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MsciSLE: MSCs in SLE Trial

This study has suspended participant recruitment.
(Opening a mini trial to determine safety and efficacy)
Sponsor:
ClinicalTrials.gov Identifier:
NCT02633163
First Posted: December 17, 2015
Last Update Posted: October 3, 2017
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
Information provided by (Responsible Party):
Medical University of South Carolina
  Purpose
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.

Condition Intervention Phase
Systemic Lupus Erythematosus Drug: Low Dose Mesenchymal Stem Cells (MSCs) Drug: High Dose Mesenchymal Stem Cells (MSCs) Drug: Placebo Infusion Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double (Participant, Investigator)
Primary Purpose: Treatment
Official Title: A Phase II Controlled Trial of Allogeneic Mesenchymal Stem Cells for the Treatment of Refractory Lupus

Resource links provided by NLM:


Further study details as provided by Medical University of South Carolina:

Primary Outcome Measures:
  • Clinical response defined by the SLE Responder Index [ 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 12 visit and be maintained at less than or equal to 10 mg/day through Week 24.



Secondary Outcome Measures:
  • Change in SLEDAI score between groups [ Time Frame: Baseline to Weeks 12, 24, and 52 ]
    Changes in Systemic Lupus Erythematosus Disease Activity Index (SLEDAI) between the experimental and placebo group.

  • Renal and non-renal organ system flares [ Time Frame: At or before Weeks 12, 24, and 52 ]
    Frequency of both renal and non-renal organ system flares defined by the British Isles Lupus Assessment Group (BILAG) criteria


Estimated Enrollment: 81
Anticipated Study Start Date: July 2018
Estimated Study Completion Date: June 2021
Estimated Primary Completion Date: June 2021 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Low Dose Mesenchymal Stem Cells (MSCs)
Mesenchymal Stem Cells (MSCs) 1 x 10^6 cells/kg in Plasma-Lyte A solution
Drug: 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.
Experimental: High Dose Mesenchymal Stem Cells (MSCs)
Mesenchymal Stem Cells MSCs 5 x 10^6 cells/kg in Plasma-Lyte A solution
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.
Placebo Comparator: Plasma Lyte A Solution
Placebo Infusion (Plasma-Lyte A solution only)
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.

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.

  Eligibility

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 65 Years   (Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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 34
  • 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 one BILAG A or two BILAG Bs at screening, despite standard of care therapy
  • If BILAG A or two BILAG Bs in the Renal organ system, must have completed at least 6 months of therapy with either mycophenolate mofetil or cyclophosphamide for the current episode of nephritis
  • 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
  • History of malignant neoplasm within the last 3 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; males or females not willing to use adequate contraception
  • History of renal transplantation
  • Herpes zoster within the past 90 days or any infection requiring hospitalization or intravenous antibiotics within the past 60 days
  • Clinically significant EKG or chest x-ray changes
  • Any other medical condition, related or unrelated to SLE, which 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 60 days) history of substance abuse
  Contacts and Locations
Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02633163


Locations
United States, California
Cedars-Sinai Medical Center
Los Angeles, California, United States, 90048
United States, Georgia
Emory University
Atlanta, Georgia, United States, 30322
United States, Illinois
Northwestern University
Chicago, Illinois, United States, 60611
United States, New York
University of Rochester Medical Center
Rochester, New York, United States, 14642
United States, North Carolina
University of North Carolina at Chapel Hill
Chapel Hill, North Carolina, United States, 27599
United States, South Carolina
Medical University of South Carolina
Charleston, South Carolina, United States, 29425
Sponsors and Collaborators
Medical University of South Carolina
Investigators
Principal Investigator: Gary S. Gilkeson, MD Medical University of South Carolina
Study Chair: Diane L. Kamen, MD, MSCR Medical University of South Carolina
  More Information

Responsible Party: Medical University of South Carolina
ClinicalTrials.gov Identifier: NCT02633163     History of Changes
Other Study ID Numbers: MUSC-UCMSC-001
First Submitted: December 15, 2015
First Posted: December 17, 2015
Last Update Posted: October 3, 2017
Last Verified: March 2017

Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Medical University of South Carolina:
Systemic Lupus Erythematosus
Stem Cell
Lupus

Additional relevant MeSH terms:
Lupus Erythematosus, Systemic
Connective Tissue Diseases
Autoimmune Diseases
Immune System Diseases
Plasma-lyte 148
Pharmaceutical Solutions
Ophthalmic Solutions