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Mesenchymal Stromal Cells (MSC´s) in Renal Lupus (MSC-ROLE)

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ClinicalTrials.gov Identifier: NCT03917797
Recruitment Status : Recruiting
First Posted : April 17, 2019
Last Update Posted : February 8, 2023
Sponsor:
Information provided by (Responsible Party):
Fernando E. Figueroa MD, Universidad de los Andes, Chile

Study Description
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Brief Summary:
Phase II Clinical Trial to Assess the dose-response and Efficacy of Umbilical Cord-derived Mesenchymal Stromal Cells (MSCs) in Severe Renal Systemic Lupus Erythematosus (SLE).

Condition or disease Intervention/treatment Phase
Lupus Erythematosus, Systemic Lupus Glomerulonephritis Biological: MSC treatment Drug: Standard of Care Drug: Placebo Phase 2

Detailed Description:
Phase IIa trial of escalating doses of intravenous (i.v.) MSCs in active SLE, followed by a Phase IIb, triple blind, controlled assessment of the selected MSC dosing versus Placebo, in SLE patients receiving Standard of Care Therapy for Severe Renal Disease,
Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 39 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Intervention Model Description: Superiority trial comparing MSCs versus Placebo in SLE patients with severe renal disease receiving Standard of Care treatment.
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Masking Description: Masking for patients, physicians providing patient care and outcome assessors.
Primary Purpose: Treatment
Official Title: Dose-response and Efficacy of Umbilical Cord-derived Mesenchymal Stromal Cells in Renal Systemic Lupus Erythematosus
Actual Study Start Date : April 2, 2019
Estimated Primary Completion Date : December 2024
Estimated Study Completion Date : December 2025

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Lupus

Arms and Interventions
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Arm Intervention/treatment
Experimental: MSC treatment
Intervention: a previously selected dose of MSCs (Phase IIa) will be administered by i.v. infusion at baseline and 6 months of follow-up (total 12 months), to patients with Severe Renal SLE subject also to Standard of Care treatment with Methylprednisolone and Cyclophosphamide followed by Mycophenolate.
 Biological: MSC treatment
Umbilical cord-derived Mesenchymal Stromal Cell
Other Name: Cellistem ® Lupus

Drug: Standard of Care
Methylprednisolone; Cyclophosphamide; Prednisone; Mycophenolate
Other Name: Standard of Care for Lupus Nephritis
Placebo Comparator: Placebo
Intervention: A Placebo (infusion vehicle) will be administered by i.v. infusion at baseline and 6 months of follow-up (total 12 months), to patients with Severe Renal SLE subject also to Standard of Care treatment with Methylprednisolone and Cyclophosphamide followed by Mycophenolate.
 Drug: Standard of Care
Methylprednisolone; Cyclophosphamide; Prednisone; Mycophenolate
Other Name: Standard of Care for Lupus Nephritis

Drug: Placebo
MSC infusion vehicle
Other Name: Placebo (for MSC)


Outcome Measures
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Primary Outcome Measures :
  1. Achievement of Global Renal Response (GR) at Study Endpoint [ Time Frame: 12 months ]
    Proportion of Patients that achieve Complete (CR) or Partial (PR) Renal Response at Endpoint


Secondary Outcome Measures :
  1. Achievement of Complete Renal Response (CR) at Study Endpoint [ Time Frame: 12 months ]
    Proportion of Patients that achieve CR criteria including: 1) Urinary Protein:Creatinine (UPC) ratio < 0.5; 2) estimated Glomerular Filtration Rate (GFR) ≥ 120 ml/min/m2, or at least 80% of baseline; 3) urinalysis < 10 red blood cells (RBC) and no RBC casts per high power field; 4) Prednisone dose ≤10 mg/day.

  2. Achievement of Partial Renal Response (PR) at Study Endpoint [ Time Frame: 12 months ]
    Proportion of Patients that achieve PR criteria including: 1) reduction of UPC ratio to at least 50% of baseline; 2) estimated GFR ≥120 ml/min/m2, or at least 80% of baseline; 3) Prednisone dose ≤10 mg/day.

  3. Treatment Failure [ Time Frame: 24 weeks and 12 months ]
    Proportion of Patients that fulfill any of the following criteria for Treatment Failure including: 1) Daily Prednisone dose cannot be reduced ≤ 10 mg at week 24; 2) Daily Prednisone is increased above 10 mg after week 24; 3) Introduction of a new immunosuppressive regimen, not included in the trial; 4) Use of Rituximab prior to month 12.

  4. Response of SLE Responder Index (SRI). [ Time Frame: 12 months ]

    Proportion of Patients that achieve SRI response, defined as a >4-point reduction in the SELENA-SLEDAI score, no new British Isles Lupus Assessment Group [BILAG] A organ domain score and no more than 1 new BILAG B score, with no worsening in physician's global assessment score versus baseline).

