Overview
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
Description
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.
Eligibility
Inclusion Criteria:
- Meets the diagnostic criteria for diabetes according to the World Health Organization (WHO) guidelines from 1999.
- Presence of at least one pancreatic autoantibody, or idiopathic type 1 diabetes mellitus (T1DM) with negative autoantibody testing.
- Ages 8 to 18 years, regardless of gender.
- Newly diagnosed T1DM within the past 6 months.
- Fasting C-peptide ≥0.1 nmol/L and postprandial 2-hour C-peptide \>0.2 nmol/L.
- Voluntary acceptance of stem cell transplantation therapy by the individual, their family members, or legal guardians, and signing of an informed consent form.
Exclusion Criteria:
- Diabetic ketoacidosis is not under control.
- Severe allergic constitution.
- BMI \<14 or \>35.
- History of other autoimmune diseases, hematologic disorders.
- HIV positive, carrier of viral hepatitis, active phase of viral hepatitis, or other uncontrolled infectious diseases.
- History of acute pancreatitis, pulmonary embolism, or other thrombotic diseases, as well as severe diseases of the heart, liver, kidneys, respiratory system, nervous system, etc.
- Suffering from gestational diabetes, monogenic diabetes, diabetes caused by pancreatic injury, or other secondary diabetes (such as Cushing's syndrome, thyroid dysfunction, or acromegaly-induced diabetes);
- Pregnancy or planning pregnancy within 3 months before or after treatment, as well as breastfeeding women.
- Mental illness, alcohol or drug abuse, inability to comply with treatment.
- Known or suspected tumors.
- According to the investigator's judgment, there are other clinical conditions that may endanger the safety of the subjects.