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Type 1 Diabetes TrialNet: A Multifaceted Approach to Bringing Disease-Modifying Therapy to Clinical Use in Type 1 Diabetes

机译:1型糖尿病TrialNet:一种将疾病改良疗法应用于1型糖尿病的临床研究的多方面方法

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摘要

What will it take to bring disease-modifying therapy to clinical use in type 1 diabetes? Coordinated efforts of investigators involved in discovery, translational, and clinical research operating in partnership with funders and industry and in sync with regulatory agencies are needed. This Perspective describes one such effort, Type 1 Diabetes TrialNet, a National Institutes of Health–funded and JDRF-supported international clinical trials network that emerged from the Diabetes Prevention Trial–Type 1 (DPT-1). Through longitudinal natural history studies, as well as trials before and after clinical onset of disease combined with mechanistic and ancillary investigations to enhance scientific understanding and translation to clinical use, TrialNet is working to bring disease-modifying therapies to individuals with type 1 diabetes. Moreover, TrialNet uses its expertise and experience in clinical studies to increase efficiencies in the conduct of trials and to reduce the burden of participation on individuals and families. Herein, we highlight key contributions made by TrialNet toward a revised understanding of the natural history of disease and approaches to alter disease course and outline the consortium’s plans for the future.
机译:将改善疾病的疗法应用于1型糖尿病的临床需要什么?需要与资助者和行业合作并与监管机构合作,参与发现,转化和临床研究的研究人员的协调努力。该观点描述了一种这样的努力,即1型糖尿病试验网,这是由美国国立卫生研究院资助并由JDRF支持的国际临床试验网络,该网络源自1型糖尿病预防试验(DPT-1)。通过纵向自然历史研究,以及疾病临床发作之前和之后的试验,并结合机械和辅助研究以增强对临床用途的科学理解和翻译,TrialNet致力于为1型糖尿病患者带来可改变疾病的疗法。此外,TrialNet利用其在临床研究中的专业知识和经验来提高试验的效率,并减轻个人和家庭参与的负担。在此,我们重点介绍TrialNet对修订疾病自然史和改变疾病进程的方法做出的重要贡献,并概述了财团的未来计划。

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