Multi-Drug Resistant Nephrotic SyndromePhase 1NCT06842589
Study of Therapeutic Efficacy of Anti-CD19 CAR-T Cell Therapy in Patients With MDR-SRNS
A Phase 1 study testing anti-CD19 CAR-T cells in people with Multi-Drug Resistant Nephrotic Syndrome and CAR-T Cell Therapy, run by The Children's Hospital of Zhejiang University School of Medicine.
Trial summary
This is an investigator-initiated trial aimed at assessing the safety and efficacy of anti-CD19 CAR-T cells in the treatment of patients with Multi-drug resistant SRNS Read more about this study
- Who is it for?
- Ages 2 years and older
- Conditions the study is for
- Multi-Drug Resistant Nephrotic Syndrome, CAR-T Cell Therapy
- What is being tested?
- anti-CD19 CAR-T cells
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- No sites listed yet
- Study dates
- 14 May 2025to 31 Jan 2028
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 2 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 1. Age ≥2 years old, gender unlimited;
- 2.Diagnosed with SRNS according to the 2021 Kidney Disease: Improving Global Outcomes (KDIGO) Guidelines and have not achieved a complete response after 12 months of treatment with two standard doses of hormone replacement drugs with different mechanisms of action or relapse of disease activity after remission (at least one of the two drugs is a calcineurin inhibitor such as cyclosporine or tacrolimus; Other hormone replacement drugs include Mycophenolate Mofetil, cyclophosphamide, Taitacept or rituximab); Or if no remission has been achieved after 3 to 6 months of adequate treatment with one calcineurin inhibitor, if the researcher judges that the benefits outweigh the risks and the patient or guardian has fully informed consent, the patient can be considered for inclusion.Patients with other diseases, such as systemic lupus erythematosus, requiring long-term systemic treatment with glucocorticoids or immunosuppressants, may be considered for inclusion after the investigator determines that the benefits outweigh the risks and the patient or guardian has fully informed consent;
- 3. Renal biopsy was performed and the pathological type was determined to be minimal lesion nephropathy(MCD) or focal segmental glomerulosclerosis (FSGS);
- 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 transaseminase (AST) and Alanine Aminotransferase (ALT)≤3.0 upper limit of normal, Total Bilirubin (TBIL) in serum ≤2.0×upper limit of normal; 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.
Who cannot join
- 1. Received CAR T cell therapy or other gene-modified cell therapy previously;
- 2. Patients had a cerebrovascular accident or seizure, or other active central nervous system disease within 6 months;
- 3. Genetic tests have confirmed hereditary kidney disease;
- 4. Renal biopsy has been confirmed as immunoglobulin A nephropathy, idiopathic membranous nephropathy or membranoproliferative glomerulonephritis;
- 5. Renal replacement therapy has been or is being performed within 3 months prior to transfusion. (if acute kidney injury factors were considered, patients with chronic kidney disease were excluded, and the benefits outweighed the risks as determined by the investigator and with the full and informed consent of the patient or guardian could be considered for inclusion);
- 6. Renal replacement therapy has been or is being performed within 3 months prior to transfusion;
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- 7. 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;
- 8. Received solid organ transplantation or hematopoietic stem cell transplantation within 3 months prior to screening; Acute graft-versus-host disease (GVHD) of grade 2 or above was present within 2 weeks prior to screening;
- 9. 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;
- 10. Macrophage activation syndrome occurred within 1 month prior to screening;
- 11. Received live vaccine within 4 weeks before screening;
- 12. Patients with malignant diseases such as tumors before screening, or with other serious life-threatening diseases;
- 13. Tested positive in Blood pregnancy test;
- 14. Patients who participated in other clinical study within 1 months prior to enrollment;
- 15. Any other conditions that the investigators deem it unsuitable for the study.
About this study
Official title: Study of Therapeutic Efficacy of Anti-CD19 Chimeric Antigen Receptor T Lymphocytes (CAR-T) Cells in Patients With Multi-drug Resistant SRNS
This is an investigator-initiated trial aimed at assessing the safety and efficacy of anti-CD19 CAR-T cells in the treatment of patients with Multi-drug resistant SRNS
Read the detailed description
At present, there is no effective treatment for Multi-drug resistant steroid resistant nephrotic syndrome (MDR-SRNS), which has a high risk of progression to kidney failure, and about 55% of patients will have disease recurrence after receiving kidney transplantation, which is in urgent need of new treatment methods.
CAR-T therapy is an adoptive cell therapy that uses genetic modification technology to reprogram T cells and eliminate target cells expressing disease-related antigens through antigen-specific recognition.Since 2019, CAR-T cell therapy has been successfully applied to autoimmune diseases. Although no clinical data related to CAR-T treatment of nephrotic syndrome has been disclosed, CAR-T is effective for systemic lupus erythematosus and systemic sclerosis.Many kinds of autoimmune diseases such as chemical syndrome and idiopathic inflammatory dermatomyositis have good therapeutic effect. These results suggest that the therapeutic effect of CAR T cells may not be limited to systemic lupus erythematosus, but may also play a role in several different B-cell-mediated and autoantibody-driven human autoimmune diseases, which is expected to bring breakthroughs in the treatment of MDR-SRNS.The purpose of this study is to assess the safety and efficacy of the anti-CD19 CAR-T cells in the treatment of patients with MDR-SRNS.
- Being tested
- anti-CD19 CAR-T cells
- Keywords
- car-t, Multi-Drug Resistant Nephrotic Syndrome
- Medical subject headings
- Nephrotic Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
anti-CD19 CAR-T cellsBiological
Three dose groups (0.3×105/kg, 1×105/kg, 3×105/kg) were set up, starting from the low dose group climbing to explore the safe and effective dose.
| Study group | Role | What they receive |
|---|---|---|
| CAR-T treatment group | Experimental | This study employs a '3+3' design with three dosage groups (0.3×10^5/kg, 1×10^5/kg, and 3×10^5/kg). Each dosage group will enroll 3 to 6 patients, starting from the lowest dosage and escalating to explore a safe and effective dose. Once a dosage group completes enrollment without observing any serious unexpected adverse reactions, and based on the efficacy and cell kinetics data, the investigator and the technical team together may consider escalating to the next dosage group to explore the optimal effective dose. The trial is expected to enroll a total of 9 to 18 patients Receives: Biological: anti-CD19 CAR-T cells |
What is being measured
The primary outcome is the main question the study is designed to answer.
The safety of CAR-T cell therapy in patients with MDR SRNS
Incidence and severity of Adverse Events (AEs) and Serious Adverse Event(SAEs),including changes in laboratory values,Electrocardiograph(ECG) and vital signs assessed by CommonTerminology Criteria for Adverse Events (CTCAE) v5.0.
Measured: 3 months
The efficiency of CAR-T cell therapy in patients with MDR SRNS
Complete response and partial response rate (complete response defined as Urinary protein/creatinine ratio (uPCR) ≤200 mg/g for 3 consecutive days, partial response is defined as a 50% or more reduction in proteinuria from baseline and 200mg/g < uPCR <2000mg/g)
Measured: 3 months
Secondary outcomes (5)
Effectiveness of other visit points
Rate of complete or partial response at other visit points after cell infusion (1 month, 2 months, 6 months, 9 months, 12 months, 18 months, 24 months)
Measured: 24 months
Pharmacokinetic Outcome Cmax
The highest concentration (Cmax) of targeted CD19 CAR-T cells expanded in peripheral blood after infusion
Measured: 3 months
Pharmacokinetic Outcome Tmax
The time to reach the maximum concentration (Tmax)
Measured: 3 months
Pharmacokinetic Outcome AUC
The area under the curve (AUC) at 28 days and 90 days post-infusion (AUC28d/90d)
Measured: 3 months
Pharmacodynamics Outcome
The degree of B cell clearance at various time points, and the concentration levels of CAR-T related serum cytokines such as IL-6, CRP, etc
Measured: 3 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Feb 2025
- Study start14 May 2025 (estimated)
- Primary completion14 Feb 2027 (estimated)
- Study completion31 Jan 2028 (estimated)
Why it stopped: no participants enrolled
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Multi-Drug Resistant Nephrotic SyndromeStudies of the same condition, open and under way.
