UCSF TrialNet: A Phase II Trial of hokT3gamma1 (Ala-Ala)

描述(由申请人提供) 该应用程序的目标是在以下位置建立TrialNet的临床中心 加州大学旧金山分校(UCSF)糖尿病中心， 最终的长期目标是开发预防类风湿关节炎发病的治疗方法 糖尿病(T1 DM)1例。加州大学旧金山分校作为一项研究具有独特的地位 这项多中心试验的地点，考虑到强大的既定糖尿病计划， 招募科目的人口基数大，并有既定的记录 对T1 DM进行临床研究和自身免疫干预。医院的临床医生 加州大学旧金山分校糖尿病中心现在为大约3400名患者提供护理，有100名患者 每年都有新的1型糖尿病患者。此外，该中心还拥有 在临床和基础科学方面都有丰富经验的研究人员 研究，并与NIH资助的儿科和普通临床研究中心合作。 调查人员处于有利地位，可以从加州大学旧金山分校内部招募受试者。 系统，并广泛地从整个北方现有的转介网络 加利福尼亚。人口的多样性表明，研究对象将是 从不同的种族招募。这个应用程序将建立在加州大学旧金山分校-S 过去在糖尿病预防试验-1(DPT-1)中的附属角色。 具体而言，研究目标是：具体目标1：执行和继续 DPT-1协议。这包括创建一个基础设施 加强对已经进入DPT-1的主题的后续行动，以及 招聘以筛选和上演新科目，随后将招收 那些处于中等风险的人进入口服胰岛素研究手臂。具体目标2： 参与通过TrialNet开发的其他试验。具体目标3： 实施一项涉及治疗高危人群的新方案 伴有非FCR结合的抗CD3单抗的1型糖尿病 (hOKT3g1(ALA-ALA)。该药正在进行的治疗新发T1 DM的I/II期试验 已经证明服用这种药物是安全的，并表明了疗效 随着时间的推移保持胰岛素的分泌能力。我们这样做的理由是 应用是，如果在患者身上看到类似的效果， 检测到β细胞功能障碍的最早迹象，然后进展到 糖尿病可能是可以预防的。

DESCRIPTION (provided by applicant) The goal of this application is to establish a Clinical Center of TrialNet at the University of California at San Francisco (UCSF) Diabetes Center, with the ultimate long-term objective to develop therapies to prevent the onset of Type 1 diabetes mellitus (T1DM). UCSF is uniquely positioned to serve as a study site for this multicenter trial, given a strong established diabetes program, a large population base for subject recruitment, and an established track record with clinical research and autoimmune interventions for T1DM. Clinicians at the UCSF Diabetes Center now provide care for approximately 3400 patients, with 100 new patients with Type 1 diabetes each year. In addition, the center has investigators with extensive experience in both clinical and basic science research, and with NIH-funded Pediatric and General Clinical Research Centers. The investigators are well positioned to recruit subjects from within the UCSF system, and widely from a preexisting referral net throughout Northern California. The diversity of the population suggests that subjects will be recruited from a variety of ethnicities. This application will build on UCSF?s past role as an Affiliate in the Diabetes Prevention Trial-1 (DPT-1). Specifically, the study goals are: Specific Aim 1: To implement and continue the DPT-1 protocols. This includes creation of an infrastructure that will enhance follow-up of subjects already entered into the DPT-1, as well as recruitment to screen and stage new subjects, with subsequent enrollment of those at intermediate risk into the oral insulin study arm. Specific Aim 2: To participate in other trials developed through TrialNet. Specific Aim 3: To implement a novel protocol involving treatment of individuals at high risk for Type 1 diabetes with a non-FcR binding anti-CD3 monoclonal antibody (mAb) (hOKT3g1(Ala-Ala)). An ongoing Phase I/II trial of this agent in new onset T1DM has shown that administration of the drug is safe, and has suggested efficacy in preserving insulin secretory capacity over time. Our rationale for this application is that if similar effects are seen in patients in whom the earliest signs of beta cell dysfunction are detected, and then progression to diabetes might be prevented.