Cellular Therapy for Type 1 Diabetes Using Mesenchymal Stem Cells

NCT04061746 | Active Not Recruiting Phase 1 FDA Drug

• 2 other identifiers: 00085542R01DK118529-01A1
• Study Type: interventional
• Target: 60
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Diabetes Mellitus, Type 1

Keywords

mesenchymal stem cells; diabetes; autoantibodies; C-peptide; Type 1 diabetes mellitus

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (4)

• 6 Month Change in C-Peptide area under the curve after a 2-hour MMTT

  6 months (plus or minus 14 days) after infusion

• 6 Month peak C-peptide after a 2-hour MMTT

  6 months (plus or minus 14 days) after infusion

• 1 year peak C-peptide after a 2-hour MMTT

  1 year (plus or minus 30 days) after infusion

• Change in 24-hour insulin dose per kilogram between baseline and 1 year measurements

  1 year (plus or minus 30 days) after infusion

Other Outcomes (11)

• Fasting and postprandial blood glucose levels after MSC infusion

  0 - 72 Hours

• Changes in basal C-peptide and hemoglobin A1c

  Over the course of 1 year (0, 1, 3, 6, 12 months)

• Change in serum glucagon levels

  Over the course of 1 year (0, 1, 3, 6, 12 months)

Study Arms (2)

Group A Treatment

EXPERIMENTAL

2.5 x 10\^6 MSC per kg will be infused intravenously on Day 1

Biological: Mesenchymal Stem Cells (MSCs)

Group B Placebo

PLACEBO COMPARATOR

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)

Interventions

Mesenchymal Stem Cells (MSCs) BIOLOGICAL

Patients in Group A will receive a single MSCs infusion

Group A Treatment

Placebo Infusion (Plasmalyte A with 0.5% human serum albumin) OTHER

Patients in Group B will receive a single infusion of placebo (Plasmalyte A with 0.5% human serum albumin)

Group B Placebo

Eligibility Criteria

• Age: 18 Years - 40 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64)

You may qualify if:

• 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.

You may not qualify if:

• 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).

Sponsors & Collaborators

• Medical University of South Carolina: lead
• National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK): collaborator

Study Sites (1)

Medical University of South Carolina

Charleston, South Carolina, 29425, United States

Location

MeSH Terms

Conditions

Diabetes Mellitus, Type 1; Diabetes Mellitus

Interventions

Plasmalyte A; Serum Albumin, Human

Condition Hierarchy (Ancestors)

Glucose Metabolism Disorders; Metabolic Diseases; Nutritional and Metabolic Diseases; Endocrine System Diseases; Autoimmune Diseases; Immune System Diseases

Intervention Hierarchy (Ancestors)

Serum Albumin; Albumins; Proteins; Amino Acids, Peptides, and Proteins; Blood Proteins

Study Officials

• Hongjun Wang, PhD

  Medical University of South Carolina

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor

Study Record Dates

• First Submitted: August 16, 2019
• First Posted: August 20, 2019
• Study Start: February 27, 2020
• Primary Completion (Estimated): May 30, 2027
• Study Completion (Estimated): May 30, 2027
• Last Updated: April 22, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)