    The Safety of Estrogens in Lupus Erythematosus National Assessment (SELENA) version of the SLE Disease Activity Index (SLEDAI) is employed for this calculation.(SELENA-SLEDAI score). The SELENA-SLEDAI score addresses 24 descriptors in 9 organ-systems. Disease worsening increases the score that ranges from 0-105.

    The BILAG addresses 97 items in organ-system domains, in an ordinal (A-E) scale, converted to a numerical (0-96) scale for usual calculations.


  5. Selena Sledai [ Time Frame: 12 months ]
    Average change in Selena Sledai Score in patients and controls

  6. BILAG score [ Time Frame: 12 months ]
    Average hange in BILAG score in patients and controls

  7. Disease Flares [ Time Frame: 12 months ]
    Proportion of patients that experience flares as defined in the Selena Flare Index (SFI). Mild/Moderate Flares are defined by change of 3 or more points in the SELENA-SLEDAI score. Severe Flares are defined as an increase in the SELENA-SLEDAI score to more than 12 points

  8. Biomarker Response [ Time Frame: 24 weeks and 12 months ]
    Changes in the levels of disease relevant biomarkers in peripheral blood/plasma, including 1) anti-dsDNA antibodies by ELISA; 2) complement proteins C3/C4 by nephelometry (mg/dL); 3) Percentage of CD4+ T helper cell subpopulations (Th1, Th17, Treg) and 4) B cell subpopulations (Naive, Memory, Transitional) by Flow cytometry; and 5) Cytokine Panel by Luminex, including Tumor Necrosis Factor (TNF) alpha, Transforming Growth Factor (TGF) Beta1, Interleukin (lL) 6, IL-17A, IL-10, B-cell activating factor/B Lymphocyte Stimulator (BAFF/BLys), Monocyte chemoattractant protein-1 (MCP-1/CCL2), C-X-C motif chemokine 10 (CXCL10), Interferon (IFN) gamma.


Eligibility Criteria
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Ages Eligible for Study:   18 Years to 75 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Fulfilling 1997 updated American College of Rheumatology (ACR) Criteria or 2012 SLICC Classification Criteria for SLE
  • Seropositive for antinuclear (≥1:80) and/or anti-DNA antibodies
  • Fulfilling following criteria for active renal disease:

Class III or IV proliferative disease (ISN/RPS) Renal Biopsy within 12 months plus...

Active Urinary Sediment (> 5 red blood cells/high-power field and/or >8 white blood cells/high-power field and/or cylindruria during the current flare).

UPC ratio ≥ 1

Exclusion Criteria:

  • Estimated GFR < 40ml/min/m2
  • Addition during prior 3 months of randomization of: Bolus methylprednisolone or new immunosuppressive drug or intravenous immunoglobulin (IVIG) or Plasmapheresis.
  • Addition during prior 6 months of randomization of Cyclophosphamide
  • Addition during prior 12 months of randomization of Biological anti-B cell therapy
  • Coexisting uncontrolled morbidity; Pregnancy or planned Pregnancy within next 12 months; uncontrolled infection or neoplastic disease. Pending unresolved surgical indication.
Contacts and Locations
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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): NCT03917797


Contacts
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Contact: Fernando F E, MD +56226181455 ffigueroa@uandes.cl
Contact: Francisco Espinoza, MD +56226181008 fespinoza@c4c.cl

Locations
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Chile
Clínica Universidad de los Andes Recruiting
Santiago de Chile, Región Metropolitana, Chile, 7591278
Contact: Fernando F E    226181455    ffigueroa@uandes.cl   
Hospital Barros Luco Trudeau Recruiting
Santiago de Chile, Región Metropolitana, Chile
Contact: Jacqueline Pefaur, M.D.    +56998221921    jacquelinepefaur@gmail.com   
Sponsors and Collaborators
Universidad de los Andes, Chile
Investigators
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Principal Investigator: Fernando F E, MD Professor School of Medicine
More Information
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Responsible Party: Fernando E. Figueroa MD, Program Director Translational Research in Cell Therapy, Universidad de los Andes, Chile
ClinicalTrials.gov Identifier: NCT03917797    
Other Study ID Numbers: Phase II Lupus MSC
First Posted: April 17, 2019    Key Record Dates
Last Update Posted: February 8, 2023
Last Verified: February 2023

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Fernando E. Figueroa MD, Universidad de los Andes, Chile:
Systemic Lupus Erythematosus
Mesenchymal Stromal Cells
Lupus nephritis
Additional relevant MeSH terms:
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Glomerulonephritis
Lupus Nephritis
Lupus Erythematosus, Systemic
Connective Tissue Diseases
Autoimmune Diseases
Immune System Diseases
Nephritis
 Kidney Diseases
Urologic Diseases
Female Urogenital Diseases
Female Urogenital Diseases and Pregnancy Complications
Urogenital Diseases
Male Urogenital Diseases