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

Study Purpose

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.

Recruitment Criteria

Accepts Healthy Volunteers Healthy volunteers are participants who do not have a disease or condition, or related conditions or symptoms	No
Study Type An interventional clinical study is where participants are assigned to receive one or more interventions (or no intervention) so that researchers can evaluate the effects of the interventions on biomedical or health-related outcomes. An observational clinical study is where participants identified as belonging to study groups are assessed for biomedical or health outcomes. Searching Both is inclusive of interventional and observational studies.	Interventional
Eligible Ages	18 Years - 75 Years
Gender	All

More Inclusion & Exclusion Criteria

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.

Trial Details

Trial ID: This trial id was obtained from ClinicalTrials.gov, a service of the U.S. National Institutes of Health, providing information on publicly and privately supported clinical studies of human participants with locations in all 50 States and in 196 countries.	NCT06737380
Phase Phase 1: Studies that emphasize safety and how the drug is metabolized and excreted in humans. Phase 2: Studies that gather preliminary data on effectiveness (whether the drug works in people who have a certain disease or condition) and additional safety data. Phase 3: Studies that gather more information about safety and effectiveness by studying different populations and different dosages and by using the drug in combination with other drugs. Phase 4: Studies occurring after FDA has approved a drug for marketing, efficacy, or optimal use.	Phase 1
Lead Sponsor The sponsor is the organization or person who oversees the clinical study and is responsible for analyzing the study data.	LiveKidney.Bio
Principal Investigator The person who is responsible for the scientific and technical direction of the entire clinical study.	N/A
Principal Investigator Affiliation	N/A
Agency Class Category of organization(s) involved as sponsor (and collaborator) supporting the trial.	Industry, Other
Overall Status	Recruiting
Countries	United States
Conditions The disease, disorder, syndrome, illness, or injury that is being studied.	SLE, Lupus, Systemic Lupus Erthematosus, Systemic Lupus Erythematosus, Systemic Lupus Erythematosus (SLE)

Arms & Interventions

Arms

Experimental: UC-MSC therapy

Interventions

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.

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 - 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: - 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: - 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: - 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: - 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.

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: - 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.

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.

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.

Contact a Trial Team

If you are interested in learning more about this trial, find the trial site nearest to your location and contact the site coordinator via email or phone. We also strongly recommend that you consult with your healthcare provider about the trials that may interest you and refer to our terms of service below.

Medical University of South Carolina, Charleston, South Carolina

Status

Recruiting

Address

Medical University of South Carolina

Charleston, South Carolina, 29425

Site Contact

Stephanie Dezzutti

[email protected]

866-859-6107

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