- More trials run by The Children's Hospital of Zhejiang University School of MedicineThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Mao Jianhua, The Children's Hospital of Zhejiang University School of Medicine
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06842589
- Sponsor's protocol ID
- 2025-SC-0001-P-01
- Status last verified
- Jun 2026
- First submitted
- 22 Jan 2025
- Last updated
- 16 Jun 2026
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 16 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Study of Therapeutic Efficacy of Anti-CD19 Chimeric Antigen Receptor T Lymphocytes (CAR-T) Cells in Patients With Multi-drug Resistant SRNS
- ClinicalTrials.gov ID
- NCT06842589
- Acronym / sponsor ID
- 2025-SC-0001-P-01
- Keywords
- car-t, Multi-Drug Resistant Nephrotic Syndrome
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: no participants enrolled
- Enrolment
- 0 participants (actual)
- Lead sponsor
- The Children's Hospital of Zhejiang University School of MedicineUniversity, hospital or other
- Study start
- 14 May 2025 (estimated)
- Primary completion
- 14 Feb 2027 (estimated)
- Study completion
- 31 Jan 2028 (estimated)
- First posted
- 24 Feb 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is an investigator-initiated trial aimed at assessing the safety and efficacy of anti-CD19 CAR-T cells in the treatment of patients with Multi-drug resistant SRNS
Detailed description
At present, there is no effective treatment for Multi-drug resistant steroid resistant nephrotic syndrome (MDR-SRNS), which has a high risk of progression to kidney failure, and about 55% of patients will have disease recurrence after receiving kidney transplantation, which is in urgent need of new treatment methods.
CAR-T therapy is an adoptive cell therapy that uses genetic modification technology to reprogram T cells and eliminate target cells expressing disease-related antigens through antigen-specific recognition.Since 2019, CAR-T cell therapy has been successfully applied to autoimmune diseases. Although no clinical data related to CAR-T treatment of nephrotic syndrome has been disclosed, CAR-T is effective for systemic lupus erythematosus and systemic sclerosis.Many kinds of autoimmune diseases such as chemical syndrome and idiopathic inflammatory dermatomyositis have good therapeutic effect. These results suggest that the therapeutic effect of CAR T cells may not be limited to systemic lupus erythematosus, but may also play a role in several different B-cell-mediated and autoantibody-driven human autoimmune diseases, which is expected to bring breakthroughs in the treatment of MDR-SRNS.The purpose of this study is to assess the safety and efficacy of the anti-CD19 CAR-T cells in the treatment of patients with MDR-SRNS.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| CAR-T treatment group | Experimental | Biological: anti-CD19 CAR-T cellsThis study employs a '3+3' design with three dosage groups (0.3×10^5/kg, 1×10^5/kg, and 3×10^5/kg). Each dosage group will enroll 3 to 6 patients, starting from the lowest dosage and escalating to explore a safe and effective dose. Once a dosage group completes enrollment without observing any serious unexpected adverse reactions, and based on the efficacy and cell kinetics data, the investigator and the technical team together may consider escalating to the next dosage group to explore the optimal effective dose. The trial is expected to enroll a total of 9 to 18 patients |
Who may qualify
- Ages
- 2 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (7 listed)
- 1. Age ≥2 years old, gender unlimited;
