Study Details Completed
Findings from the Abatacept Prevention Study were published online in Diabetes Care.
The goal of the Abatacept Prevention Study was to see if abatacept could delay or prevent type 1 diabetes (T1D) progression from stage 1 (two or more diabetes-related autoantibodies, normal blood sugar) to stage 2 (abnormal blood sugar) or to stage 3 (clinical diagnosis).
Abatacept (CTLA-4 lg) changes the way immune cells communicate. Marketed as Orencia®, abatacept is currently approved by the FDA for use in children as young as 6 for two types of juvenile arthritis and is also being studied for treatment of other autoimmune diseases.
The Abatacept Prevention Study opened in 2013 and enrolled 212 people between the ages of 6 and 45. All participants had at least one relative with T1D, two or more diabetes-related autoantibodies, and normal glucose tolerance (stage 1 T1D).
In the first year, participants received 14 intravenous (IV) infusions—three in the first month, then monthly for 11 months. Half of the participants got the study drug; the other half got a placebo infusion (looks like and is administered in the same way as the study drug but is inactive). A computer randomly selected who got the study drug, and no one knew who was in which group until the end of the study.
After the first year, participants visited a TrialNet site twice a year for monitoring until the study ended.
- While abatacept impacted immune response and preserved insulin production during the one-year treatment period, it did not meet the study goal of delaying progression to abnormal glucose tolerance (stage 2) or clinical diagnosis (stage 3).
- The group treated with abatacept maintained beta cell function better than the placebo group at 12 months. However, 12 months after treatment ended, beta cell function was the same for both groups. The early effect was not sustained.
- Overall results confirm abatacept’s potential as a disease modifying therapy in T1D and suggest direction for additional research.
- In a previous TrialNet study in people newly diagnosed with T1D, participants were treated with abatacept for 2 years. At the end of 2 years, those treated with abatacept had 59% better insulin production and a 9.6-month average delay in progression of insulin loss compared to those who received placebo. That difference extended out to 3 years.
- Studying abatacept in both stage 1 and stage 3 (clinical diagnosis) has helped us learn more about how the drug works and its impact on disease progression.
- These findings open the door to additional research asking: Would longer treatment make a difference? Would starting abatacept in stage 2 be more effective? Could combining abatacept with another therapy prolong its impact?
- TrialNet will continue to evaluate the results of mechanistic studies (evaluating test samples collected during and after the Abatacept Prevention Study) to determine additional and/or long-term effects in subgroups of participants.
- TrialNet is currently planning a new study to test abatacept in combination with rituximab, another drug that targets the immune system, in people newly diagnosed with T1D (Stage 3). Expected to open for enrollment later this year, this study will be the first to test these two drugs consecutively (one after the other). Separate TrialNet studies showed each drug individually helped preserve insulin production in people newly diagnosed. By combining the two therapies, researchers expect more individuals to maintain insulin production during and possibly after treatment.
- TrialNet is continually planning and launching new studies. The first step for those interested in participating in TrialNet research is to get screened through the Pathway to Prevention Study. TrialNet risk screening is free, quick and convenient. You can order an in-home test kit (that uses a fingerstick) or get a blood draw at a local lab or one of TrialNet’s many locations. If screening detects the early stages of T1D, you may be eligible for monitoring and clinical studies to try to change the course of the disease. To learn more, visit trialnet.org/participate.
For Study Participants
A big thank you to our amazing participants who made this research possible!
- If you have not been contacted by your study team to review your individual results, please contact your study team or email us at firstname.lastname@example.org.
- If you have questions about your study results or results from the overall study, please contact your study team or email@example.com.
- If you have not developed T1D, we invite you to continue to be monitored in the Pathway to Prevention Study.
- If you developed T1D and haven’t already joined the LIFT Study (Long-Term Investigative Follow-Up in TrialNet), we invite you to do so. This study gives participants personal ongoing monitoring by experts at the forefront of T1D research.
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