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Pilot Trial of Mesenchymal Stem Cells for Systemic Lupus Erythematosus

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.
 
ClinicalTrials.gov Identifier: NCT03171194
Recruitment Status : Completed
First Posted : May 31, 2017
Last Update Posted : May 2, 2019
Sponsor:
Information provided by (Responsible Party):
Medical University of South Carolina

Tracking Information
First Submitted Date  ICMJE May 4, 2017
First Posted Date  ICMJE May 31, 2017
Last Update Posted Date May 2, 2019
Actual Study Start Date  ICMJE April 27, 2017
Actual Primary Completion Date April 30, 2018   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: March 29, 2018)
Frequency of Grade 3 or higher adverse events [ Time Frame: Week 24 ]
The primary outcome measure is the frequency of Grade 3 or higher adverse events (AEs) experienced by participants at or prior to Week 24.
Original Primary Outcome Measures  ICMJE
 (submitted: May 26, 2017)
Primary Safety and Tolerability [ Time Frame: Week 24 ]
The primary objective is to assess effects of an infusion of MSCs when added to standard-of-care therapy in patients with mild to moderately active SLE. The primary outcome measure is the frequency of Grade 3 or higher adverse events (AEs) experienced by participants at or prior to Week 24.
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: March 29, 2018)
  • Frequency of All Adverse Events [ Time Frame: Baseline to Week 52 ]
    Frequency of all adverse events (AEs) including any serious AEs (SAEs) at or prior to Week 52.
  • Change in Disease Activity [ Time Frame: Baseline to Week 24 ]
    Change in SLE disease activity between Baseline and Week 24 measured by change in SLEDAI score and change in prednisone dose.
  • Change in Patient Reported Outcomes - Life [ Time Frame: Baseline to Week 24 ]
    Changes between Baseline and Week 24 in patient-reported quality of life
  • Change in Patient Reported Outcomes - Fatigue [ Time Frame: Baseline to Week 24 ]
    Changes between Baseline and Week 24 in patient-reported measures of fatigue.
  • Change in Patient Reported Outcomes - Pain [ Time Frame: Baseline to Week 24 ]
    Changes between Baseline and Week 24 in patient-reported measures of pain.
  • Change in Patient Reported Outcomes - Depression [ Time Frame: Baseline to Week 24 ]
    Changes between Baseline and Week 24 in patient-reported measures of depression.
  • Change in Disease Biomarkers - Cellular [ Time Frame: Baseline to Week 24 ]
    Changes between Baseline and Week 24 in cellular markers of inflammation and autoimmunity. Mechanistically, the study will test the hypothesis that MSC infusions in patients with active SLE will increase Treg numbers via enhancing TGF-beta activity while decreasing T and B cell effector subsets.
  • Change in Disease Biomarkers - Serum [ Time Frame: Baseline to Week 24 ]
    Changes between Baseline and Week 24 in serum markers of inflammation and autoimmunity. Mechanistically, the study will test the hypothesis that MSC infusions in patients with active SLE will increase Treg numbers via enhancing TGF-beta activity while decreasing T and B cell effector subsets.
Original Secondary Outcome Measures  ICMJE
 (submitted: May 26, 2017)
  • Frequency of Adverse Events [ Time Frame: Week 52 ]
    Frequency of all adverse events (AEs) including any serious AEs (SAEs) at or prior to Week 52.
  • Change in Disease Activity [ Time Frame: Week 24 ]
    Change in SLE disease activity between Baseline and Week 24 measured by change in SLEDAI score and change in prednisone dose.
  • Change in Patient Reported Outcomes - Life [ Time Frame: Week 24 ]
    Changes between Baseline and Week 24 in patient-reported quality of life
  • Change in Patient Reported Outcomes - Fatigue [ Time Frame: Week 24 ]
    Changes between Baseline and Week 24 in patient-reported quality of fatigue.
  • Change in Patient Reported Outcomes - Pain [ Time Frame: Week 24 ]
    Changes between Baseline and Week 24 in patient-reported quality of pain.
  • Change in Patient Reported Outcomes - Depression [ Time Frame: Week 24 ]
    Changes between Baseline and Week 24 in patient-reported quality of depression.
  • Change in Disease Biomarkers - Cellular [ Time Frame: Week 24 ]
    Changes between Baseline and Week 24 in cellular. Mechanistically, the study will test the hypothesis that MSC infusions in patients with active SLE will increase Treg numbers via enhancing TGF-beta activity while decreasing T and B cell effector subsets.
  • Change in Disease Biomarkers - Serum [ Time Frame: Week 24 ]
    Changes between Baseline and Week 24 in serum. Mechanistically, the study will test the hypothesis that MSC infusions in patients with active SLE will increase Treg numbers via enhancing TGF-beta activity while decreasing T and B cell effector subsets.
  • Change in Disease Biomarkers - Urine Biomarkers [ Time Frame: Week 24 ]
    Changes between Baseline and Week 24 in urine biomarkers. Mechanistically, the study will test the hypothesis that MSC infusions in patients with active SLE will increase Treg numbers via enhancing TGF-beta activity while decreasing T and B cell effector subsets.
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Pilot Trial of Mesenchymal Stem Cells for Systemic Lupus Erythematosus
Official Title  ICMJE A Phase I Safety Trial of Allogeneic Mesenchymal Stem Cells for Systemic Lupus Erythematosus
Brief Summary The purpose of this study is to evaluate the safety of mesenchymal stromal cells (MSCs) obtained from umbilical cords for the treatment of adults with active systemic lupus erythematosus (SLE).
Detailed Description

