LONG-TERM EFFECTS OF NBI 6024 IN TYPE 1 DIABETES SUBJECTS FROM NBI602-0003 TRIAL

该子项目是利用NIH/NCRR资助的中心赠款提供的资源的许多研究子项目之一。子项目和研究者（PI）可能从另一个NIH来源获得了主要资金，因此可以在其他CRISP条目中表示。所列机构为中心机构，不一定为研究者机构。1型糖尿病或胰岛素依赖型糖尿病（IDDM）影响全世界1900万人。1型糖尿病似乎是自身免疫攻击的结果，其中身体自身的免疫系统破坏胰腺的胰岛素产生细胞。免疫系统中的一种特殊类型的细胞，T细胞，似乎在导致糖尿病的自我毁灭中很重要。过去的研究表明，一种称为“改变的肽配体”（APL）的药物可能会阻止这些T细胞伤害胰腺。我们正在测试APL药物NBI-6024的安全性和有效性，以阻止新诊断的1型糖尿病患者的T细胞完全破坏产生胰岛素的胰腺细胞。 本研究旨在评估NBI-6024在成人和青少年1型糖尿病患者中的长期效果。这些患者之前接受了多次注射研究药物或“安慰剂”，一种不含药物的注射剂（相当于糖丸），然后监测一年。这些患者被随机给予药物或安慰剂，临床研究人员不知道这些患者属于哪一组。在NBI-6024给药后一年，进行血液检查以测量胰岛素产生，T细胞活性和一般健康状况。研究医生还进行了全面的体格检查，以进一步评估一般健康状况。这项后续研究将继续这些血液测试和身体检查两年，以确定药物对胰岛素产生，T细胞活性和健康的长期影响。这种长期分析是重要的，因为（1）这种药物从未用于新发患者，（2）更多的数据将改善结果分析，（3）一旦“蜜月期”结束，药物治疗患者和安慰剂治疗患者之间的胰岛素产生差异可能更加显著（蜜月期是初次诊断后的一段时间，通常不到一年，在此期间，许多患者仍然会自己制造一些胰岛素）。本研究中的所有患者都将参加既往试验（NBI-6024-0003），其中他们接受了APL药物NBI-6024或安慰剂。这项研究的结果将有助于确定NBI-6024的长期安全性，以便进一步测试这种药物作为一种方法，当给予新诊断的患者时，可以降低1型糖尿病的严重程度，或者预防高危患者的疾病。

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. Type 1 diabetes or insulin-dependent diabetes mellitus (IDDM) affects 19 million people worldwide. Type 1 diabetes appears to be a result of an autoimmune attack in which the body's own immune system destroys the insulin producing cells of the pancreas. One specific type of cell in the immune system, the T cell, appears to be important in the self-destruction that leads to diabetes. Past research has shown that a type of drug called an "altered peptide ligand" (APL) may stop these T cells from harming the pancreas. We are testing the safety and effectiveness of an APL drug, NBI-6024, to stop a newly diagnosed type 1 diabetes patients' T cells from completely destroying the insulin-producing pancreas cells. This study is designed to assess the long-term effect of NBI-6024 in adult and adolescent type 1 diabetes patients. These patients were previously given multiple injections of either the study drug or a "placebo," an injection without a drug (equivalent to a sugar-pill), and then monitored for a year. The patients were given the drug or placebo on a random basis, and the clinical investigators did not know to which group the patients belonged. The year following NBI-6024 dosing, blood tests were done to measure insulin production, T cell activity and general health. A study doctor also did a comprehensive physical exam to further assess general health. This follow-up study will continue these blood tests and physical exams for two more years in order to determine the drug's long-term effects on insulin production, T cell activity and health. This long-term analysis is important because (1) this drug has never been given to new-onset patients, (2) more data will improve the resulting analysis and (3) differences in insulin production between drug treated patients and placebo may be more significant once the "honeymoon" phase is over (honeymoon phase is a time after initial diagnosis, usually less than one year, during which many patients still make some of their own insulin). All patients in this study will have participated in the prior trial (NBI-6024-0003) in which they received the APL drug NBI-6024 or a placebo. Results from this study will help determine the long-term safety of NBI-6024 so that this drug can be further tested as a method to reduce the severity of type 1 diabetes when given to newly diagnosed patients or prevent the disease in high-risk patien