Phase 2 Product Candidate for Type I Diabetes

Caladrius 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.

A Phase 1 open-label uncontrolled dose-escalating study conducted at UCSF and Yale2 provided evidence for safety and tolerability of autologous expanded polyclonal Treg cell therapy (product representative of CLBS03) in 14 adults with established T1DM. Infused Tregs were detected in peripheral circulation for more than 12 months, indicating sustainability post administration. In addition, a Polish Phase 1 open-label controlled study of autologous expanded polyclonal Treg cell therapy in 22 children aged 5-18 with new onset T1DM3 similarly demonstrated safety and tolerability while providing evidence of potential bioactivity, as fasting C-peptide level (an indicator of beta cell function) was maintained, and increased rates of remission and insulin independence were observed in the Treg treatment group. Based on these findings, Caladrius received FDA approval to conduct a randomized, double-blind, placebo-controlled Phase 2 study to further investigate the CLBS03 therapy in adolescents with recent onset T1DM.

The Sanford Project: T-Rex Study Phase 2 in adolescents with recent onset T1D

  • Randomized, double-blind, placebo-controlled trial
  • Adolescent patients ages 12 to 17 with recent onset of T1DM
Key EndpointsPreservation of C-peptide level, insulin use, severe hypoglycemic episodes, hemoglobin A1c level
Powering80% power to detect a 0.2 pmol/mL difference in AUC mean MMTT-stimulated C-peptide between active and placebo
Study Size
  • 111 patients planned
  • Approximately 20 sites expected, including US and, possibly, Canada
  • Supported by strategic collaboration with Sanford Research – The Sanford Project
TreatmentCLBS03: Low dose (2.5 million cells/kg) or high dose (20 million cells/kg);  single dose
ControlPlacebo infusion, single dose

For more information, please visit the trial at

The Sanford Project: T-Rex Study Executive Steering Committee

  1. Lernmark Å. (2013) Depleting t cells in newly diagnosed autoimmune (type 1) diabetes—are we getting anywhere? Diabetes, 62:3669-3670.
  2. Bluestone, J., et al. (2015) Type 1 diabetes immunotherapy using polyclonal regulatory T cells. Science Translational Medicine, 7 (315).
  3. 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.