH
基于次优研究途径，最终导致跑步机训练与部分体重支持和机器人辅助设备的多中心随机临床试验（MRCT），提出了新的康复干预措施的试点研究的战略计划的连续阶段。在一个小的方便样本的参与者中描述实验干预，因此必须谨慎解释结果。第二阶段，概念发展试点，应优化干预的组成部分，确定最合适的结果措施，并检查剂量反应效应。一个设计良好的研究，显示没有疗效应该发表，以抵消确认偏倚的积极试验。第三阶段，概念示范试点，可以从已经学到的东西中建立起来，使用随机分配和盲法结果测量，在每组中测试至少15名参与者。对照组应接受旨在实现相同主要结局的积极实践干预。第三组可以接受更大剂量的实验治疗或联合干预。如果只有一个研究中心进行了本试验，则不同的研究组应旨在根据最佳运动训练剂量重现积极结局。第3阶段研究应建议效应量为0.4或更高，因此每组约50名受试者将是第4阶段概念验证MRCT中测试疗效所需的人数。通过就每个阶段可接受和必要的实践达成共识，类似于CONSORT对III期临床试验出版的建议，质量更好的试点研究可能会迅速进入设计更好和更成功的实验性干预措施MRCT。
Based on the suboptimal research pathways that finally led to multicenter randomized clinical trials (MRCTs) of treadmill training with partial body weight support and of robotic assistive devices, strategically planned successive stages are proposed for pilot studies of novel rehabilitation interventions Stage 1, consideration-of-concept studies, drawn from animal experiments, theories, and observations, delineate the experimental intervention in a small convenience sample of participants, so the results must be interpreted with caution. Stage 2, development-of-concept pilots, should optimize the components of the intervention, settle on most appropriate outcome measures, and examine dose-response effects. A well-designed study that reveals no efficacy should be published to counterweight the confirmation bias of positive trials. Stage 3, demonstration-of-concept pilots, can build out from what has been learned to test at least 15 participants in each arm, using random assignment and blinded outcome measures. A control group should receive an active practice intervention aimed at the same primary outcome. A third arm could receive a substantially larger dose of the experimental therapy or a combinational intervention. If only 1 site performed this trial, a different investigative group should aim to reproduce positive outcomes based on the optimal dose of motor training. Stage 3 studies ought to suggest an effect size of 0.4 or higher, so that approximately 50 participants in each arm will be the number required to test for efficacy in a stage 4, proof-of-concept MRCT. By developing a consensus around acceptable and necessary practices for each stage, similar to CONSORT recommendations for the publication of phase III clinical trials, better quality pilot studies may move quickly into better designed and more successful MRCTs of experimental interventions.
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影响因子：
4.2
作者：
Bowden, Mark G.;Balasubramanian, Chitralakshmi K.;Kautz, Steven A.
通讯作者：
Kautz, Steven A.
影响因子：
158.5
作者：
Dobkin, BH
通讯作者：
Dobkin, BH
影响因子：
3
作者：
Coote, Susan;Murphy, Brendan;Stokes, Emma
通讯作者：
Stokes, Emma
影响因子：
4.2
作者：
Dobkin, Bruce H.
通讯作者：
Dobkin, Bruce H.
影响因子：
4.2
作者：
Dobkin, BH;Apple, D;Scott, M
通讯作者：
Scott, M