Cellular Therapy for Type 1 Diabetes Using Mesenchymal Stem Cells

Phase 1

Recruiting

• Conditions: Diabetes Mellitus, Type 1
• Interventions: Biological: Mesenchymal Stem Cells (MSCs); Other: Placebo Infusion (Plasmalyte A with 0.5% human serum albumin)

• Registration Number: NCT04061746
• Lead Sponsor: Medical University of South Carolina

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.

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 Timeline

• Study First Submitted: 2019-08-16
• Study First Submitted QC: 2019-08-16
• Study First Posted: 2019-08-20
• Study Start: 2020-02-27
• Status Verified: 2025-04
• Last Update Submitted: 2025-04-29
• Last Update Posted: 2025-05-02
• Primary Completion: 2026-03-31
• Study Completion: 2027-03-31

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 60

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Group A Treatment	Mesenchymal Stem Cells (MSCs)	2.5 x 10\^6 MSC per kg will be infused intravenously on Day 1
Group B Placebo	Placebo Infusion (Plasmalyte A with 0.5% human serum albumin)	Plasmalyte with 0.5% Human Serum Albumin will be infused intravenously on Day 1

Primary Outcome Measures

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

Secondary Outcome Measures

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

Trial Locations

Locations (1)

Medical University of South Carolina; 🇺🇸 Charleston, South Carolina, United States