Study of Therapeutic Efficacy of CAR-T Cell Therapy in Patients With MDR-SRNS

NCT06553898 | Recruiting Phase 1

• 1 other identifier: 2024-IRB-0205-P-01
• Study Type: interventional
• Target: 18
• Locations: 1 country
• Sites: 1

Brief Summary

This is an investigator-initiated trial aimed at assessing the safety and efficacy of anti-BCMA/CD70 CAR-T cells in the treatment of patients with Multi-drug resistant SRNS

Conditions

Multi-Drug Resistant Nephrotic Syndrome; CAR-T Cell Therapy

Keywords

MDR-SRNS; CAR-T cell therapy; Multi-Drug Resistant Nephrotic Syndrome

Outcome Measures

Primary Outcomes (1)

• 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.

  3 months

Secondary Outcomes (2)

• The efficiency of CAR-T cell therapy in patients with MDR SRNS

  6 months

• Cellular kinetics

  3 months

Study Arms (1)

CAR-T treatment group

EXPERIMENTAL

The trial consists of two phases, Dose Exploration(Part A) and Dose Expansion (Part B). In Part A, three dose groups (0.3×105/kg, 1×105/kg, 3×105/kg) are set up, starting from the low dose group to explore the safe and effective dose. Each dose group will be enrolled in 3 cases.If the optimal effective dose is still not explored in the highest dose group, the dose can be increased according to the situation, and the highest dose is no more than 6×105/kg. Upon the completion of Part A, 1 optimal dose is selected by the comprehensive judgment of the investigator and the technical partner to enter into the Part B stage. The group will then be enrolled in 3\~6 cases to continue to validate the safety and efficacy.A total enrollment of 12-18 patients is expected in the whole process of the trial.

Biological: anti-BCMA/CD70 CAR-T cells

Interventions

anti-BCMA/CD70 CAR-T cells BIOLOGICAL

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.

CAR-T treatment group

Eligibility Criteria

• Age: 2 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• \. Age ≥2 years old, gender unlimited;
• \. Renal biopsy was performed and the pathological type was determined to be minimal lesion nephropathy(MCD) or focal segmental glomerulosclerosis (FSGS);
• \. 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%;
• \. Met the standards of leukapheresis or intravenous blood collection, No contraindication for cell collection;
• \. Negative pregnancy test for female Subjects of childbearing age, agree to take effective contraceptive measures the first year after CAR-T infusion;
• \. 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.

You may not qualify if:

• \. Received CAR T cell therapy or other gene-modified cell therapy previously;
• \. Patients had a cerebrovascular accident or seizure, or other active central nervous system disease within 6 months;
• \. Genetic tests have confirmed hereditary kidney disease;
• \. Renal biopsy has been confirmed as immunoglobulin A nephropathy, idiopathic membranous nephropathy or membranoproliferative glomerulonephritis;
• \. Renal replacement therapy has been or is being performed within 3 months prior to transfusion;
• \. 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;
• \. 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;
• \. 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;
• \. Macrophage activation syndrome occurred within 1 month prior to screening;
• \. Received live vaccine within 4 weeks before screening;
• \. Patients with malignant diseases such as tumors before screening, or with other serious life-threatening diseases;
• \. Tested positive in Blood pregnancy test;
• \. Patients who participated in other clinical study within 1 months prior to enrollment;
• \. Any other conditions that the investigators deem it unsuitable for the study.

Sponsors & Collaborators

• The Children's Hospital of Zhejiang University School of Medicine: lead

Study Sites (1)

Children's Hospital, Zhejiang University School of Medicine

Hangzhou, Zhejiang, China

RECRUITING

MeSH Terms

Conditions

Nephrotic Syndrome

Condition Hierarchy (Ancestors)

Nephrosis; Kidney Diseases; Urologic Diseases; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Male Urogenital Diseases

Study Officials

• Jianhua Mao, PhD

  Children's Hospital, Zhejiang University School of Medicine

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Jianhua Mao, PhD

CONTACT

13516819071; maojh88@zju.edu.cn

Guoping Huang, MD

CONTACT

13738196684; 6510018@zju.edu.cn

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor

Study Record Dates

• First Submitted: August 8, 2024
• First Posted: August 14, 2024
• Study Start: August 13, 2024
• Primary Completion (Estimated): July 31, 2026
• Study Completion (Estimated): July 31, 2027
• Last Updated: August 29, 2025

Record last verified: 2025-08

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)