UC-MSCs for Lupus

Overseen By Nadya Lisovoder, MD

Age: 18+

Sex: Any

Trial Phase: Phase 1

Sponsor: LiveKidney.Bio

Must be taking: Immunosuppressants, Antimalarials

Must not be taking: Biologics, Cellular therapy

Disqualifiers: Dialysis, Cancer, Hepatitis, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: Unknown

Duration: 24 weeks

10 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial aims to determine if UC-MSCs (umbilical cord-derived mesenchymal stem cells) can benefit individuals with systemic lupus erythematosus (SLE) by improving kidney function and overall disease symptoms. It also evaluates the treatment's safety and tolerability. Participants will receive a dose of UC-MSCs alongside their usual care. Those diagnosed with SLE, who have a positive ANA or anti-dsDNA test, and have been on specific lupus medications for at least 12 weeks may be suitable candidates. As a Phase 1 trial, the research focuses on understanding the treatment's effects in people, offering participants the opportunity to be among the first to receive this new treatment.

Do I need to stop my current medications to join the trial?

The trial does not specify that you need to stop your current medications. In fact, you must have been on at least one non-biologic medication for lupus for at least 12 weeks before joining. However, you cannot have changed or added any non-biologic immunosuppressant or antimalarial medications in the 12 weeks before the screening.

Is there any evidence suggesting that UC-MSCs are likely to be safe for humans?

Research has shown that stem cells from umbilical cords, known as UC-MSCs, are generally safe for humans. Studies have found that people with lupus tolerate UC-MSCs well. In one study, patients received UC-MSC infusions without experiencing serious side effects. Another study confirmed the treatment's safety and tolerability.

These findings suggest that UC-MSCs are a safe option for people with lupus, with a low risk of severe side effects. However, as this is a Phase 1 trial, the primary goal is to assess the safety of UC-MSCs. While early results are promising, further research is needed to confirm these findings in larger groups. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Unlike the standard treatments for lupus, which primarily involve immunosuppressants and anti-inflammatory drugs, UC-MSC therapy uses umbilical cord-derived mesenchymal stem cells to potentially modulate the immune system more precisely. Researchers are excited about this treatment because it offers a novel mechanism of action, aiming to repair and regenerate tissues while reducing harmful inflammation. This approach could provide a more targeted and less toxic alternative to current therapies, potentially improving long-term outcomes for patients with lupus.

What evidence suggests that UC-MSCs might be an effective treatment for lupus?

Research has shown that stem cells from umbilical cords, known as UC-MSCs, might help manage lupus, a disease where the immune system attacks the body. Studies have found that UC-MSCs can lower disease activity, stabilize harmful proteins in the blood, and improve symptoms in patients. Reports indicate that UC-MSCs are safe, with no major side effects observed in patients who received them. These cells can calm the immune system and help repair tissue, which may be beneficial for treating lupus. Early results are promising, but more research is needed to confirm these findings. Participants in this trial will receive UC-MSC therapy to further evaluate its effectiveness and safety in treating lupus. ^{4 6 7 8 9}

Are You a Good Fit for This Trial?

This trial is for adults with systemic lupus erythematosus (SLE), specifically those who may have kidney involvement. Participants must be willing to receive a single dose of UC-MSCs, provide blood and urine samples, and undergo regular health monitoring. Details on specific inclusion or exclusion criteria are not provided.

Inclusion Criteria

Participant able to provide written informed consent

Must be able to adhere to the study visit schedule and other protocol requirements

I am not pregnant or breastfeeding and do not plan to during the trial. I will use contraception.

Exclusion Criteria

Any other comorbidity which may render the participant unfit for study participation according to the investigator judgment

Confirmed positive test for active hepatitis B, hepatitis C, HIV or TB

I have used steroids for more than 2 weeks in the last 3 months for a condition other than SLE.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive a single dose of UC-MSCs in addition to standard of care treatment

24 weeks

Regular clinic visits for physical exams, disease activity scoring, and imaging tests

Follow-up

Participants are monitored for safety and effectiveness after treatment, including assessments for adverse events and changes in laboratory values

24 weeks

Regular clinic visits

What Are the Treatments Tested in This Trial?

Interventions

• UC-MSCs

Trial Overview The study tests the safety and effectiveness of a single dose of Umbilical Cord-derived Mesenchymal Stem Cells (UC-MSCs) in SLE patients. It includes regular clinic visits, lab assessments for biomarkers and immune profiling, physical exams, disease activity scoring, and imaging tests to monitor kidney health.

How Is the Trial Designed?

Group I: UC-MSC therapy Experimental Treatment 13 Interventions

Who Is Running the Clinical Trial?

LiveKidney.Bio

Lead Sponsor

Trials

1

Recruited

10+

Medical University of South Carolina

Collaborator

Trials

994

Recruited

7,408,000+

Galilee CBR

Industry Sponsor

Trials

4

Recruited

570+

Citations

1. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC4060570/

Umbilical cord mesenchymal stem cell transplantation in ... The overall survival rate was 92.5% (37 of 40 patients). UC-MSCT was well tolerated, and no transplantation-related adverse events were observed. Thirteen and ...

2. sciencedirect.com sciencedirect.com/science/article/pii/S1465324924001646

120 Mesenchymal Stem/Stromal Cells ALLOGENEIC ... A single infusion of MSC(UC) was safe in 8 severe SLE pts. This proof of concept study showed clinical improvement. Placebo-controlled trials are needed to ...

3. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/20506343/

Umbilical cord mesenchymal stem cell transplantation in ... Conclusion: Our findings indicate that UC MSCT results in amelioration of disease activity, serologic changes, and stabilization of proinflammatory cytokines.

4. thelancet.com thelancet.com/journals/lanrhe/article/PIIS2665-9913(24)00298-4/abstract

Allogeneic umbilical cord-derived mesenchymal stromal ... The immunomodulatory, proangiogenic, and antifibrotic properties of mesenchymal stromal cells support their use in treating patients with SLE.

5. stemcellres.biomedcentral.com stemcellres.biomedcentral.com/articles/10.1186/s13287-021-02586-1

Transplantation of mesenchymal stem cells ameliorates ... Our previous studies have found that UC-MSCs transplantation could alleviate experimental SLE and SLE patients and could modulate Treg/Th1/Th17 ...

6. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC9277402/

Safety, immunological effects and clinical response in a ... This phase 1 trial suggests that umbilical cord (UC) MSC infusions are very safe and may have efficacy in lupus. The B cell and GARP-TGFβ changes provide novel ...

7. clinicaltrials.gov clinicaltrials.gov/study/NCT03171194

Pilot Trial of Mesenchymal Stem Cells for Systemic Lupus ... The purpose of this study is to evaluate the safety of mesenchymal stromal cells (MSCs) obtained from umbilical cords for the treatment of adults with active ...

8. sciencedirect.com sciencedirect.com/science/article/pii/S2213671118300559

A Long-Term Follow-Up Study of Allogeneic Mesenchymal ... We report a long-term follow-up study of allogeneic bone marrow and/or umbilical cord MSC transplantation (MSCT) in severe and drug-refractory systemic lupus ...

9. arthritis-research.biomedcentral.com arthritis-research.biomedcentral.com/articles/10.1186/ar4520

Umbilical cord mesenchymal stem cell transplantation in ... In our present single-center pilot study, umbilical cord (UC)–derived mesenchymal stem cells (MSCs) had a good safety profile and therapeutic ...