CLINICAL AFFILIATE OF THE TYPE 1 DIABETES TRIALNET

1 型糖尿病试验网的临床附属机构

• 批准号: 8776544
• 负责人: JENNIFER B MARKS
• 金额: $ 61.45万
• 依托单位: UNIVERSITY OF MIAMI SCHOOL OF MEDICINE
• 依托单位国家: 美国
• 财政年份: 2009
• 资助国家: 美国
• 起止时间: 2009-09-30 至 2019-04-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/8776544
• 关键词: Adolescent and Young Adult; Ancillary Study; Autoimmunity; C-Peptide; CD3 Antigens; Cell physiology; Cells; Child; Chronic; Clinical; Clinical Research; Clinical Trials; Dependence; Development; Diabetes Mellitus; Diagnosis; Disease; Enrollment; Environment; Funding; Future; Goals; Hyperglycemia; Individual; Insulin; Insulin-Dependent Diabetes Mellitus; International; Intervention; Life; Measures; Mediating; Molecular; Natural History; Onset of illness; Oral; Pancreas; Participant; Pathway interactions; Positioning Attribute; Prevention; Prevention strategy; Preventive Intervention; Process; Research; Research Personnel; Risk; Site; Structure of beta Cell of islet; Testing; Time; United States National Institutes of Health; autoreactive T cell; base; biobank; design; insulin secretion; islet; prevent; public health relevance; response; success

DESCRIPTION (provided by applicant): Type 1 diabetes (T1D) occurs in genetically predisposed individuals as a result of the progressive, selective destruction of pancreatic ¿-cells, which is primarily mediated by autoreactive T cells. The disease process results in loss of insulin secretion and life-long insulin-dependence, and unfortunately, most often occurs in children, adolescents, and young adults. Years later, these individuals are at risk of developing chronic complications, which can be severely debilitating and life-threatening. The process of ¿-cell destruction begins long before clinical disease onset and continues after development of hyperglycemia; at the time of diagnosis subjects retain a significant amount of ¿-cell function as measured by C-peptide responses to a mixed meal tolerance test (MMTT). However, ¿-cell function continues to deteriorate after diagnosis with the presumed eventuality of near complete loss of secretion over time. At present there is no treatment that fully interdicts islet autoimmunity. However, clinical trials have shown promising results, demonstrating that it is possible to interfere with islet autoimmunity and preserve C-peptide secretion for at least some time. Diabetes TrialNet is a consortium of international researchers, NIH-funded, investigating interventions for the prevention or delay of type 1 diabetes. As a TrialNet Clinical Center our long-term scientific goals are to contribute to the overarching goals of TrialNet: develop a better understanding of the natural history and molecular basis of T1D and implement clinical trials evaluating new strategies to prevent this disease. The objective of this proposal, a step in pursuit of these goals, is to remain an active participant in the research network, Type 1 diabetes TrialNet, and in the process of designing and implementing new prevention strategies. Each investigative step taken toward modulating the destructive process that leads to T1D moves us closer to the ultimate goal of prevention.

描述（由申请人提供）：1 型糖尿病（T1D）发生在遗传易感个体中，是由于胰腺 β 细胞进行性、选择性破坏而发生的，这主要是由自身反应性 T 细胞介导的。该疾病过程导致胰岛素分泌丧失和终生胰岛素依赖，不幸的是，最常见于儿童、青少年和年轻人。多年后，这些人面临着出现慢性并发症的风险，这些并发症可能会严重削弱并危及生命。细胞破坏的过程早在临床疾病发作之前就开始了，并在高血糖发生后继续进行；在诊断时，根据混合膳食耐受试验 (MMTT) 的 C 肽反应来测量，受试者保留了大量的 ¿ 细胞功能。然而，在诊断后，¿细胞功能继续恶化，推测随着时间的推移，分泌几乎完全丧失。目前还没有可以完全阻止胰岛自身免疫的治疗方法。然而，临床试验已经显示出有希望的结果，表明有可能干扰胰岛自身免疫并在至少一段时间内保留 C 肽分泌。 Diabetes TrialNet 是一个由国际研究人员组成的联盟，由 NIH 资助，致力于研究预防或延缓 1 型糖尿病的干预措施。作为 TrialNet 临床中心，我们的长期科学目标是为 TrialNet 的总体目标做出贡献：开发更好的 了解 T1D 的自然史和分子基础，并实施评估预防这种疾病的新策略的临床试验。该提案的目标是实现这些目标的一步，是继续积极参与 1 型糖尿病 TrialNet 研究网络，并参与设计和实施新的预防策略的过程。为调节导致 T1D 的破坏过程而采取的每一项研究步骤都使我们更接近预防的最终目标。