Cellular Therapy for Type 1 Diabetes Using Mesenchymal Stem Cells

The goal of this study is to determine the safety and efficacy of fresh metabolically active allogeneic umbilical cord-derived mesenchymal stromal cells (UC-MSCs) for the treatment of new-onset type 1 diabetes (T1D) and to understand the mechanisms of protection. If proven effective, such a strategy can be used as a therapeutic option for T1D patients and potentially other autoimmune disorders.

Study Overview

• Status: Active, not recruiting
• Conditions: Diabetes Mellitus, Type 1
• Intervention / Treatment: Biological: Mesenchymal Stem Cells (MSCs); Other: Placebo Infusion (Plasmalyte A with 0.5% human serum albumin)

Detailed Description

This study seeks to find and enroll participants between the ages of 18 to 40 with new onset Type 1 diabetes (T1D) within 6 months of the first dose of insulin. T1D is an autoimmune disease in which T cells attack and destroy insulin-secreting pancreatic β cells leading to insulin deficiency and hyperglycemia in patients. Life-long insulin therapy is the major treatment option. However, insulin therapy is not a cure and a safer and more effective therapy is needed.

Mesenchymal Stromal Cells (MSCs) have emerged as a novel biopharmaceutical approach for many disorders. MSCs are a cellular product that can be derived from a patient's own body (autologous) or from a donor (allogeneic). This study will obtain MSCs from umbilical cords at the time of delivery from normal women who have been extensively screened for infectious diseases. These cells produced at the MUSC Center for Cellular therapy will be used within 3 passages after collection.

Evidence from animal models and clinical trials suggests that MSC infusion suppresses autoimmune and inflammatory diseases such as T1D. One clear message from these trials is that MSCs are effective at suppressing autoimmunity and seem generally safe. This study will measure safety and efficacy of MSCs over the course of 1 year.

• Study Type: Interventional
• Enrollment (Estimated): 60
• Phase: Phase 1

Contacts and Locations

Study Locations

• United States
  • South Carolina
    • Charleston, South Carolina, United States, 29425
      • Medical University of South Carolina

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 30 years (Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion criteria:

• A new diagnosis of T1D based on the ADA criteria within 6 months of randomization.
• Male and female between the ages of 18 and 40
• Mentally stable and able to comply with the procedures of the study protocol
• Positivity for at least one T1D-associated autoantibody, such as GAD, IA-2 or ZnT8 autoantibodies
• At screening, patients must have residual β cell function with a stimulated peak C-peptide >0.2 nmol/l during a 2 hour MMTT
• Must be willing to comply with "intensive diabetes management" (* See diabetes management at MUSC below) as directed by the participant's clinician with the goal of maintaining blood glucose as close to normal as possible
• Subject must be willing to comply with the schedule of study visits and protocol requirements
• Subject with normal laboratory values of: White blood cell counts: between 4,500 to 11,000 per microliter; Platelet counts: 140,000 to 450,000 platelets per microliter of blood; Serum creatinine range is 0.6-1.3 mg/dL, Hepatic function: ALT 5 to 55 units per liter (U/L), AST 5 to 48 U/L.

Exclusion criteria:

• Evidence of retinopathy at baseline based on ophthalmologic examination or medical record review.
• Body Mass Index < 14 or >35
• Presence of malignancy
• Subject has abnormally high lipid levels that exceeds > 3 times the upper limit of normal for LDL cholesterol or triglycerides
• Subject has blood pressure greater than 160 mmHg systolic or 100 mmHg diastolic at time of consent
• Subject is being treated for severe active infection of any type
• A female subject who is breast-feeding, pregnant, or intends to become pregnant during the study.
• Subject with clinically relevant uncontrolled medical condition not associated with diabetes (e.g. severe psychiatric, hematologic, renal, hepatic, neurologic, cardiac, or respiratory disorder)
• Subjects with HgbA1c >12%, and/or fasting blood glucose >270 mg/dL and/or frequent episodes of hypoglycemia (>2 episodes per week of blood glucose levels <60 mg/dL).

