Back to results

Cellular Therapy for Type 1 Diabetes Using Mesenchymal Stem Cells

Primary Purpose

Diabetes Mellitus, Type 1

Status

Recruiting

Phase

Phase 1

Locations

United States

Study Type

Interventional

Intervention

Mesenchymal Stem Cells (MSCs)

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

About this trial

This is an interventional treatment trial for Diabetes Mellitus, Type 1 focused on measuring mesenchymal stem cells, diabetes, autoantibodies, C-peptide, Type 1 diabetes mellitus

Eligibility Criteria

18 Years - 30 Years (Adult)All SexesDoes not accept healthy volunteers

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 30
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).

Sites / Locations

Medical University of South CarolinaRecruiting

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Placebo Comparator

Arm Label

Group A Treatment

Group B Placebo

Arm Description

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

Plasmalyte with 0.5% Human Serum Albumin will be infused intravenously on Day 1

Outcomes

Primary Outcome Measures

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

Change in beta cell function

Secondary Outcome Measures

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

Change in beta cell function

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

Change in beta cell function

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

Change in beta cell function

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

Change in beta cell function

Full Information

NCT ID

NCT04061746

First Posted

August 16, 2019

Last Updated

October 13, 2023

Sponsor

Medical University of South Carolina

Collaborators

National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)

1. Study Identification

Unique Protocol Identification Number

NCT04061746

Brief Title

Cellular Therapy for Type 1 Diabetes Using Mesenchymal Stem Cells

Official Title

Cellular Therapy for Type 1 Diabetes Using Mesenchymal Stem Cells

Study Type

Interventional

2. Study Status

Record Verification Date

October 2023

Overall Recruitment Status

Recruiting

Study Start Date

February 27, 2020 (Actual)

Primary Completion Date

March 31, 2025 (Anticipated)

Study Completion Date

March 31, 2026 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Principal Investigator

Name of the Sponsor

Medical University of South Carolina

Collaborators

National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Yes

Studies a U.S. FDA-regulated Device Product

5. Study Description

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 30 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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Diabetes Mellitus, Type 1

Keywords

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

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 1

Interventional Study Model

Parallel Assignment

Masking

ParticipantCare ProviderInvestigatorOutcomes Assessor

Allocation

Randomized

Enrollment

60 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title

Group A Treatment

Arm Type

Experimental

Arm Description

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

Arm Title

Group B Placebo

Arm Type

Placebo Comparator

Arm Description

Plasmalyte with 0.5% Human Serum Albumin will be infused intravenously on Day 1

Intervention Type

Biological

Intervention Name(s)

Mesenchymal Stem Cells (MSCs)

Intervention Description

Patients in Group A will receive a single MSCs infusion

Intervention Type

Other

Intervention Name(s)

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

Intervention Description

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

Primary Outcome Measure Information:

Title

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

Description

Change in beta cell function

Time Frame

1 year (plus or minus 30 days) after infusion

Secondary Outcome Measure Information:

Title

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

Description

Change in beta cell function

Time Frame

6 months (plus or minus 14 days) after infusion

Title

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

Description

Change in beta cell function

Time Frame

6 months (plus or minus 14 days) after infusion

Title

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

Description

Change in beta cell function

Time Frame

1 year (plus or minus 30 days) after infusion

Title

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

Description

Change in beta cell function

Time Frame

1 year (plus or minus 30 days) after infusion

Other Pre-specified Outcome Measures:

Title

Fasting and postprandial blood glucose levels after MSC infusion

Description

Change in beta cell function

Time Frame

0 - 72 Hours

Title

Changes in basal C-peptide and hemoglobin A1c

Description

Change in beta cell function

Time Frame