- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT04061746
Cellular Therapy for Type 1 Diabetes Using Mesenchymal Stem Cells
Study Overview
Status
Conditions
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.
Study Type
Enrollment (Estimated)
Phase
- Phase 1
Contacts and Locations
Study Contact
- Name: Leah Benn, MPH
- Phone Number: 843-792-2813
- Email: bennle@musc.edu
Study Locations
-
-
South Carolina
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Charleston, South Carolina, United States, 29425
- Recruiting
- Medical University of South Carolina
-
Contact:
- Leah Benn, MPH
- Phone Number: 843-792-2813
- Email: bennle@musc.edu
-
-
Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
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 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).
Study Plan
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: Randomized
- Interventional Model: Parallel Assignment
- Masking: Quadruple
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
|
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
|
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
Time Frame: 1 year (plus or minus 30 days) after infusion
|
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
Time Frame: 6 months (plus or minus 14 days) after infusion
|
Change in beta cell function
|
6 months (plus or minus 14 days) after infusion
|
6 Month peak C-peptide after a 2-hour MMTT
Time Frame: 6 months (plus or minus 14 days) after infusion
|
Change in beta cell function
|
6 months (plus or minus 14 days) after infusion
|
1 year peak C-peptide after a 2-hour MMTT
Time Frame: 1 year (plus or minus 30 days) after infusion
|
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
Time Frame: 1 year (plus or minus 30 days) after infusion
|
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
Time Frame: 0 - 72 Hours
|
Change in beta cell function
|
0 - 72 Hours
|
Changes in basal C-peptide and hemoglobin A1c
Time Frame: Over the course of 1 year (0, 1, 3, 6, 12 months)
|
Change in beta cell function
|
Over the course of 1 year (0, 1, 3, 6, 12 months)
|
Change in serum glucagon levels
Time Frame: Over the course of 1 year (0, 1, 3, 6, 12 months)
|
Change in alpha cell function
|
Over the course of 1 year (0, 1, 3, 6, 12 months)
|
Insulin secretion rate
Time Frame: Over the course of 1 year (0, 1, 3, 6, 12 months)
|
Change in beta cell function
|
Over the course of 1 year (0, 1, 3, 6, 12 months)
|
Changes in islet autoanitbodies
Time Frame: Over the course of 1 year (0, 1, 3, 6, 12 months)
|
Change in autoantibody presence or titer
|
Over the course of 1 year (0, 1, 3, 6, 12 months)
|
Change in beta cell death measurements
Time Frame: Over the course of 1 year (0, 1, 3, 6, 12 months)
|
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
Time Frame: Over the course of 1 year (0, 1, 3, 6, 12 months)
|
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
Time Frame: Over the course of 1 year (0, 1, 3, 6, 12 months)
|
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
Time Frame: Over the course of 1 year (0, 1, 3, 6, 12 months)
|
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
Time Frame: Over the course of 1 year (0, 1, 3, 6, 12 months)
|
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
Time Frame: Over the course of 1 year (0, 1, 3, 6, 12 months)
|
Determination of the mechanism of action
|
Over the course of 1 year (0, 1, 3, 6, 12 months)
|
Collaborators and Investigators
Investigators
- Principal Investigator: Hongjun Wang, PhD, Medical University of South Carolina
Study record dates
Study Major Dates
Study Start (Actual)
Primary Completion (Estimated)
Study Completion (Estimated)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Actual)
Study Record Updates
Last Update Posted (Actual)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Additional Relevant MeSH Terms
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)?
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Studies a U.S. FDA-regulated device product
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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