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: Group A Treatment; 2.5 x 10^6 MSC per kg will be infused intravenously on Day 1	Biological: Mesenchymal Stem Cells (MSCs); Patients in Group A will receive a single MSCs infusion
Placebo Comparator: Group B Placebo; Plasmalyte with 0.5% Human Serum Albumin will be infused intravenously on Day 1	Other: Placebo Infusion (Plasmalyte A with 0.5% human serum albumin); Patients in Group B will receive a single infusion of placebo (Plasmalyte A with 0.5% human serum albumin)

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
12 month Change in C-peptide area under the curve after a 2-hour MMTT	Change in beta cell function	1 year (plus or minus 30 days) after infusion

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
6 Month Change in C-Peptide area under the curve after a 2-hour MMTT	Change in beta cell function	6 months (plus or minus 14 days) after infusion
6 Month peak C-peptide after a 2-hour MMTT	Change in beta cell function	6 months (plus or minus 14 days) after infusion
1 year peak C-peptide after a 2-hour MMTT	Change in beta cell function	1 year (plus or minus 30 days) after infusion
Change in 24-hour insulin dose per kilogram between baseline and 1 year measurements	Change in beta cell function	1 year (plus or minus 30 days) after infusion

Other Outcome Measures

Outcome Measure	Measure Description	Time Frame
Fasting and postprandial blood glucose levels after MSC infusion	Change in beta cell function	0 - 72 Hours
Changes in basal C-peptide and hemoglobin A1c	Change in beta cell function	Over the course of 1 year (0, 1, 3, 6, 12 months)
Change in serum glucagon levels	Change in alpha cell function	Over the course of 1 year (0, 1, 3, 6, 12 months)
Insulin secretion rate	Change in beta cell function	Over the course of 1 year (0, 1, 3, 6, 12 months)
Changes in islet autoanitbodies	Change in autoantibody presence or titer	Over the course of 1 year (0, 1, 3, 6, 12 months)
Change in beta cell death measurements	Determination of the mechanism of action	Over the course of 1 year (0, 1, 3, 6, 12 months)
Change in blood T-reg number and function	Determination of the mechanism of action	Over the course of 1 year (0, 1, 3, 6, 12 months)
Change in autoantigen specific T-cell response	Determination of the mechanism of action	Over the course of 1 year (0, 1, 3, 6, 12 months)
Change in blood autoreactive B cell number, B cell survival, and function	Determination of the mechanism of action	Over the course of 1 year (0, 1, 3, 6, 12 months)
Changes in mRNA expression in peripheral blood mononuclear cells after treatment	Determination of the mechanism of action	Over the course of 1 year (0, 1, 3, 6, 12 months)
Changes in serum cytokine levels after treatment	Determination of the mechanism of action	Over the course of 1 year (0, 1, 3, 6, 12 months)

Collaborators and Investigators

• Sponsor: Medical University of South Carolina
• Collaborators: National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
• Investigators: Principal Investigator: Hongjun Wang, PhD, Medical University of South Carolina

Study record dates

Study Major Dates

• Study Start (Actual): February 27, 2020
• Primary Completion (Estimated): May 30, 2027
• Study Completion (Estimated): May 30, 2027

Study Registration Dates

• First Submitted: August 16, 2019
• First Submitted That Met QC Criteria: August 16, 2019
• First Posted (Actual): August 20, 2019

Study Record Updates

• Last Update Posted (Actual): April 22, 2026
• Last Update Submitted That Met QC Criteria: April 17, 2026
• Last Verified: April 1, 2026

More Information

Terms related to this study

• Keywords: diabetes; mesenchymal stem cells; autoantibodies; Type 1 diabetes mellitus; C-peptide
• Additional Relevant MeSH Terms: Endocrine System Diseases; Metabolic Diseases; Autoimmune Diseases; Immune System Diseases; Glucose Metabolism Disorders; Nutritional and Metabolic Diseases; Diabetes Mellitus; Diabetes Mellitus, Type 1; Amino Acids, Peptides, and Proteins; Proteins; Blood Proteins; Albumins; Serum Albumin; Serum Albumin, Human; Plasmalyte A
• Other Study ID Numbers: 00085542; R01DK118529-01A1 (U.S. NIH Grant/Contract)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

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