Using CD19 Universal CAR T Cells to Treat Severe Systemic Lupus Erythematosus

Efficacy and Safety Study of CD19 Universal CAR T Cells in Refractory Systemic Lupus Erythematosus

This study is testing if a new type of CAR T cell treatment can help children with severe lupus who haven't responded to other therapies.

Quick facts

• Phase: Phase 1
• Study type: Interventional
• Enrollment: 18 (estimated)
• Ages: 5 Years and up
• Sex: All
• Sponsor: The Children's Hospital of Zhejiang University School of Medicine (Academic / other)
• Drugs / interventions: Beliumab, rituximab, CAR-T, CAR T, methotrexate, cyclophosphamide, prednisone
• Locations: 1 site (Hangzhou, Zhejiang)
• Trial ID: NCT06691152 on ClinicalTrials.gov

What this trial studies

This clinical trial evaluates the safety and efficacy of CD19 Universal CAR T cells in patients with refractory systemic lupus erythematosus (SLE), particularly focusing on children. The study aims to address the limitations of traditional autologous CAR T-cell therapies by utilizing universal CAR T cells derived from healthy donors, which can be produced more efficiently and at a lower cost. Participants will be closely monitored for their response to the treatment and any potential side effects. The trial is designed to provide insights into the therapeutic potential of CAR T-cell therapy for a severe autoimmune condition that significantly impacts organ function.

Who should consider this trial

Good fit: Ideal candidates include children aged 5 and older diagnosed with moderate to severe systemic lupus erythematosus who have not responded to standard treatments.

Not a fit: Patients with mild SLE or those who have not yet tried standard therapies may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could provide a new, effective option for children suffering from severe systemic lupus erythematosus.

How similar studies have performed: Other studies have shown promising results with CAR T-cell therapies in autoimmune diseases, indicating potential for success in this novel application.

Eligibility criteria

Inclusion Criteria:

1. Age:≥5 years old;
2. Diagnosed with SLE according to the 2019 EULAR/ACR SLE classification criteria；Still in moderate to severe disease activity despite ≥3M of high dose glucocorticoids(prednisone≥1mg/kg/d or other equivalent amount of other steriod ), hydroxychloroquine and at least 2 of the following treatments(cyclophosphamide, MMF, azathioprine, methotrexate, cyclosporin, tacrolimus, sirolimus, leflunomide, telitacicept, Beliumab, and rituximab); or Intolerant to standard treatments;
3. SLEDAI 2K score≥8 points;
4. The functions of important organs are basically normal:

   Cardiac function: Left ventricular ejection fraction (LVEF) ≥55% with no obvious abnormality in electrocardiogram; Renal function: eGFR≥30ML/min/1.73m2； Liver function: Asparagus cochinchinensis transase (AST) and Alanine Aminotransferase (ALT)≤3.0 ULN, Total Bilirubin (TBIL) in serum ≤2.0×ULN; Lung function: No serious lung lesions, SpO2≥92%;
5. Met the standards of leukapheresis or intravenous blood collection, No contraindication for cell collection;
6. Negative pregnancy test for female Subjects of childbearing age, agree to take effective contraceptive measures the first year after CAR-T infusion;
7. Participants or their guardians agrees to participate in the clinical trial and sign the informed consent form which indicating that he/she understands the purpose and procedure of the clinical trial and is willing to participate in the study.

Exclusion Criteria:

1. Received CAR T cell therapy previously;
2. Central nervous system (CNS) disease: CNS neurolupus requires intervention within 60 days);
3. Active tuberculosis at the time of screening;
4. Have a history of congenital heart disease or acute myocardial infarction within 6 months prior to screening; Or severe arrhythmias (including multisource frequent supraventricular tachycardia, ventricular tachycardia, etc.); Or combined with moderate to massive pericardial effusion, serious myocarditis, etc; Or patients with unstable vital signs who need hypertensive drugs；
5. Suffer from other diseases that require long-term use of glucocorticoid or high-dose of immunosuppressive agents;
6. Uncontrollable infection, or active infection that requires systemic treatment within 1 week prior to screening；
7. History of organ transplantation or hematopoietic stem cell transplantation, or ≥Grade 2 GVHD within 2 weeks prior to screening；
8. Hepatitis B surface antigen (HBsAg) or hepatitis B core antibody (HBcAb) positive and peripheral blood hepatitis B virus (HBV) DNA titer greater than the normal reference value range; Or hepatitis C virus (HCV) antibody positive and peripheral blood hepatitis C virus (HCV) RNA titer greater than the normal reference value range; Or positive for human immunodeficiency virus (HIV) antibodies; Or syphilis test positive; Or cytomegalovirus (CMV) DNA test positive;
9. Received live vaccine within 4 weeks before screening;
10. Tested positive in Blood pregnancy test；
11. Previous or concurrent malignancy；
12. Patients who participated in other clinical study within 3 months prior to enrollment;
13. Any other conditions that the investigators deem it unsuitable for the study.

Where this trial is running

Hangzhou, Zhejiang

• Children's Hospital, Zhejiang University School of Medicine — Hangzhou, Zhejiang, China (Recruiting)

Study contacts

• Principal investigator: Jianhua Mao — The Children's Hospital of Zhejiang University School of Medicine
• Study coordinator: Jianhua Mao, Prof

Conditions Systemic Lupus Erythematosus; CAR T-Cells; Pediatrics