Actively Recruiting
Study of Therapeutic Efficacy of Anti-CD19 Chimeric Antigen Receptor T Lymphocytes (CAR-T) Cells in Patients With Multi-drug Resistant SRNS
Led by The Children's Hospital of Zhejiang University School of Medicine · Updated on 2025-05-07
18
Participants Needed
1
Research Sites
50 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating the safety and effectiveness of anti-CD19 CAR-T cell therapy in patients with Multi-drug resistant steroid resistant nephrotic syndrome (MDR-SRNS), a condition with limited treatment options and a high risk of progressing to kidney failure. This Phase 1 trial aims to explore a potential new treatment approach for this challenging disease, which often recurs after kidney transplantation. CAR-T therapy, a type of adoptive cell therapy, has shown promise in other autoimmune diseases, suggesting it may also be beneficial for MDR-SRNS. The study uses a 3+3 dose escalation design with three dose groups: 0.3 x 10^5/kg, 1 x 10^5/kg, and 3 x 10^5/kg of anti-CD19 CAR-T cells. Each group will enroll 3 to 6 patients, starting from the lowest dose and moving up if no serious adverse reactions occur. This approach helps determine the safest and most effective dose. The trial plans to enroll a total of 9 to 18 patients. Participants will be closely monitored over three months to assess the safety and efficacy of the CAR-T cell therapy. Researchers will collect data on how the treatment affects the disease, including pharmacokinetics and pharmacodynamics. Follow-up visits will continue up to 24 months to evaluate longer-term effects. Assessments will include laboratory tests, renal function evaluations, and monitoring for any adverse events throughout the study duration.
CONDITIONS
Brief Title
Study of Therapeutic Efficacy of Anti-CD19 CAR-T Cell Therapy in Patients With MDR-SRNS
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Age 2 years or older, any gender
- Diagnosed with steroid resistant nephrotic syndrome (SRNS) based on 2021 KDIGO Guidelines and not fully responsive after 12 months of treatment with two standard hormone drugs or relapse after remission
- If no remission after 3-6 months of adequate treatment with one calcineurin inhibitor, inclusion possible with informed consent
- Renal biopsy confirming minimal lesion nephropathy (MCD) or focal segmental glomerulosclerosis (FSGS)
- Normal major organ functions including heart (LVEF ≥55%), kidney (eGFR ≥30 mL/min/1.73m2), liver (AST/ALT ≤3x upper limit, TBIL ≤2x upper limit), lungs (SpO2 ≥92%)
- Eligible for leukapheresis or intravenous blood collection without contraindications
- Negative pregnancy test for females of childbearing age and agreement to use contraception for one year after CAR-T infusion
- Participant or guardian consents to participate and signs informed consent
You will not qualify if you...
- Previous CAR T cell therapy or other gene-modified cell therapy
- Cerebrovascular accident, seizure, or active central nervous system disease within 6 months
- Confirmed hereditary kidney disease by genetic testing
- Renal biopsy confirming immunoglobulin A nephropathy, idiopathic membranous nephropathy, or membranoproliferative glomerulonephritis
- Renal replacement therapy within 3 months prior to transfusion unless benefits outweigh risks with consent
- History of congenital heart disease, recent myocardial infarction, severe arrhythmias, significant myocarditis, or unstable vital signs
- Solid organ or hematopoietic stem cell transplantation within 3 months prior to screening or recent acute graft-versus-host disease
- Positive tests for hepatitis B, hepatitis C, HIV, syphilis, or cytomegalovirus
- Macrophage activation syndrome within 1 month prior to screening
- Live vaccine within 4 weeks before screening
- Malignancies or serious life-threatening diseases before screening
- Positive blood pregnancy test
- Participation in another clinical study within 1 month prior to enrollment
- Any other condition deemed unsuitable by investigators
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 - 3 months
Participants receive anti-CD19 CAR-T cell therapy at one of three dose levels to assess safety and efficacy.
Multiple visits during the 3 months for treatment administration and monitoring
Duration - Up to 21 months after treatment
Participants are monitored for safety and efficacy outcomes after treatment.
Visits for assessments up to 24 months
Trial Site Locations
Total: 1 location
1
Children's Hospital, Zhejiang University School of Medicine
Hangzhou, Zhejiang, China, 310000
Actively Recruiting
Research Team
J
Jianhua Mao, PhD
G
Guoping Huang, MM
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
Similar Trials
Frequently Asked Questions
Have more questions? Get in touch with our team for quick support
Not the Right Trial for You?
Explore thousands of other clinical trials that might be a better match.
Sign up to get personalized trial recommendations delivered to your inbox.
Already have an account? Log in here