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Active, not recruitingNCT04061746Updated Apr 22, 2026

Cellular Therapy for Type 1 Diabetes Using Mesenchymal Stem Cells

A Phase 1 interventional study of Mesenchymal Stem Cells (MSCs) and Placebo Infusion (Plasmalyte A with 0.5% human serum albumin) in Diabetes Mellitus, Type 1, sponsored by Medical University of South Carolina. Active, not recruiting at 1 site in United States. Open to participants aged 18 Years to 40 Years. Per ClinicalTrials.gov, last updated 2026-04-22.

Sponsored by Medical University of South Carolina · Phase 1, Interventional, and Treatment

Phase
Phase 1
Study type
Interventional
Enrollment
60
Allocation
Randomized
Ages
18 Years to 40 Years
Sex
All
01

Study 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.

Read the 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.

02

Conditions studied

  • Diabetes Mellitus, Type 1

Keywords

  • mesenchymal stem cells
  • diabetes
  • autoantibodies
  • C-peptide
  • Type 1 diabetes mellitus
03

In context

Diabetes Mellitus, Type 1

3,522 studies on the registry are indexed under Diabetes Mellitus, Type 1; 577 are open to participants now.

This study's planned enrollment of 60 is above the median of 40 across 2,649 interventional studies indexed under Diabetes Mellitus, Type 1.

Browse Diabetes Mellitus, Type 1 studies →

Lead sponsor

Medical University of South Carolina is the lead sponsor of 852 studies on the registry; 165 are open to participants now.

Of its 128 completed or terminated interventional studies of FDA-regulated products, 101 (79%) have results posted.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
18 Years to 40 Years
Sexes eligible
All
Accepts healthy volunteers
No

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

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).
05

Study design

Phase
Phase 1
Primary purpose
Treatment
Allocation
Randomized
Intervention model
Parallel assignment
Masking
Quadruple (Participant, Care provider, Investigator, Outcomes assessor)
Enrollment
60 participants (estimated)

Study arms

  • Experimental
    Group A Treatment

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

    Biological: Mesenchymal Stem Cells (MSCs)

  • 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)

Interventions

  • BiologicalMesenchymal Stem Cells (MSCs)

    Patients in Group A will receive a single MSCs infusion

  • OtherPlacebo 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)

06

What researchers measure

Primary outcomes

  1. 12 month Change in C-peptide area under the curve after a 2-hour MMTT

    Change in beta cell function

    Time frame: 1 year (plus or minus 30 days) after infusion

Secondary outcomes

  1. 6 Month Change in C-Peptide area under the curve after a 2-hour MMTT

    Change in beta cell function

    Time frame: 6 months (plus or minus 14 days) after infusion

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

    Change in beta cell function

    Time frame: 6 months (plus or minus 14 days) after infusion

  3. 1 year peak C-peptide after a 2-hour MMTT

    Change in beta cell function

    Time frame: 1 year (plus or minus 30 days) after infusion

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

    Change in beta cell function

    Time frame: 1 year (plus or minus 30 days) after infusion

Other outcomes

  1. Fasting and postprandial blood glucose levels after MSC infusion

    Change in beta cell function

    Time frame: 0 - 72 Hours

  2. Changes in basal C-peptide and hemoglobin A1c

    Change in beta cell function

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

  3. Change in serum glucagon levels

    Change in alpha cell function

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

  4. Insulin secretion rate

    Change in beta cell function

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

  5. Changes in islet autoanitbodies

    Change in autoantibody presence or titer

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

  6. Change in beta cell death measurements

    Determination of the mechanism of action

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

  7. Change in blood T-reg number and function

    Determination of the mechanism of action

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

  8. Change in autoantigen specific T-cell response

    Determination of the mechanism of action

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

  9. Change in blood autoreactive B cell number, B cell survival, and function

    Determination of the mechanism of action

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

  10. Changes in mRNA expression in peripheral blood mononuclear cells after treatment

    Determination of the mechanism of action

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

  11. Changes in serum cytokine levels after treatment

    Determination of the mechanism of action

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

07

Study locations

1 site
  • Medical University of South Carolina
    Charleston, South Carolina 29425, United States
08

References and documents

Individual participant data

Plan to share: No

No publications or documents are linked to this record.

09

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Apr 22, 2026, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
10

Registry details

Key details

Study ID
NCT04061746
Lead sponsor
Medical University of South Carolina
Collaborators
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Responsible party
Hongjun Wang (Professor, Medical University of South Carolina) — Principal investigator
First posted
Aug 20, 2019
Start date
Feb 27, 2020
Primary completion
May 30, 2027 (estimated)
Completion
May 30, 2027 (estimated)
Last update
Apr 22, 2026

Study contacts

Hongjun Wang, PhD
principal investigator · Medical University of South Carolina

Oversight

FDA-regulated drug
Yes
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

Not currently enrolling

This study is active, not recruiting, as verified in Apr 2026. You cannot join it, but the record below documents what was studied.

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