The aim is to investigate the safety and tolerability of intravenous infusion of allogeneic umbilical cord mesenchymal stem cells in pediatric patients diagnosed with newly onset type 1 diabetes
This is an IIT study, where the first part is an open, dose escalating study consisting of 9 patients, 8-18 years of age. The second part is a randomized, double-blinded, placebo-controlled, IIT study in parallel design comparing allogeneic UCMSCs treatment to placebo in pediatric patients diagnosed with newly onset type 1 diabetes. Besides safety, preservation of endogenous insulin production (measured as C-peptide concentrations) together with metabolic control, diabetes treatment satisfaction and immunological profile will be assessed. A total number of 24 patients will be enrolled in the study and followed for one year after UCMSCs/placebo treatment. Patients 8-18 years of age, both male and female, diagnosed for type 1 diabetes will be eligible. Providing informed consent and fulfillment of inclusion criteria and no exclusion criteria, they will within 6 months of diagnosis be randomized.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
24
Shanghai Changzheng Hospital
Shanghai, Shanghai Municipality, China
RECRUITINGSafety assessment
Changes in safety parameters of patients during the study period since baseline. Measured through the registration of adverse events and other safety parameters.
Time frame: Throughout the study until Week 52
Efficacy Assessment
Delta-change of C-peptide Area Under the Curve (AUC) (0-120 min) for Mixed Meal Tolerance Test (MMTT) at Week 52 following UCMSCs/Placebo infusion when compared to test performed before start of treatment.
Time frame: Week 52
Efficacy Assessment: HbA1c
Changes in patient HbA1c levels from baseline during the study period
Time frame: Week 52
Efficacy Assessment: Dose of exogenous insulin
Changes in daily exogenous insulin requirements during the study period
Time frame: Week 52
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