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

被引:64
作者
Bingley, Polly J. [1 ]
Wherrett, Diane K. [2 ]
Shultz, Ann [3 ]
Rafkin, Lisa E. [4 ]
Atkinson, Mark A. [5 ,6 ]
Greenbaum, Carla J. [3 ]
机构
[1] Univ Bristol, Bristol Med Sch, Diabet & Metab, Bristol, Avon, England
[2] Univ Toronto, Hosp Sick Children, Dept Paediat, Toronto, ON, Canada
[3] Benaroya Res Inst, Diabet Res Program, Seattle, WA 98101 USA
[4] Univ Miami, Diabet Res Inst, Miami, FL USA
[5] Univ Florida, Coll Med, Dept Pathol, Gainesville, FL USA
[6] Univ Florida, Coll Med, Dept Pediat, Gainesville, FL USA
关键词
BETA-CELL FUNCTION; RANDOMIZED CONTROLLED-TRIAL; B-LYMPHOCYTE DEPLETION; DOUBLE-BLIND TRIAL; RECENT-ONSET; C-PEPTIDE; MULTIPLE AUTOANTIBODIES; MYCOPHENOLATE-MOFETIL; RHEUMATOID-ARTHRITIS; IMMUNE-RESPONSES;
D O I
10.2337/dc17-0806
中图分类号
R5 [内科学];
学科分类号
1002 ; 100201 ;
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.
引用
收藏
页码:653 / 661
页数:9
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