This open label trial will evaluate the safety of allogeneic MSCs for the treatment of adults with moderate to severely active systemic lupus erythematosus (SLE). MSCs will be derived from healthy donor umbilical cord cells and 1 dose of MSCs will be tested. MUSC has a good manufacturing practice (GMP) quality Clean Cell Facility to ensure the quality and safety of the MSCs prior to infusing into study participants. The goal of this study is to determine the safety of MSC infusion in patients with SLE when added to standard of care for SLE.

The MSCs used in this trial are cells that are obtained from the umbilical cords of healthy donors having an elective Caesarean section and who have been screened to be sure that they are free of any infectious diseases. These investigational cells will be collected and processed so that they can be used as an infusion treatment. An infusion is when a drug (in this case the MSCs) is administered directly into the blood stream via a vein, usually located in the arm or hand. All participants will receive standard of care and their safety will be monitored throughout the study.

Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE System; Lupus Erythematosus
Intervention  ICMJE Drug: Low Dose Mesenchymal Stem Cells (MSCs)
Mesenchymal stromal/stem cells (MSCs) are cells that can be derived from umbilical cords, bone marrow, adipose tissue, and dental pulp, among other sites. MSCs have the ability to mediate a range of immuno-modulatory actions for both the innate and adaptive immune systems.
Study Arms  ICMJE Experimental: 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.
Intervention: Drug: Low Dose Mesenchymal Stem Cells (MSCs)
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 Completed
Actual Enrollment  ICMJE
 (submitted: May 26, 2017)
6
Original Estimated Enrollment  ICMJE Same as current
Actual Study Completion Date  ICMJE October 25, 2018
Actual Primary Completion Date April 30, 2018   (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
  • Definite SLE by meeting either SLICC or ACR Classification Criteria for SLE
  • Evidence of a positive ANA (≥1:80 titer) or positive dsDNA antibody test within 6 months of screening
  • Clinically mild to moderately active SLE determined by SLEDAI score ≥4 and ≤10 at screening, despite SOC therapy
  • If the patient has BILAG A or two BILAG Bs in the renal organ system, he/she 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 abnormalities
  • 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 3 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
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 information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03171194
Other Study ID Numbers  ICMJE 00061632
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
Plan to Share IPD: No
Responsible Party Medical University of South Carolina
Study Sponsor  ICMJE Medical University of South Carolina
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Chair: Diane L. Kamen, MD, MSCR Medical University of South Carolina
PRS Account Medical University of South Carolina
Verification Date April 2019

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