Systemic Lupus ErythematosusPhase 2NCT02633163
Phase 2 Trial of Mesenchymal Stem Cells in Systemic Lupus Erythematosus (MiSLE)
A Phase 2 study testing Low Dose Mesenchymal Stem Cells (MSCs) and High Dose Mesenchymal Stem Cells (MSCs) in people with Systemic Lupus Erythematosus, run by Medical University of South Carolina at 9 sites in United States.
Trial summary
The purpose of this study is to evaluate the efficacy and safety of mesenchymal stem cells (MSCs) obtained from umbilical cords for the treatment of adults with systemic lupus erythematosus (SLE). Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Systemic Lupus Erythematosus
- What is being tested?
- Low Dose Mesenchymal Stem Cells (MSCs) and High Dose 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
- 81 participants
- Where
- 9 sitesUnited States
- Study dates
- 18 Nov 2018to 3 Feb 2026
- Phase
- Phase 2
- Are there results?
- None posted in the registry
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 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Patients between 18 and 65 years old, male or female, of any race
- Historical presence of at least 4 of 11 of the ACR Classification Criteria
- Evidence of a positive ANA (≥1:80 titer) or positive dsDNA antibody test within 6 months of screening
- Clinically active SLE determined by SLEDAI score ≥6 and the presence of at least one BILAG A or BILAG B at screening, despite standard-of-care therapy
- If the patient has a BILAG A or BILAG B score in the renal organ system, he/she must have completed at least 6 months of therapy for the current episode of nephritis prior to Screening. Therapy must include at least 6 months of mycophenolate or at least 3 months of cyclophosphamide followed by mycophenolate or azathioprine
- Able and willing to give written informed consent
Who cannot join
- Active CNS lupus affecting mental status
- Active lupus nephritis requiring dialysis
- Laboratory exclusions: eGFR <30, WBC <2.0/mm3, hemoglobin <8 g/dL, platelet count <30,000/mm3, liver enzymes AST or ALT >4 times upper limit normal.
- Positive testing for HIV, hepatitis B or hepatitis C, tuberculosis (TB), or chest X-ray (CXR) findings consistent with TB or latent fungal infection.
- History of malignant neoplasm within the last 5 years, except for adequately treated cancers of the skin (basal or squamous cell) or carcinoma in situ of the uterine cervix
- Pregnant or breast feeding
Show all 16Show fewer
- A woman of childbearing potential (not post-menopausal or surgically sterile) who is not willing to use adequate contraception
- History of renal transplantation
- Herpes zoster within the past 90 days or any infection requiring hospitalization or intravenous or intramuscular antibiotics within the past 60 days
- Clinically significant EKG or chest X-ray changes
- Any other medical condition, related or unrelated to SLE, that in the opinion of the investigator would render the patient inappropriate or too unstable to complete study protocol
- Use of prednisone >0.5 mg/kg/day (or equivalent corticosteroid) within 1 month of Baseline visit
- Change or addition to immunosuppressant regimen within 3 months of Baseline visit (except corticosteroids); Use of other experimental therapeutic agents within 3 months of Baseline visit
- Having received belimumab within 2 months of Baseline, or having received rituximab or other B cell depleting biologic therapy within 6 months of Baseline.
- Comorbidities requiring corticosteroid therapy
- Current substance abuse or recent (within one year) history of substance abuse
Trial locations and contacts
9 sites in 1 country.
9 sites in 1 country
United States9 sites
Emory University
Atlanta, Georgia, 30322
University of North Carolina at Chapel Hill
Chapel Hill, North Carolina, 27599
Medical University of South Carolina
Charleston, South Carolina, 29425
Northwestern University
Chicago, Illinois, 60611
Cedars-Sinai Medical Center
Los Angeles, California, 90048
The Feinstein Institute for Medical Research
Manhasset, New York, 11030
Oklahoma Medical Research Foundation
Oklahoma City, Oklahoma, 73104
University of Rochester Medical Center
Rochester, New York, 14642
University of California - San Diego
San Diego, California, 92093
About this study
Official title: A Phase II Controlled Trial of Allogeneic Mesenchymal Stem Cells for the Treatment of Refractory Lupus
The purpose of this study is to evaluate the efficacy and safety of mesenchymal stem cells (MSCs) obtained from umbilical cords for the treatment of adults with systemic lupus erythematosus (SLE). The goal of this study is to determine if patients receiving an MSC infusion plus standard of care respond better than patients receiving placebo infusion plus standard of care.
