Previous clinical studies have shown the benefit of teplizumab in prolonging insulin production in people recently diagnosed with T1D.
This is the first study to test teplizumab’s ability to delay or prevent disease progression in those at high risk for developing T1D.
All study participants had two or more T1D autoantibodies and abnormal blood sugar levels (Stage 2 T1D) prior to enrollment, as identified by TrialNet Pathway to Prevention risk screening. Individuals in Stage 2 have a lifetime risk of clinical diagnosis (Stage 3 T1D) nearing 100%.
Researchers enrolled 76 people between the ages of 8 and 49; 55 were under age 18. All participants had at least one relative with type 1 diabetes.
Participants were randomly assigned to either the treatment group, which received a 14-day course of teplizumab, or the control group, which received a placebo. All participants regularly underwent oral glucose tolerance tests either until the study was completed or until they developed clinical type 1 diabetes.
- This is the first study to show any drug can delay type 1 diabetes diagnosis a median of 2 years in adults and children at high risk.
- 72% of people in the control group developed clinical diabetes, compared to only 43% of the teplizumab group.
- The median time for people in the control group to develop clinical diabetes (stage 3) was just over 24 months, while those in the treatment group had an average of 48 months before progressing to clinical diabetes (stage 3).
- Researchers observed, as with other trials involving teplizumab, short-term side effects of rash and low white blood cell counts.
- Subgroups of individuals may have had a better response to therapy than others.
- In addition to being able to accurately predict who is going to develop T1D, TrialNet has now found a way to delay it. This is an incredible advancement that gets us one step closer to our ultimate goal: a future without T1D.
- We know immunotherapy can slow the progression T1D, and we now hope to conduct additional studies to look for ways to extend the benefits of teplizumab, while continuing to test other immune therapies.
- Samples collected during the Teplizumab Prevention Study are being studied to help researchers understand why some group of individuals had a better response to therapy than others.
- In addition, TrialNet is conducting extended follow up with participants electing to continue being tested every 6 months.
- The latest findings, published in Science Translational Medicine on March 3, 2021, show ongoing delay in diabetes in the teplizumab group. The median time to diabetes diagnosis in the teplizumab group was approximately 60 months (5 years), vs. approximately 27 months (2.3 years) in the placebo group.
- In addition to having even more pronounced disease delay, those treated with teplizumab showed improved rates of insulin production. Participants receiving a placebo continued to show a decline in insulin production consistent with disease advancement.
- TrialNet will continue testing participants every 6 months, so we can continue to track and report changes. Our hope is that disease delay will extend even further over time.
In addition, TrialNet is continually planning and launching new studies. As always, we encourage family members of people with T1D to get screened through the Pathway to Prevention Study.
A big thank you to our amazing participants who made this research possible! Teplizumab participant resources are available here.