TrialNet:
ATG/GCSF New Onset Study

About the ATG/GCSF New Onset Study

In a recent pilot study, two drugs— low-dose antithymocyte globulin (ATG) and granulocyte-colony stimulating factor (GCSF) — maintained insulin production in people who had type 1 diabetes for 4 months to 2 years. Those receiving this combination continued making insulin for up to a year after treatment. The untreated group experienced nearly a 40% decline.

Based on these promising results, TrialNet researchers will see if the same drug combination proves even more effective when started within 100 days of diagnosis.

Any healthy insulin-producing beta cells that remain at the time of your diagnosis are precious. This study will use a combination of two drugs—low-dose Thymoglobulin® (ATG) and Neulasta® (GCSF) — to try to stop the immune system’s attack on these remaining cells. This could extend your ability to keep producing insulin. Currently, there are no approved treatments able to do this.

About the Study Drugs

Thymoglobulin® is the brand name of anti-thymocyte globulin (ATG). It is already approved by the Food and Drug Administration (FDA) to prevent rejection in kidney transplant patients.  ATG does this by suppressing the immune system. It may also help control certain immune cells involved in type 1 diabetes. Given by intravenous (IV) infusion, this study’s dose of ATG will be 2.5 mg/kg of body weight.

Neulasta® is the brand name of pegylated granulocyte CSF (GCSF). It is already approved by the FDA to help reduce risk of infection in chemotherapy patients.  Study participants will get a standard dose (6mg) by injection (just under the skin), like an insulin injection.

Eligibility Criteria

You may be eligible to participate in the ATG/GCSF Study if you:

  • Are between the ages of 12 to 45, and
  • Have recently been diagnosed with type 1 diabetes and can start the study within 100 days of diagnosis, and
  • Test positive for at least one type 1 autoantibody (the screening test will reveal this), and
  • Are still making a minimal amount of insulin (based on results of a Mixed Meal Tolerance Test or MMTT), and
  • Are in good general health

Participating in the ATG/GCSF New Onset Study

We will compare the progression of diabetes in three groups of people:

  1. The first treatment group getting infusions of low-dose ATG and injections of GCSF
  2. The second treatment group getting infusions of low-dose ATG and injections of a placebo (looks like the study drug but has no active ingredients)
  3. The control group getting placebo infusions and injections

A computer will randomly choose which group you are in. No one will know who is in which group—not even the study staff—until the end of the study.

During the treatment phase, you will stay at a hospital for 2 to 3 days (2 nights). During that time, you will get two infusions (ATG or placebo) and one injection (GCSF or placebo). For the next 10 weeks, you will visit a TrialNet site every other week for an injection. After that, you will come in for clinic visits at 3 months and 6 months following treatment.

How to participate

If you have questions about the study or are interested in participating, please contact Gail at ggannon@medicine.bsd.uchicago.edu.