Read the detailed description
A phase 2 multicenter (several medical research centers participating), placebo controlled, randomized (assigned by chance), double blind (neither the participant nor the investigator will know if active drug or placebo is assigned) trial to evaluate the safety and efficacy of mesenchymal stem cells (MSCs) for the treatment of systemic lupus erythematosus (SLE) in adults.
The MSCs will be obtained from healthy donor umbilical cords and two doses of MSCs will be tested. The cells will be produced at the Medical University of South Carolina (MUSC) and will be shipped to other participating centers for patients with SLE. Participants will receive either active drug or placebo through a single IV infusion. All participants will receive standard of care and their safety will be monitored throughout the study.
- Conditions
- Systemic Lupus Erythematosus
- Being tested
- Low Dose Mesenchymal Stem Cells (MSCs), High Dose Mesenchymal Stem Cells (MSCs), Placebo Infusion
- Keywords
- Systemic Lupus Erythematosus, Stem Cell, Lupus
- Medical subject headings
- Lupus Erythematosus, Systemic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Low Dose Mesenchymal Stem Cells (MSCs)Drug
Participants will receive a single IV infusion of Mesenchymal Stem Cells (MSCs) 1 x 10^6 cells/kg in Plasma-Lyte A solution. All participants will receive the infusion at the Baseline (Day 0) visit. All participants will continue on their standard-of-care therapy during the trial.
High Dose Mesenchymal Stem Cells (MSCs)Drug
Participants will receive a single IV infusion of Mesenchymal Stem Cells MSCs 5 x 10^6 cells/kg in Plasma-Lyte A solution.
Placebo InfusionDrug
Participants will receive a placebo infusion that does not contain any mesenchymal stem cells.The placebo infusion will consist of Plasma-Lyte A, which is the same vehicle used to deliver the MSCs in the experimental groups.
| Study group | Role | What they receive |
|---|---|---|
| Low Dose Mesenchymal Stem Cells (MSCs) | Experimental | Mesenchymal Stem Cells (MSCs) 1 x 10^6 cells/kg in Plasma-Lyte A solution Receives: Drug: Low Dose Mesenchymal Stem Cells (MSCs) |
| High Dose Mesenchymal Stem Cells (MSCs) | Experimental | Mesenchymal Stem Cells MSCs 5 x 10^6 cells/kg in Plasma-Lyte A solution Receives: Drug: High Dose Mesenchymal Stem Cells (MSCs) |
| Plasma Lyte A Solution | Placebo comparator | Placebo Infusion (Plasma-Lyte A solution only) Receives: Drug: Placebo Infusion |
What is being measured
The primary outcome is the main question the study is designed to answer.
Clinical response at Week 24 as defined by the SLE Responder Index (SRI):
Systemic Lupus Erythematosus Responder Index (SRI) is defined as a greater than or equal to (≥) 4 point reduction in the Systemic Lupus Erythematosus Disease Activity Index score (SLEDAI), no new British Isles Lupus Assessment Group (BILAG) A or no more than 1 new BILAG B domain score, and no deterioration from Baseline in the Physician's Global Assessment (PGA) by greater than or equal to (≥) 0.3 points.
Additionally, to be a "responder", corticosteroid dose must be less than of equal to (≤)10 mg/day of prednisone or equivalent by the Week 20 visit and be maintained at less than or equal to 10 mg/day through Week 24.
Measured: Week 24
Secondary outcomes (11)
Change in SLEDAI score between groups
Changes in Systemic Lupus Erythematosus Disease Activity Index (SLEDAI) between the experimental and placebo group.
Measured: Baseline to Weeks 12, 24, and 52
Renal and non-renal organ system flares
Frequency of renal and non-renal organ system flares at or before Weeks 12, 24, and 52, defined by the BILAG criteria.
