DPT-1 TrialNet: Clinical Centers

描述(由申请人提供) 美国有100多万人患有1型糖尿病 世界各地还有更多的人。1型糖尿病是由遗传易感性引起的 作为免疫介导的胰腺破坏的结果的个体 胰岛分泌胰岛素的β细胞。糖尿病临床症状的出现 代表胰岛β细胞功能慢性进行性下降的终点 当大多数β细胞丢失时，就会发生这种情况。一级学位 1型糖尿病患者的亲属患糖尿病的可能性是普通人的10倍 罹患该病的人数比一般人群要多。这种疾病可能会开始 在任何年龄，典型的是从童年或年轻人开始的。这个 目前治疗1型糖尿病的方法包括早期使用胰岛素， 严格的饮食干预和频繁的血糖监测，当 实施得当，可以延缓或预防可怕的长期并发症 糖尿病(即失明、肾功能衰竭和截肢)。然而，适当的 糖尿病的治疗是相当困难的，昂贵的，而且对 糖尿病患者？S的生活方式。糖尿病也是影响健康的一个主要因素。 护理费用。预防1型糖尿病是可能的吗？到目前为止，有几个免疫 干预措施已经在遗传易感人群中进行了试验，但没有 成功。其他试验也试图在早期干预 1型糖尿病，以保护β细胞功能。这些免疫 干预也同样失败了。因此，识别出 要么预防疾病，要么减缓疾病的发展，都会带来重大的健康 节省护理成本并减少与糖尿病相关的并发症 个人在没有患上这种疾病方面的巨额储蓄。我们的长期合作 目标是通过使用阿司匹林预防1型糖尿病的发展 创新的以免疫为基础的疗法旨在防止 遗传易感个体的疾病。这样做的目的是 为了实现这一目标并响应《框架协议》，申请已经完成。 DPT-1协议和TrialNet的发展，一个增强的网络，以 开发和测试创新的干预措施，以防止或减缓 1型糖尿病。一种这样的创新方法可以减缓1型糖尿病的进展 糖尿病是我们建议的试验方案，“噻唑烷二酮保留C-肽” 这项拟议的工作具有创新性，因为它使用了一种药物 它已经被证明是安全的，并且已经被广泛用于治疗 它不仅是2型糖尿病的致病因子，也是免疫调节剂。我们的中心有 经验、人员和接触适当患者的途径 成为TrialNet的成功成员，并能够成功完成我们的 建议的试点协议或TrialNet可能提出的其他协议。

DESCRIPTION (provided by applicant) Type 1 diabetes affects more than one million individuals in the United States and many more worldwide. Type 1 diabetes arises in genetically predisposed individuals as a consequence of immune-mediated destruction of the pancreatic islet insulin-secreting beta-cells. The onset of clinical symptoms of diabetes represents the endpoint of a chronic progressive decline in beta-cell function and occurs when the majority of beta-cells have been lost. First-degree relatives of individuals with Type 1 diabetes are ten-fold more likely to develop the disease than the general population. The disease, which can begin at any age, characteristically begins in childhood or in young adults. The present treatment for Type 1 diabetes, which includes the early use of insulin, rigid dietary intervention, and frequent blood glucose monitoring, when implemented properly, can delay or prevent the horrible long-term complications of diabetes (i.e. blindness, renal failure, and amputation). However, proper diabetes treatment is quite difficult to do, expensive, and very invasive to the diabetic patient?s lifestyle. Diabetes is also a major factor in health care costs. Is it possible to prevent Type 1 diabetes? To date, several immune interventions have been tried in genetically susceptible individuals without success. Other trials have been attempted to intervene early in the course of Type 1 diabetes, in order to preserve beta-cell function. These immune interventions have likewise failed. Thus, the identification of agents that either prevent the disease or slow its progression would result in major health care cost savings and reduce complications related to diabetes in addition to the huge individual savings in terms of not having the disease. Our long-term goal is to prevent the development of Type 1 diabetes through the use of innovative immune-based therapies designed to prevent the development of the disease in genetically predisposed individuals. The objectives of this application, in pursuit of that goal and in response to the RFA, is completion of the DPT-1 protocol and the development of TrialNet, an enhanced network to develop and test innovative interventions to prevent or slow the progression of Type 1 diabetes. One such innovative approach to slow the progression of Type 1 diabetes is our proposed pilot protocol, "Thiazolidinediones Preserve C-Peptide in Type 1 Diabetes." The proposed work is innovative because it utilizes a drug that has a proven safety profile and is already widely used for the treatment of Type 2 diabetes but also is an immune modulator. Our Center has the experience, the personnel, and access to the appropriate patients to be a successful member of TrialNet and to be able to successfully complete our proposed pilot protocol or others that TrialNet may bring forward.