TrialNet: University of Florida Clinical Center and Network

描述（由申请人提供）：佛罗里达大学打算继续作为临床中心，作为国家组成的糖尿病试验网络（1型糖尿病TrialNet）的一部分，以最终研究旨在预防或延缓1型糖尿病（T1 D）发展的疗法。佛罗里达大学临床中心参与了9项已批准的NIH TrialNet方案，包括自然史研究、吗替麦考酚酯（MMF）/ Dacluzimab（DZB）试验、抗CD 20试验、CTLA-4 lg试验、口服胰岛素研究、比较性混合膳食耐受（MMTT）-胰高血糖素刺激（GST）C肽研究、T细胞测定验证研究、营养干预预防糖尿病（NIP）研究和T1 D遗传学联盟。该中心在受试者招募和TrialNet（和其他1型研究）开展方面一直是表现最强的机构之一，并将继续努力监督其成功的附属中心网络和参与TrialNet研究招募和随访的参与医生，并进一步扩大这些努力。我们打算继续在新发糖尿病患者中进行研究，旨在保护β细胞功能和/或减少β细胞破坏，如果安全性和有效性得到证明，则使用这些药物进行旨在预防疾病的研究。基于（一）令人兴奋的初步在NOD小鼠中使用低剂量抗胸腺细胞球蛋白（ATG）和粒细胞集落刺激因子（GCSF）组合的（疗效、机制和安全性）数据，（ii）在其他人类自身免疫性疾病和移植中的令人鼓舞的数据，（iii）在新发患者中单独使用每种药物的初步研究，我们建议进行一项旨在确定安全性的研究，该组合作为新诊断的T1 D个体中减缓疾病进展的干预的功效和机制。

DESCRIPTION (provided by applicant): The University of Florida intends to continue as a Clinical Center as part of the nationally constituted diabetes trial network (Type 1 Diabetes TrialNet), in order to ultimately study therapies aimed at preventing or delaying the development of type 1 diabetes (T1D). The University of Florida Clinical Center has participated in nine approved NIH TrialNet protocols including the Natural History Study, the Mycophenolate Mofetil (MMF) / Dacluzimab (DZB) Trial, the Anti-CD20 Trial, the CTLA-4 lg Trial, the Oral Insulin Study, the comparative Mixed Meal Tolerance (MMTT) - Glucagon Stimulation (GST) C-peptide Study, the T-Cell Assay Validation Study, the Nutrition Intervention to Prevent Diabetes (NIP) Study, and the T1D Genetics Consortium. The Center has consistently been one of the strongest performers both in the recruitment of subjects and the conduct of TrialNet (and other Type 1 studies) and will work to continue oversight of its successful network of Affiliate Centers and participating physicians involved in TrialNet study recruitment and follow-up and to further expand these efforts. We intend to continue to conduct studies in patients with recent-onset diabetes aimed at preserving beta cell function and/or decreasing beta cell destruction, and, if safety and efficacy is demonstrated, implement studies using these agents aimed at the prevention of the disease. Based on (i) exciting preliminary (efficacy, mechanistic and safety) data using a combination of low-dose Anti-Thymocyte Globulin (ATG) and Granulocyte Colony Stimulating Factor (GCSF) in NOD mice, (ii) encouraging data in other human autoimmune disorders and transplantation (iii) pilot studies using each agent separately in new-onset patients, we propose to conduct a study aimed at establishing the safety, efficacy and mechanism of this combination as an intervention to slow disease progression in individuals with newly-diagnosed T1D.