Measured: At or before Weeks 12, 24, and 52
Changes in SLICC-DI
- Change in SLICC-DI from Baseline to Week 52 to assess for accumulation of new damage (SLE-related or treatment-related)
Measured: Baseline to Week 52
Changes in HR-QOL
Changes in HR-QOL (measured by SF36 v2) from Baseline to Weeks 12, 24, and 52
Measured: Baseline to Week 52
Changes in Fatigue
Changes in fatigue (measured by PROMIS Fatigue Short Form (SF)) from Baseline to Weeks 12, 24, and 52
Measured: Baseline to Week 52
Changes in Pain
Changes in pain (measured by PROMIS Pain SF) from Baseline to Weeks 12, 24, and 52
Measured: Baseline to Week 52
Changes in Depression
Changes in depression (measured by PROMIS Depression SF) from Baseline to Weeks 12, 24, and 52
Measured: Baseline to Week 52
Changes in patient-reported lupus-specific disease status
Changes in patient-reported lupus-specific disease status (measured by the LupusPro and LIT) from Baseline to Weeks 12, 24, and 52
Measured: Baseline to Week 52
Steroid-sparing effect
Steroid-sparing effect (measured by discontinuation of corticosteroids and time to discontinuation among those taking corticosteroids)
Measured: Baseline to Week 52
Cumulative systemic steroid dose
Cumulative systemic steroid dose (PO, IV, IM) at Week 52
Measured: Week 52
Changes in the presence of serum and urine biomarkers of SLE activity:
Changes in the presence of serum and urine biomarkers of SLE activity: SLE-related cytopenias, low serum complement levels, anti-dsDNA levels or urine protein measures from Baseline to Weeks 12, 24, and 52.
Measured: Baseline to Week 52
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Dec 2015 (estimated)
- Study start18 Nov 2018
- Primary completion5 Aug 2025
- Study completion3 Feb 2026
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Systemic Lupus ErythematosusStudies of the same condition, open and under way.
- More trials run by Medical University of South CarolinaThe sponsor's other registered studies.
- 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 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 81 participants (actual)
Sponsor and investigators
- Lead sponsor
- Medical University of South Carolina
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Gary Gilkeson, Medical University of South Carolina
- Investigators
- Gary S. Gilkeson, MD, Principal investigator, Medical University of South CarolinaDiane L. Kamen, MD, MSCR, Study chair, Medical University of South Carolina
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02633163
- Sponsor's protocol ID
- MUSC-UCMSC-001
- Status last verified
- Aug 2026
- First submitted
- 15 Dec 2015
- Last updated
- 24 Aug 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 24 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase II Controlled Trial of Allogeneic Mesenchymal Stem Cells for the Treatment of Refractory Lupus
- ClinicalTrials.gov ID
- NCT02633163
- Acronym / sponsor ID
- MUSC-UCMSC-001
- Conditions
- Systemic Lupus Erythematosus
- Keywords
- Systemic Lupus Erythematosus, Stem Cell, Lupus
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 81 participants (actual)
- Lead sponsor
- Medical University of South CarolinaUniversity, hospital or other
- Study start
- 18 Nov 2018 (actual)
- Primary completion
- 5 Aug 2025 (actual)
- Study completion
- 3 Feb 2026 (actual)
- First posted
- 17 Dec 2015 (estimated)
- 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 purpose of this study is to evaluate the efficacy and safety of mesenchymal stem cells (MSCs) obtained from umbilical cords for the treatment of adults with systemic lupus erythematosus (SLE). The goal of this study is to determine if patients receiving an MSC infusion plus standard of care respond better than patients receiving placebo infusion plus standard of care.
Detailed description
A phase 2 multicenter (several medical research centers participating), placebo controlled, randomized (assigned by chance), double blind (neither the participant nor the investigator will know if active drug or placebo is assigned) trial to evaluate the safety and efficacy of mesenchymal stem cells (MSCs) for the treatment of systemic lupus erythematosus (SLE) in adults.
The MSCs will be obtained from healthy donor umbilical cords and two doses of MSCs will be tested. The cells will be produced at the Medical University of South Carolina (MUSC) and will be shipped to other participating centers for patients with SLE. Participants will receive either active drug or placebo through a single IV infusion. All participants will receive standard of care and their safety will be monitored throughout the study.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Low Dose Mesenchymal Stem Cells (MSCs) | Experimental | Drug: Low Dose Mesenchymal Stem Cells (MSCs)Mesenchymal Stem Cells (MSCs) 1 x 10^6 cells/kg in Plasma-Lyte A solution |
| High Dose Mesenchymal Stem Cells (MSCs) | Experimental | Drug: High Dose Mesenchymal Stem Cells (MSCs)Mesenchymal Stem Cells MSCs 5 x 10^6 cells/kg in Plasma-Lyte A solution |
| Plasma Lyte A Solution | Placebo comparator | Drug: Placebo InfusionPlacebo Infusion (Plasma-Lyte A solution only) |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- Patients between 18 and 65 years old, male or female, of any race
- Historical presence of at least 4 of 11 of the ACR Classification Criteria
- Evidence of a positive ANA (≥1:80 titer) or positive dsDNA antibody test within 6 months of screening
- Clinically active SLE determined by SLEDAI score ≥6 and the presence of at least one BILAG A or BILAG B at screening, despite standard-of-care therapy
- If the patient has a BILAG A or BILAG B score in the renal organ system, he/she must have completed at least 6 months of therapy for the current episode of nephritis prior to Screening. Therapy must include at least 6 months of mycophenolate or at least 3 months of cyclophosphamide followed by mycophenolate or azathioprine
Key exclusion criteria (16 listed)
- Active CNS lupus affecting mental status
- Active lupus nephritis requiring dialysis
- Laboratory exclusions: eGFR <30, WBC <2.0/mm3, hemoglobin <8 g/dL, platelet count <30,000/mm3, liver enzymes AST or ALT >4 times upper limit normal.
