UC-MSC Cell Therapy Study for Systemic Lupus Erythematosus (SLE) Patients

A Phase I, Open-Label Study to Evaluate the Safety and Tolerability of Subcutaneous Administration of Umbilical Cord Derived - Mesenchymal Stromal Cell Therapy in Addition to Standard of Care as A Treatment For Active Systemic Lupus Erythematosus

The goal of this clinical trial is to evaluate the safety and effectiveness of UC-MSCs in adults with systemic lupus erythematosus (SLE).

The main questions this study aims to answer are:

1. Can UC-MSCs improve kidney function and reduce SLE disease activity?
2. Are UC-MSCs safe and well-tolerated in this patient population?

Participants in this study will:

• Receive UC-MSCs in a single dose in addition to standard of care treatment.
• Provide blood and urine samples for laboratory assessments, including biomarkers and immune profiling (e.g., cytokines, complement proteins, and autoantibodies).
• Attend regular clinic visits for physical exams, disease activity scoring, and imaging tests to monitor kidney health.
• Complete assessments for safety, such as monitoring for adverse events and changes in laboratory values.

This study aims to provide new insights into treatment options for SLE and lupus nephritis, addressing an unmet medical need in this population.

Study Overview

• Status: Recruiting
• Conditions: Systemic Lupus Erythematosus; SLE; Systemic Lupus Erythematosus (SLE); Lupus; Systemic Lupus Erthematosus
• Intervention / Treatment: Other: SF-36 questionnaire; Diagnostic test: Clinical laboratory evaluations - Hematology; Diagnostic test: Urinalysis; Diagnostic test: SLE activity evaluation; Biological: single dose of UC-MSCs; Diagnostic test: Clinical laboratory evaluations - Biochemistry; Diagnostic test: SLE biomarker profiling; Diagnostic test: Physical examination; Diagnostic test: Electrocardiogram; Diagnostic test: Clinical laboratory evaluations - Serology; Diagnostic test: Pregnancy Test; Diagnostic test: Cytokines and chemokine profiling; Other: PROMIS instruments

• Study Type: Interventional
• Enrollment (Estimated): 10
• Phase: Phase 1

Contacts and Locations

• Study Contact: Name: Nadya Lisovoder, MD; Phone Number: +972524753435; Email: nadyal@galilee-cbr.com

Study Locations

• United States
  • South Carolina
    • Charleston, South Carolina, United States, 29425
      • Recruiting
      • Medical University of South Carolina
      • Principal Investigator: Kathleen Maksimowicz McKinnon, MD
      • Contact: Stephanie Dezzutti; Phone Number: 866-859-6107; Email: lupusresearch@musc.edu

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria

1. Age 18-75 years at the time of screening
2. Diagnosis of systemic lupus erythematosus (SLE), meeting at least 4 of the 11 criteria included in the American College of Rheumatology (ACR) Classification Criteria and/or 4 of the 17 criteria (with at least one of those being clinical and at least one being immunologic) included in the Systemic Lupus International Collaborating Clinics (SLICC) Criteria, at the screening visit.
3. Must have a positive ANA (≥1:160 titer) or positive anti-dsDNA antibody test within 6 months of the screening visit
4. An eGFR of ≥ 30 mL/min/1.73 m2 in the screening period
5. Prior SLE background therapy with at least one non-biologic medication (e.g. immunosuppressant and/or antimalarial), not including corticosteroids, is required for ≥ 12 weeks before the screening visit.
6. SLEDAI-2K ≥6 at the time of screening
7. Participant able and willing to provide written informed consent
8. Must be able and willing to adhere to the study visit schedule and other protocol requirements.

