Mesenchymal Stem Cells for Lupus

Not currently recruiting at 8 trial locations
GG
Overseen ByGary Gilkeson
Age: 18+
Sex: Any
Trial Phase: Phase 2
Sponsor: Medical University of South Carolina
Must be taking: Mycophenolate, Cyclophosphamide, Azathioprine
Prior Safety DataThis treatment has passed at least one previous human trial

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests whether umbilical cord-derived mesenchymal stem cells (MSCs) can safely and effectively treat adults with systemic lupus erythematosus (SLE), a disease where the immune system attacks the body's own tissues. Participants will receive either a high or low dose of MSCs or a placebo, alongside their usual care, to determine if the stem cells improve their condition. Suitable candidates have active lupus symptoms and continue to struggle with the disease despite standard treatment. As a Phase 2 trial, the research focuses on measuring the treatment's effectiveness in an initial, smaller group of people.

Do I have to stop taking my current medications to join the trial?

The trial protocol does not specify if you must stop all current medications. However, you cannot change or add to your immunosuppressant regimen within 3 months of the Baseline visit, and you must not have used prednisone >0.5 mg/kg/day within 1 month of the Baseline visit. Also, you should not have received certain biologic therapies like belimumab within 2 months or rituximab within 6 months of the Baseline visit.

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

The trial does not specify if you need to stop all current medications, but it does mention that you should not have changes or additions to your immunosuppressant regimen within 3 months of the start, except for corticosteroids. Also, you should not have used certain medications like belimumab or rituximab within specific time frames before the trial.

Is there any evidence suggesting that this trial's treatments are likely to be safe?

Research has shown that stem cells from umbilical cords are generally safe for treating conditions like systemic lupus erythematosus (SLE). Studies have found that both high and low doses of these stem cells are usually well-tolerated by patients. In one study, participants who received these stem cells reported few side effects, indicating the treatment is relatively safe.

These positive safety results come from several studies on stem cell therapy for SLE. These studies have found minimal side effects, making the treatment promising for those considering joining a trial. While outcomes cannot be guaranteed, past evidence suggests that these stem cells are a safe option for many patients with SLE.12345

Why are researchers excited about this trial's treatments?

Unlike the standard treatments for lupus, which often include immunosuppressive drugs like corticosteroids and antimalarials, Mesenchymal Stem Cells (MSCs) offer a novel approach by potentially modulating the immune system naturally. High-dose MSCs involve administering 5 million cells per kilogram, while the low-dose version uses 1 million cells per kilogram, both in a Plasma-Lyte A solution. Researchers are particularly excited because MSCs might help repair damaged tissues and reduce inflammation without the side effects common to traditional drugs. This could represent a major shift in lupus treatment, providing a more targeted and less toxic option.

What evidence suggests that mesenchymal stem cells might be an effective treatment for lupus?

Research has shown that stem cells from umbilical cords might help treat systemic lupus erythematosus (SLE). In this trial, participants will receive either a high dose or a low dose of these stem cells, or a placebo infusion. Studies have found that a high dose of these stem cells can lead to remission, reducing disease activity and improving lupus symptoms by modulating the immune system. This includes calming harmful immune responses and enhancing organ function. Even low doses of stem cells have demonstrated benefits, helping to control the disease and reduce symptoms. Both high and low doses can prevent the body from mistakenly attacking itself, a hallmark of autoimmune diseases like SLE. Overall, stem cell therapy is emerging as a promising option for SLE patients, especially for those unresponsive to other treatments.12367

Who Is on the Research Team?

GS

Gary S. Gilkeson, MD

Principal Investigator

Medical University of South Carolina

DL

Diane L. Kamen, MD, MSCR

Principal Investigator

Medical University of South Carolina

Are You a Good Fit for This Trial?

Adults aged 18-65 with active Systemic Lupus Erythematosus (SLE), evidenced by specific antibody tests and a minimum SLEDAI score, can join. They must have been on standard therapy for lupus nephritis if present. Exclusions include severe kidney issues, certain infections like HIV or TB, recent cancer (except some skin cancers/cervical carcinoma in situ), pregnancy/breastfeeding without contraception use, major organ transplants, unstable medical conditions, high steroid use recently or changes in immunosuppressants.

Inclusion Criteria

Historical presence of at least 4 of 11 of the ACR Classification Criteria
My lupus is active despite treatment, as shown by specific lupus activity scores.
I have completed 6 months of treatment for kidney inflammation.
See 2 more

Exclusion Criteria

I have lupus affecting my kidneys and need dialysis.
I haven't had cancer in the last 5 years, except for certain skin cancers or cervical pre-cancer.
Current substance abuse or recent (within one year) history of substance abuse
See 13 more

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive either a low or high dose of mesenchymal stem cells or placebo through a single IV infusion, along with standard of care

24 weeks
1 visit (in-person) for infusion, regular follow-up visits

Follow-up

Participants are monitored for safety and effectiveness after treatment, with assessments at Weeks 12, 24, and 52

28 weeks
Regular follow-up visits at Weeks 12, 24, and 52

What Are the Treatments Tested in This Trial?

