SIOUX FALLS, S.D. - A clinical trial studying type 1 diabetes has reached full enrollment.
The Sanford Project: T-Rex Study, a Phase 2 clinical trial conducted collaboratively by Sanford Health and Caladrius Biosciences, Inc., (Caladrius)(Nasdaq: CLBS), has completed enrollment of 110 children with type 1 diabetes. The study started with two sites at Sanford Health in Sioux Falls, South Dakota, and Fargo, North Dakota, and expanded to 13 additional sites across the United States.
The project is studying the potential of CLBS03, Caladrius' cell therapy consisting of each patient's own regulatory T cells, or Tregs, to help the body fight type 1 diabetes.
Subjects will be followed for two years, with the primary endpoint of persistence of insulin production at one year after treatment. A planned, interim analysis of the first half of the participants at six months after treatment is expected by the end of the first quarter.
"I am thrilled to have reached this important milestone," said Kurt Griffin, M.D., Ph.D., director of clinical trials for The Sanford Project. "It has taken a tremendous amount of work from a large team to get this far. We still have another year of follow-up before we can really see how this treatment may be working."
Griffin and Fargo-based pediatric endocrinologist Luis Casas, M.D., are the study's principal investigators at Sanford Health.
Individuals with type 1 diabetes experience a loss of insulin-producing beta cells as their immune system targets these cells inappropriately. Treg cells usually keep the immune system under control, but they are lacking in number and activity in people with type 1 diabetes. The Sanford Project: T-Rex Study is exploring whether expanding the body's supply of Treg cells can rebalance the immune system, stop destruction of beta cells and preserve insulin production. Participants were randomized to either of two doses in the treatment arms or to placebo. For those in the treatment groups, the participant's own Treg cells were extracted from the body, purified, expanded in culture, and returned to blood circulation. The cell identification and expansion process is patented technology licensed by Caladrius, a cell-therapy development company.
The therapy being used in this trial, CLBS03, has received fast track designation from the U.S. Food and Drug Administration (FDA), a first for any type 1 diabetes intervention. That designation is reserved for drugs or biologics that address a serious health condition, like type 1 diabetes, where there is an unmet medical need. This status allows for more frequent communication with the FDA and faster feedback about the therapy during the approval process. It also allows researchers to submit data and reports on a rolling basis. CLBS03 also has been granted European Medicines Agency's Advanced Therapeutic Medicinal Product classification and FDA Orphan Drug designation as a potential new treatment for recent-onset T1D.
The Sanford Project is a cornerstone initiative at Sanford Health focusing on finding a cure for type 1 diabetes. The initiative was launched as part of a $400 million gift from philanthropist Denny Sanford in 2007.
About Caladrius Biosciences
Caladrius Biosciences, Inc. is a clinical stage biopharmaceutical company with multiple technology platforms targeting autoimmune and select cardiology indications. The Company is investigating its lead product candidate, CLBS03, an ex vivo expanded polyclonal T regulatory cell therapy for the treatment of recent-onset type 1 diabetes, in an ongoing Phase 2 trial. CLBS14, CD34+ cell therapy intended as a treatment for coronary microvascular dysfunction, is Caladrius' proprietary and patent protected formulation of CD34 cells designed specifically to enhance the potency of the CD34 cells for repair and regeneration of cardiovascular tissue. Its companion product, CLBS12, is specifically formulated for intramuscular administration for the treatment of lower extremity ischemia. A phase 2 study of CLBS12 as a treatment for critical limb ischemia has initiated in Japan, a successful outcome of which will qualify the program for consideration of early conditional approval based on discussions with the Japanese regulatory authorities as provided for under Japan's progressive regenerative medicine regulations. For more information about Caladrius please visit http://www.
About Sanford Health
Sanford Health is one of the largest health care systems in the nation, with 44 hospitals and nearly 300 clinics in nine states and four countries. Headquartered in Sioux Falls, South Dakota, and serving the Upper Midwest, with nearly 1,400 physicians, Sanford Health is dedicated to several initiatives, including global clinics, genomic medicine and specialized centers researching cures for type 1 diabetes, breast cancer and other diseases. Sanford Health has 28,000 employees, making it the largest employer in the Dakotas. Nearly $1 billion in gifts from philanthropist Denny Sanford over the past decade have transformed how Sanford Health can improve the human condition. For information, visit sanfordhealth.org.