Exclusion Criteria

1. History of any non-systemic lupus erythematosus (non-SLE) disease that required treatment with oral or parenteral corticosteroids for more than a total of 2 weeks within the 12 weeks preceding the screening visit.
2. History of dialysis within 12 months prior to the screening visit or expected need for renal replacement therapy (dialysis or renal transplant) within a six-month period after enrollment.
3. Use of prednisone >0.5 mg/kg/day (or equivalent corticosteroid) in the 4 weeks prior to the screening visit.
4. Any change or addition to a non-biologic immunosuppressant and/ or antimalarial regimen (not including corticosteroids) ≤ 12 weeks prior to the Screening visit.
5. Treatment with an interventional agent within the washout time of 90 days or 5 half-lives prior to Baseline (Day 0), whichever is longer.
6. Receipt of any commercially available biologic agent within the washout period described above prior to Baseline (Day 0).
7. Receipt of prior MSC therapy within the washout time of 52 weeks prior to Baseline (Day 0).
8. Previous treatment with any type of cellular therapy e.g., Tregs or CAR-T cells, with the exception of previous MSCs.
9. Major surgery within 90 days prior to Baseline (Day 0) or major surgery planned during the study period
10. Confirmed positive test for active hepatitis B, hepatitis C, human immunodeficiency virus (HIV) or tuberculosis (TB).
11. Any active infection that has not been adequately treated or completely resolved prior to Baseline (Day 0).
12. History of cancer, apart from adequately treated squamous or basal cell carcinoma of the skin, or cervical carcinoma in situ
13. Pregnant or breast-feeding women and women with intention to become pregnant/to breast-feed during the duration of the trial.
14. Women and men who do not agree to use a medically acceptable form of contraception for the duration of the trial.
15. Any other comorbidity which may render the participant unfit for study participation according to the investigator's judgement.
16. Any other medical condition, which in the opinion of the investigator, may impact the quality or interpretation of the data obtained from the study.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm

