May 20, 2024

C-peptide, a biomarker that indicates insulin production, is a validated surrogate endpoint in type 1 diabetes clinical trials

New York, May 20, 2024 — A paper published in the June edition of the Journal Diabetes demonstrates that C-peptide, a biomarker that indicates the production of insulin, is a validated surrogate endpoint, or predictor of clinical benefit, for clinical trials of disease-modifying therapies for type 1 diabetes (T1D) in the new-onset stage. The paper, written by Breakthrough T1D Vice President of Research Esther Latres, Ph.D., and co-authored by Breakthrough T1D staff and other leaders in the field, reviewed published evidence and clearly demonstrated the association between the preservation of C-peptide and clinical benefits. If accepted by the regulators, the use of C-peptide as a validated endpoint has the potential to transform T1D clinical trials and accelerate the development of disease-modifying therapies for T1D.

“Through a comprehensive and extensive review of studies showing clinical benefits and endpoints traditionally used in type 1 diabetes, there is support for the acceptance of stimulated C-peptide as a validated surrogate endpoint for clinical trials in disease-modifying therapies in people with newly diagnosed type 1 diabetes,” said Esther Latres, Breakthrough T1D Vice President of Research. “There remains an urgent need for new therapies that can change the course of T1D, particularly in its early stages. Such acceptance would be a breakthrough that could rapidly advance the development of life-changing therapies and lead the way to cures for type 1 diabetes.”

Decades of research have resulted in tremendous progress and therapeutic options to improve T1D, yet there is still a significant unmet need to improve outcomes for those who live with the condition. The authors highlight the role and importance of disease-modifying therapies in addressing this need. However, the currently accepted efficacy endpoints, HbA1c, low blood sugar events, and complications, are limiting for disease-modifying therapy clinical trials, particularly in new-onset T1D, where low blood sugar events are uncommon and complications take years to develop.

“Disease-modifying therapies are a key element of Breakthrough T1D’s research strategy to develop cures for type 1 diabetes,” said Sanjoy Dutta, Ph.D., co-author and Breakthrough T1D Chief Scientific Officer. “The research in this area is promising, with the approval of the first disease-modifying therapy for people in Stage 2 T1D, teplizumab, in 2022. Importantly, several drugs approved for other autoimmune diseases have shown they can protect and preserve C-peptide at the time of diagnosis, and it’s Breakthrough T1D’s role to help accelerate the development pathway for disease-modifying therapies in T1D.”

About Breakthrough T1D
JDRF’s mission is to accelerate life-changing breakthroughs to cure, prevent, and treat T1D and its complications. To accomplish this, Breakthrough T1D has invested more than $2.5 billion in research funding since our inception. We are an organization built on a grassroots model of people connecting in their local communities, collaborating regionally and globally for efficiency and broader fundraising impact, and uniting on a global stage to pool resources, passion, and energy. We collaborate with academic institutions, policymakers, and corporate and industry partners to develop and deliver a pipeline of innovative therapies to people living with T1D. Our staff and volunteers throughout the United States and our five international affiliates are dedicated to advocacy, community engagement, and our vision of a world without T1D. For more information, please visit or follow us on Twitter (@JDRF), Facebook (@myjdrf), and Instagram (@jdrfhq).

About Type 1 Diabetes (T1D)
T1D is an autoimmune condition that causes the pancreas to make very little insulin or none at all. This leads to dependence on insulin therapy and the risk of short or long-term complications, which can include highs and lows in blood sugar; damage to the kidneys, eyes, nerves, and heart; and even death if left untreated. Globally, it impacts nearly 9 million people. Many believe T1D is only diagnosed in childhood and adolescence, but diagnosis in adulthood is common and accounts for nearly 50% of all T1D diagnoses. The onset of T1D has nothing to do with diet or lifestyle. While its causes are not yet entirely understood, scientists believe that both genetic factors and environmental triggers are involved. There is currently no cure for T1D.