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

NCT02633163 | Completed Phase 2 DMC FDA Drug

• 1 other identifier: MUSC-UCMSC-001
• Study Type: interventional
• Target: 81
• Locations: 1 country
• Sites: 9

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.

Conditions

Systemic Lupus Erythematosus

Keywords

Systemic Lupus Erythematosus; Stem Cell; Lupus

Outcome Measures

Primary Outcomes (1)

• 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.

  Week 24

Secondary Outcomes (11)

• Change in SLEDAI score between groups

  Baseline to Weeks 12, 24, and 52

• Renal and non-renal organ system flares

  At or before Weeks 12, 24, and 52

• Changes in SLICC-DI

  Baseline to Week 52

• Changes in HR-QOL

  Baseline to Week 52

• Changes in Fatigue

  Baseline to Week 52

Study Arms (3)

Low Dose Mesenchymal Stem Cells (MSCs)

EXPERIMENTAL

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

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

Drug: High Dose Mesenchymal Stem Cells (MSCs)

Plasma Lyte A Solution

PLACEBO COMPARATOR

Placebo Infusion (Plasma-Lyte A solution only)

Drug: Placebo Infusion

Interventions

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.

High Dose Mesenchymal Stem Cells (MSCs)

Placebo Infusion 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.

Plasma Lyte A Solution

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.

Low Dose Mesenchymal Stem Cells (MSCs)

Eligibility Criteria

• Age: 18 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• Active CNS lupus affecting mental status
• Active lupus nephritis requiring dialysis
• 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

Sponsors & Collaborators

• Medical University of South Carolina: lead

Study Sites (9)

Cedars-Sinai Medical Center

Los Angeles, California, 90048, United States

Location

University of California - San Diego

San Diego, California, 92093, United States

Location

Emory University

Atlanta, Georgia, 30322, United States

Location

Northwestern University

Chicago, Illinois, 60611, United States

Location

The Feinstein Institute for Medical Research

Manhasset, New York, 11030, United States

Location

University of Rochester Medical Center

Rochester, New York, 14642, United States

Location

University of North Carolina at Chapel Hill

Chapel Hill, North Carolina, 27599, United States

Location

Oklahoma Medical Research Foundation

Oklahoma City, Oklahoma, 73104, United States

Location

Medical University of South Carolina

Charleston, South Carolina, 29425, United States

Location

MeSH Terms

Conditions

Lupus Erythematosus, Systemic

Condition Hierarchy (Ancestors)

Connective Tissue Diseases; Skin and Connective Tissue Diseases; Autoimmune Diseases; Immune System Diseases

Study Officials

• Gary S. Gilkeson, MD

  Medical University of South Carolina

  PRINCIPAL INVESTIGATOR

• Diane L. Kamen, MD, MSCR

  Medical University of South Carolina

  STUDY CHAIR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 15, 2015
• First Posted: December 17, 2015
• Study Start: November 18, 2018
• Primary Completion: August 5, 2025
• Study Completion: February 3, 2026
• Last Updated: February 5, 2026

Record last verified: 2026-02

Locations

US (9)