Actively Recruiting
Exploratory Clinical Study on Safety and Efficacy of Second-generation CD19/BCMA CAR NK Cell Injection in Treating Pediatric B Cell-related Autoimmune Diseases
Led by The Children's Hospital of Zhejiang University School of Medicine · Updated on 2026-02-03
12
Participants Needed
1
Research Sites
52 weeks
Total Duration
On this page
Sponsors
T
The Children's Hospital of Zhejiang University School of Medicine
Lead Sponsor
R
Rui Therapeutics
Collaborating Sponsor
AI-Summary
What this Trial Is About
Researchers are evaluating the safety and effectiveness of second-generation anti-CD19/BCMA CAR-NK cell injections for treating children with B cell-related autoimmune diseases. This early-phase clinical trial focuses on pediatric patients and aims to better understand how these engineered immune cells perform in managing conditions such as systemic lupus erythematosus (SLE), steroid-resistant nephrotic syndrome, IgA nephropathy, systemic sclerosis, and ANCA-associated vasculitis. Participants will receive a treatment regimen starting with lymphodepletion using fludarabine and cyclophosphamide, followed by infusion of the anti-CD19/BCMA CAR NK cells. The study follows a dose-escalation and expansion design to carefully assess safety and response. All participants undergo the same treatment process without placebo comparison. Throughout the study, participants will be closely monitored for adverse effects and treatment response using measures like dose-limiting toxicity, treatment-emergent adverse events, response rates, and CRISS scores over one to six months. Researchers will evaluate cardiac, liver, kidney, and lung function before and during treatment. The total study duration allows for detailed safety and efficacy assessments under medical supervision.
CONDITIONS
Brief Title
Exploratory Clinical Study of Second-generation CD19/BCMA Chimeric Antigen Receptor NK Cell Injection in Treating Paediatric B Cell-related Autoimmune Diseases
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Participant and guardian provide written informed consent.
- Cardiac function with no significant abnormalities and LVEF 65%.
- Liver function with ALT 4 times upper limit normal (ULN), AST 4 times ULN, and total bilirubin 2 times ULN (up to 3 times for Gilbert syndrome).
- Renal function with eGFR 30 mL/min/1.73 m8; may include lower if benefits outweigh risks with consent.
- No severe lung disease; oxygen saturation (SpO2) 92% or higher.
- Women of childbearing potential must use contraception or abstain during treatment and for 12 months after.
- Specific disease criteria including age 5 years for SLE, MDR-SRNS, IgA nephropathy, systemic sclerosis, and ANCA-associated vasculitis.
- Must meet disease-specific diagnostic and treatment history requirements detailed in the protocol.
You will not qualify if you...
- Known severe allergic reactions or hypersensitivity to trial medications.
- Uncontrollable or active infections needing treatment at screening.
- Severe heart failure (NYHA class III or IV), recent heart attack, congenital heart disease, severe arrhythmias, or unstable vital signs.
- Positive tests for hepatitis B or C virus, HIV, syphilis, or cytomegalovirus with elevated viral loads.
- History of severe herpes infections or recent herpes/varicella-zoster infections within 12 weeks.
- Active central nervous system disease or cancer.
- Secondary or congenital immunodeficiency.
- Major medical conditions preventing cell therapy except lupus.
- Recent organ or stem cell transplantation or acute graft-versus-host disease.
- Live vaccine within 4 weeks before screening.
- Positive pregnancy test.
- Participation in other clinical trials with interventions within 3 months before enrollment.
- Any clinically significant abnormal lab results or investigator judgment increasing risk or interfering with study outcomes.
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Up to 1 month
Participants receive fludarabine and cyclophosphamide for lymphodepletion followed by anti-CD19/BCMA CAR NK cell infusion.
1 treatment visit and weekly visits for up to 1 month
Duration - Up to 6 months
Participants are monitored for safety, response rate, and clinical status after treatment.
Monthly visits for up to 6 months
Trial Site Locations
Total: 1 location
1
Children's Hospital of Zhejiang University School of Medicine
Hangzhou, Zhejiang, China, 310052
Actively Recruiting
Research Team
J
Jianhua Mao, Ph.D
Q
Qiuyu Li, Ph.D
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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