Intervention / Treatment

Experimental: UC-MSC therapy

Other: SF-36 questionnaire; Patients will fill out the SF 36 survey, assessing physical and mental health outcomes prior to dosing and/or any other clinical assessments, on visits 1 (baseline), visit 5 (day 14), visit 6 (day 28), visit 11 (day 168) and early termination visit.; Diagnostic test: Clinical laboratory evaluations - Hematology; The complete blood count (CBC) will include: hemoglobin, hematocrit, white blood cells (WBCs) with complete manual or automated differential (total neutrophils, lymphocytes, monocytes, eosinophils, basophils; absolute or percentage will be acceptable), red blood cells (RBCs), platelet count. For CBC, 1mL blood will be collected at each study visit.; Diagnostic test: Urinalysis; General urinalysis will include analysis of pH, specific gravity, protein, glucose, ketones, bilirubin, urobilinogen, leukocytes, erythrocytes. In addition samples will be assessed for presence of urinary casts. Spot urine protein/creatinine measure will be done at each visit. If no casts are observed at Screening, then machine assessment of further urinary samples is allowed. For urinalysis, 10mL urine will be collected at each study visit.; Diagnostic test: SLE activity evaluation; SLE status will be assessed using the Safety of Estrogens in Lupus Erythematosus National Assessment- SLE Disease Activity Index (SLEDAI 2K scale).SLE activity will also be assessed using British Isles Lupus Assessment Group (BILAG), physician global and participant global assessment allowing use of the Systemic Lupus Responder Index (SRI 4) and the BILAG-based Composite Lupus Assessment (BICLA) instruments to assess clinical response.; Biological: single dose of UC-MSCs; participants will be treated on Baseline Day 1 with a UC-MSCs product administered subcutaneously. Participants will be observed for 2 to 3 hours after end of study treatment and then followed up for safety assessments over the subsequent 6-month period.; Diagnostic test: Clinical laboratory evaluations - Biochemistry; The panel will include analysis of albumin, blood urea nitrogen (BUN), creatinine, glucose, uric acid, calcium, phosphorus, potassium, sodium, lactate dehydrogenase (LDH), total protein, magnesium, alkaline phosphatase, alanine aminotransferase (ALT), aspartate aminotransferase (AST), total bilirubin and hs-CRP. For biochemistry, 3 mL blood will be collected at each study visit. Results relating to inclusion criteria must be confirmed within 7 days prior to Day 1.; Diagnostic test: SLE biomarker profiling; SLE biomarkers will be profiled at the study visit (Day 1) and at every subsequent follow up visit. For this purpose, urine and 10 ml blood will be collected. Anti-ENA, Anti-dsDNA antibodies, total serum IgG levels, CBC, C3, C4, anti-nucleosome antibodies, erythrocyte sedimentation rate, CRP, protein/creatinine ratio in spot urine, 24-hour urine protein - Day 1 - Baseline and at end of study visit only; Mononuclear cell changes. This analysis includes lymphocyte subsets and innate immune cell subsets; Diagnostic test: Physical examination; The physical examination will be performed at the Screening visit and at each subsequent study and safety in clinic follow-up visit. The exam will include: measurement of vital signs (heart rate, peripheral arterial blood pressure, respiratory rate, and temperature), weight, height (at Screening visit only), cardiovascular and respiratory systems, abdominal examination, skin evaluation, mouth and eye evaluation, and neurological assessment. Abnormal findings will be recorded in the CRF.; Diagnostic test: Electrocardiogram; A twelve-lead electrocardiogram (ECG), including corrected QT interval (QTc interval), will be performed in all participants at the Screening visit.; Diagnostic test: Clinical laboratory evaluations - Serology; Serologic testing for HIV, HBV, HCV, TB, and CMV will be performed; HBV and HCV PCR will be done only if serology is positive. Autoimmune tests (ANA, anti-ENA, total IgG, anti-nucleosome) will be done at Day 0 and end of study; ANA only at Screening if no positive ANA or anti-dsDNA in prior 6 months. Inflammatory markers (hs-CRP, ESR) will be tested at Day 0 and end of study. CBC with differential, biochemistry, anti-dsDNA, and complement C3/C4 will be assessed at each scheduled visit.; Diagnostic test: Pregnancy Test; A serum b-hCG pregnancy test will be performed for women of child-bearing potential at the Screening visit;urine pregnancy tests will be performed for women of child-bearing potential at each study visit noted above, including on Day 0 prior to study treatment. Pregnancy tests are not required for women unable to become pregnant for one of the following reasons: Menopause confirmed by healthcare provider, verbally reported by a participant that she has had her uterus or both ovaries or both fallopian tubes removed.; Diagnostic test: Cytokines and chemokine profiling; Samples will be analyzed for the following cytokines/chemokines: IFNγ, IL-6, IL-10, IL-18, TNFα, TGFβ1, CXCL10, CCL2, N-GAL, urine endothelin, KIM1. Serum and urine samples will be collected on Days 0, 28, 56, 140, 168, and early termination visit.; Other: PROMIS instruments; PROMIS Fatigue, PROMIS, Depression, PROMIS Pain prior to dosing and/or any other clinical assessments, on visits 1 (baseline, day 0), visit 5 (day 14), visit 6 (day 28), visit 11 (day 168) and early termination visit.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Incidence of Treatment-Emergent Adverse Events	Analysis for the primary endpoint will be descriptive in nature. safety and tolerability will be measured by the number of AEs and SAEs observed. Adverse event incidents will be summarized descriptively. The severity grade of each recorded AE, and their relationship to study treatment will be analyzed. The action taken and outcome will also be analyzed accordingly.	day 1 to day 168

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change from baseline in the SLEDAI 2K following administration	Total score ranging from 0 to 105. Interpretation: Scores are categorized to indicate disease activity: 0: No activity 1-5: Mild activity 6-10: Moderate activity 11-19: High activity ≥20: Very high activity Improvement is defined as a decrease in score.	day 1 to day 168
Change from baseline in the BILAG-2004 Index score following administration	Organ involvement is categorized into grades: A: Severe active disease B: Moderate active disease C: Stable disease D: Resolved disease E: No involvement Improvement is defined as an increase in score.	day 1 to day 168
Change from baseline in prednisone-equivalent corticosteroid doses at each visit performed.	Reduction in prednisone-equivalent corticosteroid dose from Day 1 to Day 168.	day 1 to day 168.
Change from baseline in PROMIS(Patient-Reported Outcomes Measurement Information System) score	Change from baseline in scores for quality of life, fatigue, pain, and depression as reported by the patient. Scores range from 0 to 100. Higher scores indicate worse symptoms.	day 1 to day 168
Efficacy by SRI-4 Responder assessment at Day 168.	Change from baseline in SRI-4, defined as a ≥4-point reduction in SLEDAI, with no significant worsening in BILAG or PGA. A higher SRI-4 response rate reflects greater clinical improvement.	day 1 to day 168

