UCMSCs Combined With Standard Therapy for the Treatment of Newly Diagnosed Type 1 Diabetes

Investigation of Dose Escalation and Cohort Expansion Study on the Safety and Efficacy of Allogeneic Umbilical Cord Mesenchymal Stem Cells(UCMSCs) Combined With Standard Therapy for Newly Diagnosed Type 1 Diabetes

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

Study Overview

• Status: Recruiting
• Conditions: Newly Diagnosed Type 1 Diabetes Mellitus
• Intervention / Treatment: Drug: UCMSCs; Drug: Placebo

Detailed 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.

• Study Type: Interventional
• Enrollment (Estimated): 24
• Phase: Not Applicable

Contacts and Locations

• Study Contact: Name: Hao Yin; Phone Number: 13901677738; Email: yinhaoshanghai@163.com

Study Locations

• China
  • Shanghai
    • Shanghai, Shanghai, China
      • Recruiting
      • Shanghai Changzheng Hospital
      • Contact: Hao Yin; Phone Number: 13901677738; Email: yinhaoshanghai@163.com

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult
• Accepts Healthy Volunteers: Yes

Description

Inclusion Criteria:

1. Meets the diagnostic criteria for diabetes according to the World Health Organization (WHO) guidelines from 1999.
2. Presence of at least one pancreatic autoantibody, or idiopathic type 1 diabetes mellitus (T1DM) with negative autoantibody testing.
3. Ages 8 to 18 years, regardless of gender.
4. Newly diagnosed T1DM within the past 6 months.
5. Fasting C-peptide ≥0.1 nmol/L and postprandial 2-hour C-peptide >0.2 nmol/L.
6. 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:

1. Diabetic ketoacidosis is not under control.
2. Severe allergic constitution.
3. BMI <14 or >35.
4. History of other autoimmune diseases, hematologic disorders.
5. HIV positive, carrier of viral hepatitis, active phase of viral hepatitis, or other uncontrolled infectious diseases.
6. 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.
7. 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);
8. Pregnancy or planning pregnancy within 3 months before or after treatment, as well as breastfeeding women.
9. Mental illness, alcohol or drug abuse, inability to comply with treatment.
10. Known or suspected tumors.
11. According to the investigator's judgment, there are other clinical conditions that may endanger the safety of the subjects.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Quadruple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Experimental	Drug: UCMSCs; Peripheral intravenous infusion of umbilical cord mesenchymal stem cells
Placebo Comparator: Placebo Comparator	Drug: Placebo; Peripheral intravenous infusion of Placebo

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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.	Week 52
Safety assessment	Changes in safety parameters of patients during the study period since baseline. Measured through the registration of adverse events and other safety parameters.	Throughout the study until Week 52

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Efficacy Assessment: HbA1c	Changes in patient HbA1c levels from baseline during the study period	Week 52
Efficacy Assessment: Dose of exogenous insulin	Changes in daily exogenous insulin requirements during the study period	Week 52

Collaborators and Investigators

• Sponsor: Shanghai Changzheng Hospital
• Investigators: Principal Investigator: Hao Yin, Shanghai Changzheng Hospital

Study record dates

Study Major Dates

• Study Start (Actual): May 23, 2024
• Primary Completion (Estimated): September 30, 2025
• Study Completion (Estimated): May 1, 2026

Study Registration Dates

• First Submitted: April 16, 2024
• First Submitted That Met QC Criteria: May 6, 2024
• First Posted (Actual): May 9, 2024

Study Record Updates

• Last Update Posted (Actual): March 25, 2025
• Last Update Submitted That Met QC Criteria: December 22, 2024
• Last Verified: May 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Endocrine System Diseases; Metabolic Diseases; Autoimmune Diseases; Immune System Diseases; Glucose Metabolism Disorders; Diabetes Mellitus; Diabetes Mellitus, Type 1
• Other Study ID Numbers: CZYZ-T1DM-2023-IIT

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: UNDECIDED

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No