- 2.Diagnosed with SRNS according to the 2021 Kidney Disease: Improving Global Outcomes (KDIGO) Guidelines and have not achieved a complete response after 12 months of treatment with two standard doses of hormone replacement drugs with different mechanisms of action or relapse of disease activity after remission (at least one of the two drugs is a calcineurin inhibitor such as cyclosporine or tacrolimus; Other hormone replacement drugs include Mycophenolate Mofetil, cyclophosphamide, Taitacept or rituximab); Or if no remission has been achieved after 3 to 6 months of adequate treatment with one calcineurin inhibitor, if the researcher judges that the benefits outweigh the risks and the patient or guardian has fully informed consent, the patient can be considered for inclusion.Patients with other diseases, such as systemic lupus erythematosus, requiring long-term systemic treatment with glucocorticoids or immunosuppressants, may be considered for inclusion after the investigator determines that the benefits outweigh the risks and the patient or guardian has fully informed consent;
- 3. Renal biopsy was performed and the pathological type was determined to be minimal lesion nephropathy(MCD) or focal segmental glomerulosclerosis (FSGS);
- 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 transaseminase (AST) and Alanine Aminotransferase (ALT)≤3.0 upper limit of normal, Total Bilirubin (TBIL) in serum ≤2.0×upper limit of normal; Lung function: No serious lung lesions, SpO2≥92%;
- 5. Met the standards of leukapheresis or intravenous blood collection, No contraindication for cell collection;
Key exclusion criteria (15 listed)
- 1. Received CAR T cell therapy or other gene-modified cell therapy previously;
- 2. Patients had a cerebrovascular accident or seizure, or other active central nervous system disease within 6 months;
- 3. Genetic tests have confirmed hereditary kidney disease;
- 4. Renal biopsy has been confirmed as immunoglobulin A nephropathy, idiopathic membranous nephropathy or membranoproliferative glomerulonephritis;
- 5. Renal replacement therapy has been or is being performed within 3 months prior to transfusion. (if acute kidney injury factors were considered, patients with chronic kidney disease were excluded, and the benefits outweighed the risks as determined by the investigator and with the full and informed consent of the patient or guardian could be considered for inclusion);
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| anti-CD19 CAR-T cellsThree dose groups (0.3×105/kg, 1×105/kg, 3×105/kg) were set up, starting from the low dose group climbing to explore the safe and effective dose. | Biological | CAR-T treatment group |
Primary outcome
The safety of CAR-T cell therapy in patients with MDR SRNS
Time frame: 3 months
Incidence and severity of Adverse Events (AEs) and Serious Adverse Event(SAEs),including changes in laboratory values,Electrocardiograph(ECG) and vital signs assessed by CommonTerminology Criteria for Adverse Events (CTCAE) v5.0.
The efficiency of CAR-T cell therapy in patients with MDR SRNS
Time frame: 3 months
Complete response and partial response rate (complete response defined as Urinary protein/creatinine ratio (uPCR) ≤200 mg/g for 3 consecutive days, partial response is defined as a 50% or more reduction in proteinuria from baseline and 200mg/g < uPCR <2000mg/g)
5 secondary outcomes
Effectiveness of other visit points
Time frame: 24 months
Pharmacokinetic Outcome Cmax
Time frame: 3 months
Pharmacokinetic Outcome Tmax
Time frame: 3 months
Pharmacokinetic Outcome AUC
Time frame: 3 months
Pharmacodynamics Outcome
Time frame: 3 months
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Jun 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing anti-CD19 CAR-T cells in people with Multi-Drug Resistant Nephrotic Syndrome and CAR-T Cell Therapy, run by The Children's Hospital of Zhejiang University School of Medicine.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 15 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: withdrawn, as of the sponsor's last update on 16 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was withdrawn before enrolling participants, so there are no results.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
The safety of CAR-T cell therapy in patients with MDR SRNS (time frame: 3 months). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 16 Jun 2026.