Other Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change of pre-treatment versus post-treatment levels of Anti-ENA Antibodies	Improvement is defined as a decrease in anti-ENA antibody levels from pre-treatment to post-treatment measurements.	day 1 to day 168.
Change of pre-treatment versus post-treatment levels of Anti-dsDNA Antibodies	Improvement is defined as a reduction in antibody levels.	day 1 to day 168
Change of pre-treatment versus post-treatment levels of Total Serum IgG Levels	Improvement is defined as a reduction in serum IgG levels.	day 1 to day 168
Change of pre-treatment versus post-treatment results of CBC	Improvement is defined as any change in values compared to normal range.	day 1 to day 168
Change of pre-treatment versus post-treatment levels of Anti-nucleosome antibodies	Improvement is defined as a reduction in Anti-nucleosome antibodies levels.	day 1 to day 168
Change of pre-treatment versus post-treatment levels of Erythrocyte sedimentation rate	Improvement is defined as a reduction in Erythrocyte sedimentation rate.	day 1 to day 168
Change of pre-treatment versus post-treatment levels of CRP	Improvement is defined as a reduction in CRP levels.	day 1 to day 168
Change of pre-treatment versus post-treatment levels of Cytokines and Chemokines	Improvement is defined as a reduction in cytokine and chemokine levels.	day 1 to day 168
Change of pre-treatment versus post-treatment Proteinuria measured by protein/creatinine ratio in spot urine	Improvement is defined as a reduction in protein/creatinine ratio in spot urine	day 1 to day 168
Change of pre-treatment versus post-treatment 24-hour urine protein measured by protein/creatinine ratio in 24-hour urine collection	Improvement is defined as a reduction in Protein/Creatinine ratio in in 24-hour urine collection	day 1 to day 168
Change of pre-treatment versus post-treatment levels of C3 ; C4 levels	Improvement is defined as an increase in C3 and/or C4 levels from pre-treatment to post-treatment measurements.	day 1 to day 168

Collaborators and Investigators

• Sponsor: LiveKidney.Bio
• Collaborators: Medical University of South Carolina; Galilee CBR

Study record dates

Study Major Dates

• Study Start (Actual): January 7, 2025
• Primary Completion (Estimated): June 1, 2026
• Study Completion (Estimated): July 1, 2026

Study Registration Dates

• First Submitted: December 11, 2024
• First Submitted That Met QC Criteria: December 15, 2024
• First Posted (Actual): December 17, 2024

Study Record Updates

• Last Update Posted (Estimated): September 11, 2025
• Last Update Submitted That Met QC Criteria: September 4, 2025
• Last Verified: September 1, 2025

More Information

Terms related to this study

• Keywords: Lupus; systemic lupus erythematosus; SLE
• Additional Relevant MeSH Terms: Connective Tissue Diseases; Autoimmune Diseases; Immune System Diseases; Skin and Connective Tissue Diseases; Lupus Erythematosus, Systemic; Investigative Techniques; Clinical Laboratory Techniques; Diagnostic Techniques and Procedures; Diagnosis; Diagnostic Techniques, Cardiovascular; Heart Function Tests; Diagnostic Techniques, Urological; Electrodiagnosis; Clinical Chemistry Tests; Diagnostic Techniques, Obstetrical and Gynecological; Electrocardiography; Physical Examination; Urinalysis; Pregnancy Tests
• Other Study ID Numbers: LK-23-01

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: UNDECIDED

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No