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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 : Not yet recruiting
First Posted : December 17, 2015
Last Update Posted : July 16, 2018
Sponsor:
Information provided by (Responsible Party):
Medical University of South Carolina

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.

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

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 : Interventional  (Clinical Trial)
Estimated Enrollment : 81 participants
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
Estimated Study Start Date : August 2018
Estimated Primary Completion Date : June 2021
Estimated Study Completion Date : June 2021

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Lupus
Drug Information available for: PlasmaLyte

Arm Intervention/treatment
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.




Primary Outcome Measures :
  1. 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.



Secondary Outcome Measures :
  1. 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.

  2. 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.

  3. 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)

  4. 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

  5. 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

  6. 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

  7. 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

  8. 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

  9. 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)

  10. Cumulative systemic steroid dose [ Time Frame: Week 52 ]
    Cumulative systemic steroid dose (PO, IV, IM) at Week 52

  11. 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.



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Ages Eligible for Study:   18 Years to 65 Years   (Adult, Older 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
  • 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

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


Contacts
Contact: Gary Gilkeson (843) 789-6799 gilkeson@musc.edu

Locations
United States, California
Cedars-Sinai Medical Center Not yet recruiting
Los Angeles, California, United States, 90048
Contact: Julie Thai    310-423-2979    julie.thai@cshs.org   
Principal Investigator: Mariko L. Ishimori, MD         
Sub-Investigator: Daniel J. Wallace, MD         
University of California - San Diego Not yet recruiting
San Diego, California, United States, 92093
Contact: Kenneth Kalunian, MD    858-246-2381    kkalunian@ad.ucsd.edu   
Principal Investigator: Kenneth Kalunian, MD         
United States, Georgia
Emory University Not yet recruiting
Atlanta, Georgia, United States, 30322
Contact: Karla Caylor, RN    404-616-7553    kcaylor@emory.edu   
Principal Investigator: S. Sam Lim, MD, MPH         
Sub-Investigator: Arezou Khosroshahi, MD         
United States, Illinois
Northwestern University Not yet recruiting
Chicago, Illinois, United States, 60611
Contact: Danielle Lerner    312-503-1919    danielle.lerner1@northwestern.edu   
Principal Investigator: Rosalind Ramsey-Goldman, MD         
Sub-Investigator: Cybele Ghossein, MD         
United States, New York
University of Rochester Medical Center Not yet recruiting
Rochester, New York, United States, 14642
Contact: Debbie Campbell, RN    585-275-1647    debbie_campbell@urmc.rochester.edu   
Principal Investigator: Richard J. Looney, MD         
Sub-Investigator: Jennifer H. Anolik, MD, PhD         
United States, North Carolina
University of North Carolina at Chapel Hill Not yet recruiting
Chapel Hill, North Carolina, United States, 27599
Contact: Brenda Meier, RN    919-445-2730    brenda_meier@med.unc.edu   
Principal Investigator: William Pendergraft, MD         
United States, South Carolina
Medical University of South Carolina Not yet recruiting
Charleston, South Carolina, United States, 29425
Contact: Gary Gilkeson, MD    843-789-6799    gilkeson@musc.edu   
Contact: Angela R Brown, MA    843-792-6043    robia@musc.edu   
Principal Investigator: Gary S. Gilkeson, MD         
Sub-Investigator: Diane L. Kamen, MD, MSCR         
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

Responsible Party: Medical University of South Carolina
ClinicalTrials.gov Identifier: NCT02633163     History of Changes
Other Study ID Numbers: MUSC-UCMSC-001
First Posted: December 17, 2015    Key Record Dates
Last Update Posted: July 16, 2018
Last Verified: July 2018

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