March 29, 2016 8:21am
The initial safety analysis on the first 18 subjects will be provided by early 2017.
CLBS treated the first subject in The Sanford Project: T-Rex Study. The T-Rex Study is a P2 trial of Caladrius’ product candidate CLBS03 (autologous expanded regulatory T cells, or Tregs) for the treatment of recent-onset type 1 diabetes (T1D) in adolescents.
The prospective, randomized, placebo-controlled, double-blind Phase 2 clinical trial to evaluate the safety and efficacy of CLBS03 will enroll 111 subjects age 12 to 17 in two cohorts (18 subjects followed by 93 subjects) and is being conducted in collaboration with Sanford Research, a subsidiary of Sanford Health. Subjects will be randomized into one of three groups and will receive either a high dose of CLBS03, a low dose of CLBS03 or placebo.
Investigational product will be administered as a single infusion and the subjects will be evaluated over the course of 2 years. The key endpoints for the trial are the standard medical and regulatory endpoints for a type 1 diabetes trial and include preservation of C-peptide, an accepted measure for pancreatic beta cell function; insulin use; severe hypoglycemic episodes; and hemoglobin A1c level in comparison to placebo.
The Bottom Line: The scientific basis for this program stems from the use of Tregs to treat diseases caused by imbalances in an individual's immune system, such as T1D. An initial safety analysis of the data and early analysis of immunological biomarkers will occur after the first cohort of 18 subjects has completed the three-month post-treatment visit. Pending review of the data from the first cohort and recommendation of the independent Data Safety Monitoring Board, a second cohort of 93 subjects will be enrolled.
This “novel - ?” approach seeks to restore immune balance by enhancing Treg cell number and function. Tregs are a natural part of the human immune system and regulate the activity of T effector cells, which are responsible for protecting the body from viruses and other foreign antigens.
When Tregs function … “properly”, only harmful foreign materials are attacked by T effector cells. In autoimmune diseases, it is thought that deficient Treg activity permits the T effector cells to attack the body's own beneficial cells, and in the case of T1D, insulin-producing pancreatic beta cells.
CLBS closed at $0.75 <which was down -$0.04>. The spin will be positive but, balancing the number of companies “working” on the diabetes paradigm – just another P2 and “spend” with 2017 results of …?
CLBS can’t afford to wait as share pricing has been more than dismal.
