ATG/GCSF

TrialNet ATG-GCSF New Onset Study

Dear TrialNet Family,

As we announce the 2-year findings for the ATG-GCSF New Onset Study, I’d like to take a moment to give a big and well-deserved thank you to everyone who made this study possible:

  • The families who participated—for being the focus and essential element in everything we do at TrialNet. 
  • The TrialNet nurses and healthcare providers- for upholding the highest research standards and taking such good care of participating families.
  • Our fellow diabetes researchers around the globe- study by study, volunteer by volunteer, we are unraveling and conquering type 1 diabetes.
  • Everyone at the National Institutes of Health (NIH), National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), JDRF, and the American Diabetes Association- for your continued support. We also thank Congress for funding the Special Diabetes Program, an important program that provides critical funding for NIH T1D research.
  • The Helmsley Charitable Trust for providing funding over the last 10 years.
  • Sanofi for providing ATG and funding.
  • Amgen for providing GCSF.

Until then,

Michael Haller, M.D.
TrialNet ATG/GCSF Study Chair

University of Florida

Carla Greenbaum, M.D.
TrialNet Chair
Diabetes Clinical Research Program Director, Benaroya Research Institute at Virginia Mason

Eligibility 
Participants in Stage 3
Type 
Newly Diagnosed
Status 
Completed

About the Study

Study Details

This study was designed to build on prior findings of a pilot study suggesting thymoglobulin (ATG) combined with pegylated granulocyte colony stimulating factor (GCSF) preserved insulin production for more than 1 year after treatment in people who had type 1 diabetes for 4 months to 2 years.

ATG is approved by the Food and Drug Administration (FDA) for prevention and treatment of kidney transplant rejection. GCSF is an FDA-approved drug used to increase white blood cell counts in patients receiving chemotherapy.

TrialNet researchers wanted to know if ATG alone or in combination with GCSF could slow insulin loss when started earlier. To find out, we enrolled 89 people between ages 12 and 45 diagnosed with type 1 diabetes within the past 100 days.

Participants were divided into three groups: one group received ATG, one group received ATG combined with GCSF, and one group received a placebo (looks like the study drug but is inactive). Everyone participated in the study for 2 years.

Study Results

Key Findings

Two-year findings (Diabetes, March 2019) confirmed continuation of the positive effects reported 1 year after the start of treatment (Diabetes Care, September 2018):

  • Low-dose ATG preserved beta cell function and improved insulin production.
  • Hemoglobin A1c levels were significantly lower (indicating better long-term blood sugar control) in people treated with ATG alone and those treated with ATG combined with GCSF, as compared to placebo.

 “The fact that results are being maintained for 2 years is highly significant,” said Study Chair and TrialNet Investigator at University of Florida, Michael Haller, M.D.  “Any approach that allows people with type 1 diabetes to produce more of their own insulin and maintain better blood glucose control for this long demonstrates progress towards our ultimate goal of preventing and reversing this terrible disease.”

What's Next

Next Steps

"This project shows the power of collaboration in moving research forward," said Helmsley Type 1 Diabetes Program Director Gina Agiostratidou, Ph.D. "Thanks to long-term support from several groups, we're a step closer to using this knowledge to benefit people with type 1 diabetes."

  • We will continue to conduct mechanistic studies with the samples collected during the study to learn all we can.
  • TrialNet will consider future studies to learn whether ATG alone, or in combination with other drugs, will prevent or delay disease onset.
  • This approach is still experimental and requires additional research prior to being widely available to people with type 1 diabetes.

 

In addition, TrialNet is continually planning and launching new studies. And, we always encourage family members of people with T1D to get screened through the Pathway to Prevention study.

Participant Experience

For Study Participants

A big thank you to our amazing participants who made this research possible!

Your study team will be in touch shortly to go over your individual results.

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