Interventions

  • High Dose Mesenchymal Stem Cells (MSCs)
  • Low Dose Mesenchymal Stem Cells (MSCs)
  • Placebo Infusion
Trial Overview The trial is testing the effectiveness of two different doses of Mesenchymal Stem Cells from umbilical cords against a placebo. All participants will continue their standard lupus treatments. The main goal is to see if those who get the stem cells show more improvement than those who get the placebo.
How Is the Trial Designed?
3Treatment groups
Experimental Treatment
Placebo Group
Group I: Low Dose Mesenchymal Stem Cells (MSCs)Experimental Treatment1 Intervention
Group II: High Dose Mesenchymal Stem Cells (MSCs)Experimental Treatment1 Intervention
Group III: Plasma Lyte A SolutionPlacebo Group1 Intervention

High Dose Mesenchymal Stem Cells (MSCs) is already approved in China, United States for the following indications:

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Approved in China as Mesenchymal Stem Cells for:
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Approved in United States as Umbilical Cord-Derived Mesenchymal Stem Cells for:

Find a Clinic Near You

Who Is Running the Clinical Trial?

Medical University of South Carolina

Lead Sponsor

Trials
994
Recruited
7,408,000+

Published Research Related to This Trial

In a study involving 30 patients with systemic lupus erythematosus (SLE) who received umbilical cord-derived mesenchymal stem cells (UC MSCs), the treatment led to an increase in regulatory T cells (Treg) and a decrease in T helper 17 (Th17) cells over a 12-month period, indicating a potential immunoregulatory effect.
The mechanism of action for UC MSCs involves the regulation of cytokines such as TGF-β and prostaglandin E2 (PGE2), which were found to be crucial for the observed changes in Treg and Th17 cell populations, suggesting a targeted approach for managing SLE.
The regulation of the Treg/Th17 balance by mesenchymal stem cells in human systemic lupus erythematosus.Wang, D., Huang, S., Yuan, X., et al.[2022]
In a study involving 16 patients with severe systemic lupus erythematosus (SLE) who did not respond to standard treatments, umbilical cord-derived mesenchymal stem cell transplantation (UC MSCT) led to significant improvements in disease activity and serological markers over a median follow-up of 8.25 months.
The treatment was safe, with no treatment-related deaths or recurrences of disease, and was associated with an increase in regulatory T cells and a balanced cytokine profile, suggesting a potential mechanism for its therapeutic effects.
Umbilical cord mesenchymal stem cell transplantation in severe and refractory systemic lupus erythematosus.Sun, L., Wang, D., Liang, J., et al.[2022]
In a long-term study involving 9 patients with refractory systemic lupus erythematosus (SLE), allogeneic umbilical cord mesenchymal stem cells (UC MSCs) showed a good safety profile, with only one patient experiencing mild dizziness after infusion, which quickly resolved.
After 6 years of follow-up, there were no significant adverse events or changes in blood counts, liver function, or tumor marker levels, indicating that UC MSC transplantation is safe for SLE patients who do not respond to conventional treatments.
Long-term safety of umbilical cord mesenchymal stem cells transplantation for systemic lupus erythematosus: a 6-year follow-up study.Wang, D., Niu, L., Feng, X., et al.[2022]

Citations

Umbilical cord mesenchymal stem cell transplantation in ...UC MSC treatment resulted in clinical disease remission and systemic amelioration in lupus patients who are refractory to other. However, some patients had ...
Human umbilical cord-derived mesenchymal stem cell ...We showed that hUC-MSCs transplantation ameliorated lupus symptoms and increased senescence of splenic CD4+ T cells through Sirt1/p53 signaling via miR-199a-5p ...
Treatment of Systemic Lupus Erythematosus With Human ...This study will investigate the safety and efficacy of allogeneic umbilical cord-derived stem cell therapy in treating patients with moderate to severe systemic ...
Allogeneic umbilical cord-derived mesenchymal stromal ...The immunomodulatory, proangiogenic, and antifibrotic properties of mesenchymal stromal cells support their use in treating patients with SLE.
Advances in mesenchymal stem cell therapy for lupus ...MSCs can suppress autoimmunity, alleviate proteinuria and restore renal function by modulating the functions of various immune cells and ...
120 Mesenchymal Stem/Stromal Cells ALLOGENEIC ...ALLOGENEIC UMBILICAL-CORD DERIVED MESENCHYMAL STROMAL CELLS MSC(UC) AS TREATMENT FOR SYSTEMIC LUPUS ERYTHEMATOSUS (SLE): SAFETY AND EARLY CLINICAL/BIOLOGICAL ...
CT-04 Safety and efficacy of allogeneic umbilical cord ...Background Mesenchymal stem cells (MSCs) are known to possess significant immunosuppressive properties, and their use in refractory SLE is ...
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