- Positive testing for HIV, hepatitis B or hepatitis C, tuberculosis (TB), or chest X-ray (CXR) findings consistent with TB or latent fungal infection.
- History of malignant neoplasm within the last 5 years, except for adequately treated cancers of the skin (basal or squamous cell) or carcinoma in situ of the uterine cervix
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 |
|---|---|---|
| Low Dose Mesenchymal Stem Cells (MSCs)Participants will receive a single IV infusion of Mesenchymal Stem Cells (MSCs) 1 x 10^6 cells/kg in Plasma-Lyte A solution. All participants will receive the infusion at the Baseline (Day 0) visit. All participants will continue on their standard-of-care therapy during the trial. | Drug | Low Dose Mesenchymal Stem Cells (MSCs) |
| High Dose Mesenchymal Stem Cells (MSCs)Participants will receive a single IV infusion of Mesenchymal Stem Cells MSCs 5 x 10^6 cells/kg in Plasma-Lyte A solution. | Drug | High Dose Mesenchymal Stem Cells (MSCs) |
| Placebo InfusionParticipants will receive a placebo infusion that does not contain any mesenchymal stem cells.The placebo infusion will consist of Plasma-Lyte A, which is the same vehicle used to deliver the MSCs in the experimental groups. | Drug | Plasma Lyte A Solution |
Primary outcome
Clinical response at Week 24 as defined by the SLE Responder Index (SRI):
Time frame: Week 24
Systemic Lupus Erythematosus Responder Index (SRI) is defined as a greater than or equal to (≥) 4 point reduction in the Systemic Lupus Erythematosus Disease Activity Index score (SLEDAI), no new British Isles Lupus Assessment Group (BILAG) A or no more than 1 new BILAG B domain score, and no deterioration from Baseline in the Physician's Global Assessment (PGA) by greater than or equal to (≥) 0.3 points. Additionally, to be a "responder", corticosteroid dose must be less than of equal to (≤)10 mg/day of prednisone or equivalent by the Week 20 visit and be maintained at less than or equal to 10 mg/day through Week 24.
11 secondary outcomes
Change in SLEDAI score between groups
Time frame: Baseline to Weeks 12, 24, and 52
Renal and non-renal organ system flares
Time frame: At or before Weeks 12, 24, and 52
Changes in SLICC-DI
Time frame: Baseline to Week 52
Changes in HR-QOL
Time frame: Baseline to Week 52
Changes in Fatigue
Time frame: Baseline to Week 52
Changes in Pain
Time frame: Baseline to Week 52
Changes in Depression
Time frame: Baseline to Week 52
Changes in patient-reported lupus-specific disease status
Time frame: Baseline to Week 52
Steroid-sparing effect
Time frame: Baseline to Week 52
Cumulative systemic steroid dose
Time frame: Week 52
Changes in the presence of serum and urine biomarkers of SLE activity:
Time frame: Baseline to Week 52
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Aug 2026.
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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.
Gary S. Gilkeson, MD principal investigator
Medical University of South Carolina
Diane L. Kamen, MD, MSCR study chair
Medical University of South Carolina
9 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Low Dose Mesenchymal Stem Cells (MSCs) and High Dose Mesenchymal Stem Cells (MSCs) in people with Systemic Lupus Erythematosus, run by Medical University of South Carolina at 9 sites 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 6 inclusion and 16 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: finished, as of the sponsor's last update on 24 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 9 sites 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Clinical response at Week 24 as defined by the SLE Responder Index (SRI): (time frame: Week 24). 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 24 Aug 2026.