Umbilical-cord stem cell treatment for adult lupus

Phase I/II Clinical Study on the Safety, Efficacy, Pharmacodynamics and Immunogenicity of Human Umbilical Cord Mesenchymal Stem Cell Injection (hUCMSCs) in the Treatment of Patients With Moderate to Severe Systemic Lupus Erythematosus

This trial will test whether infusions of donated umbilical-cord mesenchymal stem cells can help adults aged 18–65 with moderate to severe systemic lupus erythematosus.

Quick facts

• Phase: PHASE1; PHASE2
• Study type: Interventional
• Enrollment: 58 (estimated)
• Ages: 18 Years to 65 Years
• Sex: All
• Sponsor: Shenzhen Beike Bio-Technology Co., Ltd. (industry)
• Locations: 1 site (Shenzhen, Guangdong)
• Trial ID: NCT07041801 on ClinicalTrials.gov

What this trial studies

This Phase 1/2, randomized, placebo-controlled trial administers allogeneic human umbilical cord–derived mesenchymal stem cells (hUC-MSCs) at multiple dose levels, including a double-dose arm, to adults with moderate to severe SLE to monitor safety and early signs of benefit. Participants are assigned to low, medium, high, double-dose hUC-MSCs, or placebo and are followed for clinical disease activity, laboratory markers, and adverse events. Key enrollment criteria include age 18–65, meeting the 2019 EULAR/ACR SLE classification, and contraceptive requirements for 12 months after dosing. The single study site is Shenzhen Beike Bio-Technology Co. Ltd., with visits required for infusions and follow-up.

Who should consider this trial

Good fit: Ideal candidates are adults 18–65 with moderate to severe SLE who meet 2019 EULAR/ACR criteria, have relevant positive autoantibodies at screening, and can commit to contraception for 12 months after treatment.

Not a fit: Patients with mild or well-controlled SLE, those who are pregnant or planning pregnancy, those outside the age range, or with contraindicating medical conditions are unlikely to benefit from participation.

Why it matters

Potential benefit: If successful, the treatment could reduce lupus disease activity and lessen the need for long-term steroids or other immunosuppressive drugs.

How similar studies have performed: Small early-phase studies and case series of mesenchymal stem cell therapy in SLE have shown encouraging safety and some clinical responses, but larger confirmatory trials are still needed.

Eligibility criteria

Inclusion Criteria:

1. Fully understand the purpose, nature, methods of the trial and possible adverse reactions, voluntarily become a subject, and sign the informed consent form.
2. Age 18-65 years old (inclusive of the boundary values, based on the time of signing the informed consent form), no gender restrictions;
3. The subjects (male and female) must agree not to have a reproductive plan during the trial period and after the injection administration for at least 12 months, and voluntarily take effective contraceptive measures with their partners (see Appendix 1), and have no plans for sperm donation or egg donation;
4. According to the diagnostic classification criteria of the European League Against Rheumatism (EULAR)/American College of Rheumatology (ACR) in 2019, diagnosed with systemic lupus erythematosus (SLE);
5. Meet one of the following conditions: the antinuclear antibody (ANA) is positive at 1:80 during screening or the anti-dsDNA antibody is positive during screening or the anti-Sm antibody is positive during screening;

Exclusion Criteria:

1. Those who are judged by the researchers to be likely to be allergic to the investigational drug or any component thereof;
2. Those who have had central nervous system diseases within 8 weeks before administration (including but not limited to epilepsy, mental illness, interstitial encephalopathy syndrome, stroke, encephalitis, central nervous system vasculitis, etc.);
3. Those who have undergone major organ transplantation (such as heart, lung, kidney, liver) or hematopoietic stem cell/marrow transplantation;
4. Those who have any major diseases/diseases or unstable clinical conditions (such as liver, kidney, hematological, endocrine, pulmonary, immune, mental, etc.) or active infections/infectious diseases with evidence, and according to the researchers' clinical judgment, if the subjects participate in the study, it will significantly increase the risks for the subjects;
5. Those who currently have known or suspected malignant tumors;
6. Those who have severe pulmonary arterial hypertension (\>70 mmHg, 1 mmHg = 0.133 kPa), or mild to moderate pulmonary arterial hypertension patients with severe cardiopulmonary insufficiency;
7. Those who have antiphospholipid syndrome (APS), or have a history of catastrophic antiphospholipid syndrome (CAPS), or although not diagnosed as APS, but are evaluated by the researchers as having an increased risk of thrombosis;
8. Those who have herpes zoster infection within 90 days before administration, or any infection that requires hospitalization treatment or intravenous or intramuscular injection of antibiotics within 60 days before administration;
9. Those who have undergone major surgical operations within 28 days before administration, or those who are expected to undergo major surgical operations during the trial;
10. Subjects who have positive serological tests for viral hepatitis during the screening period, positive human immunodeficiency virus antibody (HIV-Ab), positive hepatitis B surface antigen (HBsAg), positive hepatitis C antibody (HCV-Ab), or positive Treponema pallidum antibody (TP-Ab);
11. Other situations that the researchers consider may affect the subjects' willingness to provide informed consent or follow the trial protocol, or situations where the subjects' participation in the trial may affect the trial results or their own safety.

Where this trial is running

Shenzhen, Guangdong

• Shenzhen Beike Bio-Technology Co. Ltd. — Shenzhen, Guangdong, China (RECRUITING)

Conditions: Systemic Lupus Erythematosus