Diabetes Mellitus, Type 1Phase 1NCT04061746
Cellular Therapy for Type 1 Diabetes Using Mesenchymal Stem Cells
A Phase 1 study testing Mesenchymal Stem Cells (MSCs) in people with Diabetes Mellitus, Type 1, run by Medical University of South Carolina at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 40 years
- Conditions the study is for
- Diabetes Mellitus, Type 1
- What is being tested?
- Mesenchymal Stem Cells (MSCs)
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 60 participants
- Where
- 1 siteUnited States
- Study dates
- 27 Feb 2020to 30 May 2027
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 40 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- 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.
Who cannot join
- 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
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- 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).
Trial locations and contacts
1 site in 1 country.
United States1 site
Medical University of South Carolina
Charleston, South Carolina, 29425
About this study
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.
- Conditions
- Diabetes Mellitus, Type 1
- Being tested
- Mesenchymal Stem Cells (MSCs), Placebo Infusion (Plasmalyte A with 0.5% human serum albumin)
- Keywords
- mesenchymal stem cells, diabetes, autoantibodies, C-peptide, Type 1 diabetes mellitus
- Medical subject headings
- Diabetes Mellitus, Type 1, Diabetes Mellitus
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Mesenchymal Stem Cells (MSCs)Biological
Patients in Group A will receive a single MSCs infusion
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)
| Study group | Role | What they receive |
|---|---|---|
| Group A Treatment | Experimental | 2.5 x 10^6 MSC per kg will be infused intravenously on Day 1 Receives: Biological: Mesenchymal Stem Cells (MSCs) |
| Group B Placebo | Placebo comparator | Plasmalyte with 0.5% Human Serum Albumin will be infused intravenously on Day 1 Receives: Other: Placebo Infusion (Plasmalyte A with 0.5% human serum albumin) |
What is being measured
The primary outcome is the main question the study is designed to answer.
12 month Change in C-peptide area under the curve after a 2-hour MMTT
Change in beta cell function
Measured: 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
Change in beta cell function
Measured: 6 months (plus or minus 14 days) after infusion
6 Month peak C-peptide after a 2-hour MMTT
Change in beta cell function
Measured: 6 months (plus or minus 14 days) after infusion
1 year peak C-peptide after a 2-hour MMTT
Change in beta cell function
Measured: 1 year (plus or minus 30 days) after infusion
Change in 24-hour insulin dose per kilogram between baseline and 1 year measurements
Change in beta cell function
Measured: 1 year (plus or minus 30 days) after infusion
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Aug 2019
- Study start27 Feb 2020
- Primary completion30 May 2027 (estimated)
- Study completion30 May 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Diabetes Mellitus, Type 1Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 60 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Medical University of South Carolina
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Hongjun Wang, Medical University of South Carolina
- Investigators
- Hongjun Wang, PhD, Principal investigator, Medical University of South Carolina
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04061746
- Sponsor's protocol ID
- 00085542
- Other IDs
- R01DK118529-01A1
- Status last verified
- Apr 2026
- First submitted
- 16 Aug 2019
- Last updated
- 22 Apr 2026
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 22 Apr 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Cellular Therapy for Type 1 Diabetes Using Mesenchymal Stem Cells
- ClinicalTrials.gov ID
- NCT04061746
- Acronym / sponsor ID
- 00085542
- Conditions
- Diabetes Mellitus, Type 1
- Keywords
- mesenchymal stem cells, diabetes, autoantibodies, C-peptide, Type 1 diabetes mellitus
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 60 participants (estimated)
- Lead sponsor
- Medical University of South CarolinaUniversity, hospital or other
- Collaborators
- National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
- Study start
- 27 Feb 2020 (actual)
- Primary completion
- 30 May 2027 (estimated)
- Study completion
- 30 May 2027 (estimated)
- First posted
- 20 Aug 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Group A Treatment | Experimental | Biological: Mesenchymal Stem Cells (MSCs)2.5 x 10^6 MSC per kg will be infused intravenously on Day 1 |
| Group B Placebo | Placebo comparator | Other: Placebo Infusion (Plasmalyte A with 0.5% human serum albumin)Plasmalyte with 0.5% Human Serum Albumin will be infused intravenously on Day 1 |
Who may qualify
- Ages
- 18 to 40 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (8 listed)
- 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
Key exclusion criteria (9 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Mesenchymal Stem Cells (MSCs)Patients in Group A will receive a single MSCs infusion | Biological | Group A Treatment |
| Placebo 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) | Other | Group B Placebo |
Primary outcome
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
4 secondary outcomes
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
6 Month peak C-peptide after a 2-hour MMTT
Time frame: 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 24-hour insulin dose per kilogram between baseline and 1 year measurements
Time frame: 1 year (plus or minus 30 days) after infusion
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Apr 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Hongjun Wang, PhD principal investigator
Medical University of South Carolina
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Mesenchymal Stem Cells (MSCs) in people with Diabetes Mellitus, Type 1, run by Medical University of South Carolina at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 22 Apr 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 22 Apr 2026; AzTrials last refreshed its copy on 27 Oct 2026.