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

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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