PHASE II MULTIPLE-DOSE TREATMENT OF TYPE 1 DIABETES MELLITUS WITH ALA-ALA

这个子项目是利用由NIH/NCRR资助的中心拨款提供的资源的许多研究子项目之一。子项目和调查员(PI)可能从另一个NIH来源获得了主要资金，因此可能会出现在其他CRISE条目中。列出的机构是针对中心的，而不一定是针对调查员的机构。这是一项多中心、双臂、随机、开放标签的II期试验，涉及使用免疫抑制药物hOKT3yl(ALA-ALA)治疗新发的1型糖尿病(T1 DM)患者，每隔一年进行0或2个周期的治疗。据推测，hOKT3将在确诊后的头两年阻止胰岛β细胞胰岛素产生的损失，并改善血糖控制。研究表明，血糖控制越好，远期TIDM发病率越低。参与者将被随机分为药物治疗组(A组)或对照组(B组)。在每个周期内都有剂量递增，但周期之间没有变化。随机选择2名药物受试者和1名对照受试者，并按研究地点分层。目前的方案是基于哥伦比亚大学进行的I/II期和PK/安全性试验，这些试验建议了2002年5月发表在《新英格兰医学杂志》上的临床疗效，并证明了该药物的安全性。

This subproject is one of many research subprojects utilizing the resources provided by a Center grant funded by NIH/NCRR. The subproject and investigator (PI) may have received primary funding from another NIH source, and thus could be represented in other CRISP entries. The institution listed is for the Center, which is not necessarily the institution for the investigator. This is a multi-centered, 2 arm, randomized, open label, Phase II trial involving 0 or 2 cycles of treatment one year apart using the immune suppressive drug hOKT3yl (Ala-Ala) in patients with new onset type 1 Diabetes Mellitus (T1DM). It is hypothesized that hOKT3 will arrest the loss of insulin production of the pancreatic beta cells during the first 2 years from diagnosis and improve glycemic control. Greater glycemic control has been shown to result in less long-term TIDM morbidity. Participants will be randomized to drug treatment (Group A) or control (Group B). There is dose escalation within each cycle, but no change between cycles. The randomization will be 2 drug subjects to 1 control subject and will be stratified by study site. The current protocol is based on Phase I/II and PK/Safety trials conducted at Columbia University that suggested clinical efficacy as published in the NEJM in May 2002, and demonstrated safety of the drug.