Actively Recruiting
Exploratory Clinical Study on the Safety and Efficacy of Anti-CD19/BCMA CAR-NK Cell Injection for Treating Relapsed/Refractory Pediatric Rheumatic Diseases
Led by The Children's Hospital of Zhejiang University School of Medicine · Updated on 2026-03-24
36
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 Co., Ltd
Collaborating Sponsor
AI-Summary
What this Trial Is About
Researchers are evaluating the safety and effectiveness of anti-CD19/BCMA CAR-NK cell injection in children aged 5 years and older with refractory pediatric rheumatic diseases. This early phase 1 pilot study focuses on patients whose conditions have not responded well to standard treatments, aiming to explore this new biological therapy's potential benefits and risks. Participants will receive chemotherapy drugs fludarabine and cyclophosphamide on days -5, -4, and -3 before receiving multiple doses of the investigational anti-CD19/BCMA CAR-NK cells (KN5601) using a dose-escalation approach. This single-arm, open-label study does not include a comparison group, and the treatment phase includes careful dose adjustments. Throughout the study, participants will be closely monitored for dose-limiting toxicities up to 28 days after infusion. Researchers will assess clinical remission and inactive disease status at 3, 6, 9, and 12 months, as well as monitor B cell depletion rates for up to 52 weeks. Safety evaluations, laboratory tests, and clinical assessments will be conducted regularly to track responses and adverse effects during the study period ending in late 2027.
CONDITIONS
Brief Title
Exploratory Clinical Study on the Safety and Efficacy of Anti- CD19/BCMA CAR-NK Cell Injection for the Treatment of Refractory Pediatric Rheumatic Diseases
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Participants of any gender aged 5 years or older
- Patient or legal guardian agrees to participate and signs informed consent
- Peripheral blood B cells express CD19 with count above 5 cells/uL
- If previously treated with B cell therapy, blood B cell count normalized or above pre-treatment level
- Normal cardiac structure and left ventricular ejection fraction of 55% or higher
- Liver function: AST and ALT up to 3 times upper limit of normal, total bilirubin up to 2 times upper limit
- Renal function: eGFR of 30 mL/min/1.73m² or higher; exceptions evaluated by investigator
- No severe pulmonary lesions and blood oxygen saturation of 92% or higher
- Female participants of childbearing potential must have negative pregnancy test and use contraception during and 1 year after treatment
- Disease-specific criteria for polyarticular juvenile idiopathic arthritis, Sjögren's syndrome, juvenile dermatomyositis, systemic sclerosis, systemic lupus erythematosus, and mixed connective tissue disease as detailed in protocol
You will not qualify if you...
- History or current malignancy except certain cured skin or cervical cancers
- Allergy or intolerance to study drugs or components including fludarabine, cyclophosphamide, or tocilizumab
- Active severe infections or uncontrolled systemic fungal infections
- Cardiac insufficiency NYHA Class III or IV, congenital heart disease, recent myocardial infarction, severe arrhythmias, or unstable vital signs
- Positive tests for hepatitis B, C, HIV, syphilis, or cytomegalovirus with active infection
- History of severe herpes infections or recent herpes/varicella-zoster virus infections
- Active tuberculosis or positive interferon-gamma release assay for tuberculosis
- Interstitial lung disease with severe impairment, or requiring oxygen therapy
- Pulmonary arterial hypertension with severe symptoms or recent hospitalization
- History of epilepsy or active central nervous system diseases
- Acquired or congenital immunodeficiency
- Significant cardiac, endocrine, hematological, hepatic, immunological, metabolic, urological, pulmonary, neurological, dermatological, psychiatric, or renal diseases precluding treatment
- Recent organ or stem cell transplantation or acute graft-versus-host disease
- Recent live vaccination
- Recent use of specified immunosuppressive or biologic therapies within set timeframes
- Participation in another clinical trial within 3 months
- Any other condition increasing risk or interfering with study results per investigator judgment
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 screening and enrollment visit
Duration - Up to 28 days after infusion
Participants receive Fludarabine and Cyclophosphamide on Day -5, -4, and -3, followed by multiple doses of anti-CD19/BCMA CAR-NK cells (KN5601) using a dose-escalation strategy.
Multiple visits for dosing and monitoring during treatment
Duration - Up to 52 weeks after infusion
Participants are monitored for safety and efficacy, including clinical remission and B cell depletion rates over 12 months after infusion.
Visits at months 3, 6, 9, and 12 for assessments
Trial Site Locations
Total: 1 location
1
The Children's Hospital of Zhejiang University School of Medicine
Hangzhou, Zhejiang, China
Actively Recruiting
Research Team
M
Meiping Lu, Prof.
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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