Lupus Erythematosus, SystemicPhase 2MSC-ROLENCT03917797
Mesenchymal Stromal Cells (MSC´s) in Renal Lupus
A Phase 2 study testing MSC treatment in people with Lupus Erythematosus, Systemic and Lupus Glomerulonephritis, run by Universidad de los Andes, Chile at 2 sites in Chile.
Trial 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). Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Lupus Erythematosus, Systemic, Lupus Glomerulonephritis
- What is being tested?
- MSC treatment
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 39 participants
- Where
- 2 sitesChile
- Study dates
- 2 Apr 2019to Dec 2025
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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.
Trial locations and contacts
2 sites in 1 country.
Chile2 sites, 2 recruiting
- Recruiting
Clínica Universidad de los Andes
Santiago, Santiago Metropolitan, 7591278
Fernando F E, 226181455, ffigueroa@uandes.cl
- Recruiting
Hospital Barros Luco Trudeau
Santiago, Santiago Metropolitan
Jacqueline Pefaur, M.D., +56998221921, jacquelinepefaur@gmail.com
About this study
Official title: Dose-response and Efficacy of Umbilical Cord-derived Mesenchymal Stromal Cells in Renal Systemic Lupus Erythematosus
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).
Read the 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,
- Being tested
- MSC treatment, Standard of Care, Placebo
- Keywords
- Systemic Lupus Erythematosus, Mesenchymal Stromal Cells, Lupus nephritis
- Medical subject headings
- Lupus Erythematosus, Systemic, Lupus Nephritis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MSC treatmentBiological
Also called Cellistem ® Lupus
Umbilical cord-derived Mesenchymal Stromal Cell
Standard of CareDrug
Also called Standard of Care for Lupus Nephritis
Methylprednisolone; Cyclophosphamide; Prednisone; Mycophenolate
PlaceboDrug
Also called Placebo (for MSC)
MSC infusion vehicle
| Study group | Role | What they receive |
|---|---|---|
| MSC treatment | Experimental | 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. Receives: Biological: MSC treatment; Drug: Standard of Care |
| Placebo | Placebo comparator | 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. Receives: Drug: Standard of Care; Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Achievement of Global Renal Response (GR) at Study Endpoint
Proportion of Patients that achieve Complete (CR) or Partial (PR) Renal Response at Endpoint
Measured: 12 months
Secondary outcomes (8)
Achievement of Complete Renal Response (CR) at Study Endpoint
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.
Measured: 12 months
Achievement of Partial Renal Response (PR) at Study Endpoint
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.
Measured: 12 months
Treatment Failure
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.
Measured: 24 weeks and 12 months
Response of SLE Responder Index (SRI).
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.
Measured: 12 months
Selena Sledai
Average change in Selena Sledai Score in patients and controls
Measured: 12 months
BILAG score
Average hange in BILAG score in patients and controls
Measured: 12 months
Disease Flares
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
Measured: 12 months
Biomarker Response
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.
Measured: 24 weeks and 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Apr 2019
- Study start2 Apr 2019
- Primary completionDec 2024 (estimated)
- Study completionDec 2025 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Lupus Erythematosus, SystemicStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- Superiority trial comparing MSCs versus Placebo in SLE patients with severe renal disease receiving Standard of Care treatment.
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 39 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Universidad de los Andes, Chile
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Fernando E. Figueroa MD, Universidad de los Andes, Chile
- Investigators
- Fernando F E, MD, Principal investigator, Professor School of Medicine
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03917797
- Sponsor's protocol ID
- Phase II Lupus MSC
- Acronym
- MSC-ROLE
- Status last verified
- Feb 2023
- First submitted
- 2 Apr 2019
- Last updated
- 8 Feb 2023
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 8 Feb 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Dose-response and Efficacy of Umbilical Cord-derived Mesenchymal Stromal Cells in Renal Systemic Lupus Erythematosus
- ClinicalTrials.gov ID
- NCT03917797
- Acronym / sponsor ID
- MSC-ROLE / Phase II Lupus MSC
- Keywords
- Systemic Lupus Erythematosus, Mesenchymal Stromal Cells, Lupus nephritis
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 39 participants (estimated)
- Lead sponsor
- Universidad de los Andes, ChileUniversity, hospital or other
- Study start
- 2 Apr 2019 (actual)
- Primary completion
- Dec 2024 (estimated)
- Study completion
- Dec 2025 (estimated)
- First posted
- 17 Apr 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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).
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
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
- Model description
- Superiority trial comparing MSCs versus Placebo in SLE patients with severe renal disease receiving Standard of Care treatment.
| Arm | Type | Receives |
|---|---|---|
| MSC treatment | Experimental | Biological: MSC treatment, Drug: Standard of CareIntervention: 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. |
| Placebo | Placebo comparator | Drug: Standard of Care, Drug: PlaceboIntervention: 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. |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- 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).
Key exclusion criteria (5 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| MSC treatmentAlso: Cellistem ® LupusUmbilical cord-derived Mesenchymal Stromal Cell | Biological | MSC treatment |
| Standard of CareAlso: Standard of Care for Lupus NephritisMethylprednisolone; Cyclophosphamide; Prednisone; Mycophenolate | Drug | MSC treatment, Placebo |
| PlaceboAlso: Placebo (for MSC)MSC infusion vehicle | Drug | Placebo |
Primary outcome
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
8 secondary outcomes
Achievement of Complete Renal Response (CR) at Study Endpoint
Time frame: 12 months
Achievement of Partial Renal Response (PR) at Study Endpoint
Time frame: 12 months
Treatment Failure
Time frame: 24 weeks and 12 months
Response of SLE Responder Index (SRI).
Time frame: 12 months
Selena Sledai
Time frame: 12 months
BILAG score
Time frame: 12 months
Disease Flares
Time frame: 12 months
Biomarker Response
Time frame: 24 weeks and 12 months
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Feb 2023.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Fernando F E, MD principal investigator
Professor School of Medicine
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing MSC treatment in people with Lupus Erythematosus, Systemic and Lupus Glomerulonephritis, run by Universidad de los Andes, Chile at 2 sites in Chile.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 8 Feb 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in Chile. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Fernando F E, MD (+56226181455, ffigueroa@uandes.cl), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Achievement of Global Renal Response (GR) at Study Endpoint (time frame: 12 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 8 Feb 2023.