UC-MSC Cell Therapy Study for 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. Adults participants with the diagnosis of systemic lupus erythematosus (SLE) by meeting at least 4 of the11 criteria included in the American College of Rheumatology (ACR) classification and/or the Systemic Lupus International Collaborating Clinics (SLICC) criteria, at the screening visit. 3. Must have a positive ANA (≥1:160 titer) or positive dsDNA antibody test within 6 months of screening. 4. An eGFR of ≥ 30 mL/min/1.73 m2 at screening. 5. At least one SLE background therapy (e.g. immunosuppressant and/or antimalarial) is required for ≥ 12 weeks before the screening visit, must be at a stable dose for ≥ 8 weeks before the screening visit, and must remain stable until randomization and throughout study participation or have taken at least one background therapy and have discontinued due to intolerance. 6. Participants on corticosteroids must be at a stable dose for ≥ 4 weeks before the screening visit; the prednisone-equivalent dose cannot exceed 0.5mg/kg body weight and will be tapered during the study to 10mg or less by week 20 at the discretion of the investigators. 7. SLEDAI-2K ≥6 at the time of screening. 8. Non-pregnant (via negative pregnancy test)/non-breast-feeding women and women with no intention to become pregnant/to breast-feed during the term of the trial, both women and men should commit to use a proper contraceptive. 9. Participant able to provide written informed consent. 10. Must be able 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 3 months preceding informed consent to participate in the study. 2. History of dialysis within 12 months prior to signing the ICF or expected need for renal replacement therapy (dialysis or renal transplant) within a six-month period after enrollment. 3. Concurrent enrollment in another interventional lupus clinical study within the predicted washout time of the investigational product prior to signing ICF. 4. Receipt of any commercially available biologic agent within the washout period described above prior to signing the ICF. 5. Receipt of IV pulse corticosteroid within 6 months prior to signing the ICF. 6. Previous treatment with any type of cellular therapy e.g., Tregs or CAR-T cells. 7. Major surgery within 3 months prior to signing the ICF or major surgery planned during the study period. 8. Has other inflammatory diseases that might confound the evaluations of safety, including but not limited to rheumatoid arthritis (RA), psoriatic arthritis (PsA), RA/lupus overlap, psoriasis, Crohn's disease, or active Lyme disease. 9. Confirmed positive test for active hepatitis B, hepatitis C, HIV or TB. 10. History of cancer, apart from squamous or basal cell carcinoma of the skin and cervical carcinoma in situ. 11. Any other comorbidity which may render the participant unfit for study participation according to the investigator's judgement.

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 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 and at the final 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

Serological assessment for history of HIV, active hepatitis B and C infection and TB will be performed at the Screening visit only (2ml). CMV screen will also be performed but CMV positive subjects will not be excluded. Participants that are CMV IgG antibody positive but not actively infected will receive cells from a CMV antibody + positive donor while CMV antibody negative participants will receive cells from a CMV antibody negative donor.

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 STAT serum b-hCG will be performed in women of child- bearing age, within 7 days prior to study treatment. A negative result must be available prior to study treatment administration. Urine pregnancy tests will also be performed in women of child-bearing age at each study visit. 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

Serum cytokines and chemokines will be profiled at the study visit (Day 1) and at every subsequent follow-up visit. Cytokines (Type 1 IFN: IFN-α, IFN-β, IFN- γ, IFN-ω, IFN-κ, IL 2, 4, 12, 18, 23, 10).

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.

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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