Phase 2 Trial of Mesenchymal Stem Cells in Systemic Lupus Erythematosus (MiSLE)

NCT ID: NCT02633163

Last Updated: 2025-08-21

Basic Information

• Recruitment Status: ACTIVE_NOT_RECRUITING
• Clinical Phase: PHASE2
• Total Enrollment: 81 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2018-11-18
• Study Completion Date: 2026-02-03

Brief 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). 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.

Conditions

Systemic Lupus Erythematosus

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: DOUBLE

Study Groups

Low Dose Mesenchymal Stem Cells (MSCs)

Mesenchymal Stem Cells (MSCs) 1 x 10\^6 cells/kg in Plasma-Lyte A solution

Group Type: EXPERIMENTAL

Low Dose Mesenchymal Stem Cells (MSCs)

Intervention Type: 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)

Mesenchymal Stem Cells MSCs 5 x 10\^6 cells/kg in Plasma-Lyte A solution

Group Type: EXPERIMENTAL

High Dose Mesenchymal Stem Cells (MSCs)

Intervention Type: DRUG

Participants will receive a single IV infusion of Mesenchymal Stem Cells MSCs 5 x 10\^6 cells/kg in Plasma-Lyte A solution.

Plasma Lyte A Solution

Placebo Infusion (Plasma-Lyte A solution only)

Group Type: PLACEBO_COMPARATOR

Placebo Infusion

Intervention Type: DRUG

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.

Interventions

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.

Intervention Type: DRUG

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.

Intervention Type: DRUG

Placebo Infusion

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.

Intervention Type: DRUG

Eligibility Criteria

Inclusion Criteria

• 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

Exclusion Criteria

• 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
• 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

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 65 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Medical University of South Carolina

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Gary S. Gilkeson, MD

Role: PRINCIPAL_INVESTIGATOR

Medical University of South Carolina

Diane L. Kamen, MD, MSCR

Role: STUDY_CHAIR

Medical University of South Carolina

Locations

Cedars-Sinai Medical Center

Los Angeles, California, United States

University of California - San Diego

San Diego, California, United States

Emory University

Atlanta, Georgia, United States

Northwestern University

Chicago, Illinois, United States

The Feinstein Institute for Medical Research

Manhasset, New York, United States

University of Rochester Medical Center

Rochester, New York, United States

University of North Carolina at Chapel Hill

Chapel Hill, North Carolina, United States

Oklahoma Medical Research Foundation

Oklahoma City, Oklahoma, United States

Medical University of South Carolina

Charleston, South Carolina, United States

Countries

United States

Other Identifiers

MUSC-UCMSC-001

Identifier Type: -

Identifier Source: org_study_id