Phase 2 Product Candidate for Type I DiabetesCaladrius is currently focused on developing a T-regulatory (Treg) cell-based therapy (CLBS03) for the treatment of type 1 diabetes mellitus (T1DM), using the patient’s own numerically and functionally enhanced Tregs. At the time of T1DM diagnosis, up to 20% of the patient’s insulin producing beta cells remain in the pancreas and, if protected from immune destruction, can be salvaged, and restored to function, thus regaining glycemic control and decreasing long-term risks of related complications1. Restoration of beta cells' viability and function can also potentially improve the patient’s quality of life by decreasing the patient’s dependence on exogenous insulin injections, and risk for major hypoglycemia and long term micro- and macrovascular complications.
The Sanford Project: T-Rex Study Phase 2 in adolescents with recent onset T1D
|Key Endpoints||Preservation of C-peptide level, insulin use, severe hypoglycemic episodes, hemoglobin A1c level|
|Powering||80% power to detect a 0.2 pmol/mL difference in AUC mean MMTT-stimulated C-peptide between active and placebo|
|Treatment||CLBS03: Low dose (2.5 million cells/kg) or high dose (20 million cells/kg); single dose|
|Control||Placebo infusion, single dose|
For more information, please visit the trial at www.clinicaltrials.gov.
The Sanford Project: T-Rex Study Executive Steering Committee
Steve Gitelman, MD
University of California, San Francisco
Peter Gottlieb, MD
University of Colorado
Kurt Griffin, MD, PhD
Kevan Herold, MD
Yale Diabetes Center
Antoinette Moran, MD
University of Minnesota
- Lernmark Å. (2013) Depleting t cells in newly diagnosed autoimmune (type 1) diabetes—are we getting anywhere? Diabetes, 62:3669-3670.
- Bluestone, J., et al. (2015) Type 1 diabetes immunotherapy using polyclonal regulatory T cells. Science Translational Medicine, 7 (315).
- Marek-Trzonkowsa, N., et al. (2014) Therapy of type 1 diabetes with CD4(+)CD25(high)CD127-regulatory T cells prolongs survival of pancreatic islets - results of one year follow-up. Clin Immunol., 7 (315): 315ra189. http://www.ncbi.nlm.nih.gov/pubmed/24704576