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

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ClinicalTrials.gov Identifier: NCT02633163
Recruitment Status : Recruiting
First Posted : December 17, 2015
Last Update Posted : April 1, 2019
Sponsor:
Information provided by (Responsible Party):
Medical University of South Carolina

Tracking Information
First Submitted Date  ICMJE December 15, 2015
First Posted Date  ICMJE December 17, 2015
Last Update Posted Date April 1, 2019
Actual Study Start Date  ICMJE October 26, 2018
Estimated Primary Completion Date June 2021   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: March 29, 2018)
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.
Original Primary Outcome Measures  ICMJE
 (submitted: December 16, 2015)
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.
Change History Complete list of historical versions of study NCT02633163 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: March 29, 2018)
  • 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 renal and non-renal organ system flares at or before Weeks 12, 24, and 52, defined by the BILAG criteria.
  • Changes in SLICC-DI [ Time Frame: Baseline to Week 52 ]
    • Change in SLICC-DI from Baseline to Week 52 to assess for accumulation of new damage (SLE-related or treatment-related)
  • Changes in HR-QOL [ Time Frame: Baseline to Week 52 ]
    Changes in HR-QOL (measured by SF36 v2) from Baseline to Weeks 12, 24, and 52
  • Changes in Fatigue [ Time Frame: Baseline to Week 52 ]
    Changes in fatigue (measured by PROMIS Fatigue Short Form (SF)) from Baseline to Weeks 12, 24, and 52
  • Changes in Pain [ Time Frame: Baseline to Week 52 ]
    Changes in pain (measured by PROMIS Pain SF) from Baseline to Weeks 12, 24, and 52
  • Changes in Depression [ Time Frame: Baseline to Week 52 ]
    Changes in depression (measured by PROMIS Depression SF) from Baseline to Weeks 12, 24, and 52
  • Changes in patient-reported lupus-specific disease status [ Time Frame: Baseline to Week 52 ]
    Changes in patient-reported lupus-specific disease status (measured by the LupusPro and LIT) from Baseline to Weeks 12, 24, and 52
  • Steroid-sparing effect [ Time Frame: Baseline to Week 52 ]
    Steroid-sparing effect (measured by discontinuation of corticosteroids and time to discontinuation among those taking corticosteroids)
  • Cumulative systemic steroid dose [ Time Frame: Week 52 ]
    Cumulative systemic steroid dose (PO, IV, IM) at Week 52
  • Changes in the presence of serum and urine biomarkers of SLE activity: [ Time Frame: Baseline to Week 52 ]
    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.
Original Secondary Outcome Measures  ICMJE
 (submitted: December 16, 2015)
  • 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
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Phase 2 Trial of Mesenchymal Stem Cells in Systemic Lupus Erythematosus (MiSLE)
Official Title  ICMJE A Phase II Controlled Trial of Allogeneic Mesenchymal Stem Cells for the Treatment of Refractory Lupus
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.

Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double (Participant, Investigator)
Primary Purpose: Treatment
Condition  ICMJE Systemic Lupus Erythematosus
Intervention  ICMJE
  • 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.
  • 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.
  • 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.
Study Arms  ICMJE
  • Experimental: Low Dose Mesenchymal Stem Cells (MSCs)
    Mesenchymal Stem Cells (MSCs) 1 x 10^6 cells/kg in Plasma-Lyte A solution
    Intervention: Drug: Low Dose Mesenchymal Stem Cells (MSCs)
  • Experimental: High Dose Mesenchymal Stem Cells (MSCs)
    Mesenchymal Stem Cells MSCs 5 x 10^6 cells/kg in Plasma-Lyte A solution
    Intervention: Drug: High Dose Mesenchymal Stem Cells (MSCs)
  • Placebo Comparator: Plasma Lyte A Solution
    Placebo Infusion (Plasma-Lyte A solution only)
    Intervention: Drug: Placebo Infusion
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: December 16, 2015)
81
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE June 2021
Estimated Primary Completion Date June 2021   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years to 65 Years   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Gary Gilkeson (843) 792 - 6043 gilkeson@musc.edu
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT02633163
Other Study ID Numbers  ICMJE MUSC-UCMSC-001
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Medical University of South Carolina
Study Sponsor  ICMJE Medical University of South Carolina
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: Gary S. Gilkeson, MD Medical University of South Carolina
Study Chair: Diane L. Kamen, MD, MSCR Medical University of South Carolina
PRS Account Medical University of South Carolina
Verification Date March 2019

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP