New Onset of Type 1 Diabetes - Age 12 to 35 Years Within 100 Days of Diagnosis - Thymoglobin

Treatment of Type 1 Diabetes Mellitus with Thymoglobulin

Study Name: Effect of Antithymocyte Globulin (ATG) on Preserving Beta-Cell Function in New Onset Type 1 Diabetes Mellitus

Study Sponsors: National Institute of Allergy and Infectious Disease (NIAID) and the Immune Tolerance Network (ITN)

Principal Investigator: Stephen E. Gitelman, M.D., Director of the Pediatric Diabetes Program at UCSF

Who can sign up for this study?

• New onset type 1 diabetes, within 100 days of diagnosis
• 12 to 35 years of age
• No active infections

What is this study about? The purpose of this research study is to see if 4 doses of the experimental medication Thymoglobulin® given to people with recent onset type 1 diabetes will help save the remaining beta cells in the pancreas from further destruction by the immune system. Thymoglobulin is a man-made antibody that has been approved by the Food and Drug Administration (FDA) to treat and prevent organ transplant rejection. Thymoglobulin acts on white blood cells, known as T cells, some of which are responsible for the destruction of insulin-producing beta cells in type 1 diabetes. This study will test whether the antibody can destroy the destructive T cells while permitting new ones to grow in their place that will not attack the remaining functional beta cells.

What will happen in this study? If you decide that you want to determine if you qualify for this study, you will be scheduled to come to the research clinic at UCSF for a screening visit where you can expect:

• We will ask you questions about your medical history
• You will have a physical examination
• We will obtain blood and urine samples from you for testing

Eligible volunteers that consent to participate will be randomly assigned to one of two groups. Group 1 will receive Thymoglobulin treatment and intensive diabetes management. Group 2 is a control group that will receive a placebo and intensive diabetes management. Neither you nor the study physicians or nurses will know to which group you have been assigned. Volunteers in both groups will be admitted to the UCSF hospital for 5-8 days to receive 4 doses of either the study medication or placebo.

All participants will have 12 visits over the first 6 months of the study, then additional visits every 3 months until the end of the study. Assessment of diabetes control, insulin use and blood collection will occur at every visit. In addition, at some visits, participants will undergo physical exams and mixed-meal tolerance testing, which involves drinking a special protein drink and having blood specimens taken over a 4-hour period.

All participants in the study will undergo “intensive diabetes management.” You will be expected to perform blood glucose testing at least 4 times a day and take insulin to keep your blood glucose within the target range as needed. You will be asked to discuss your blood glucose control with the study team every two weeks, and the team will work in conjunction with your regular diabetes provider to advise you on how to optimize your diabetes management plan so as to achieve the target goals.

How long will this study last? Volunteers are in the study for 2 years.

Reimbursement: Participants will receive compensation for participating in the study. In addition, parking at UCSF for the time of study visits will be paid for, and if you live more than 50 miles from UCSF, travel costs will be covered by the study. You will not be charged for any of the study treatments or procedures.

For more information: Please contact Kathleen Fraser at 415-353-9084 or email kfraser@diabetes.ucsf.edu.