Chimeric Antigen Receptors T Cells for Refractory/Recurrent Lupus Nephritis in Children (CAR-T)

March 27, 2025 updated by: Guangzhou Women and Children's Medical Center

An Exploratory Clinical Study of the Safety and Efficacy of CAR-T in Children With Refractory/Recurrent Lupus Nephritis Disease

The goal of this prospective, open, single-arm clinical trial was to evaluate the safety and potential efficacy of CAR T cell therapy in children with refractory/recurrent lupus nephritis. The persistence and cell phenotype of CAR-T cells in vivo and CAR-T treatment-related inflammatory factors were evaluated after treatment. To explore new therapeutic methods, in order to reduce the side effects of traditional therapeutic drugs, increase curative effect, and finally make patients obtain long-term survival and improve survival quality.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Recruiting

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Estimated)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
50

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact

The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.

Study Locations

  • China
    • Guangdong
      • Guangzhou, Guangdong, China, 510000
        • Recruiting
        • Guangzhou Women and Children Medical Center
        • Contact:
        • Principal Investigator:
          • Xia Gao

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

  • Child
  • Adult

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  1. Age 6-18 years old (including critical value);
  2. Diagnosed with SLE according to the 2019 EULAR/ACR SLE classification criteria;
  3. According to the 2018 ISN/RPS LN standards diagnosed with active Class III or IV LN, with or without a membranous component and the biopsy must be performed within 6 months prior to screening;
  4. SLEDAI-2000 score ≥8 points;

  5. Meeting the diagnosis of refractory lupus nephritis,

    1. defined as treatment with two or more immunosuppressants (including glucocorticoids, cyclophosphamide, tacrolimus, mycophenolic acid analogues, leflunomide, and cyclosporine) for more than 6 months without inducing remission or relapse after remission,
    2. accompanied by proteinuria without remission;
  6. Positive expression of CD19 in peripheral blood B cells determined by flow cytometry;
  7. Participants had good venous access, no contraindications for cell collection;
  8. Participants and their guardians sign the informed consent, understand the study procedures and participate in the clinical study voluntarily;

  9. The functions of important organs are basically normal:

    1. Hematopoietic function (blood routine should meet):

      • Lymphocyte count ≥1×109/L,
      • White blood cell count ≥3×109/L,
      • Neutrophil count ≥1×109/L (no colony-stimulating factor treatment within 2 weeks prior to examination),
      • Hemoglobin ≥60g/L;

    2. Liver function:

      • ALT≤3×ULN (except elevated ALT caused by inflammatory myopathy),
      • AST≤3×ULN (except for elevated AST caused by inflammatory myopathy),
      • TBIL≤1.5×ULN (except Gilbert syndrome, total bilirubin ≤3.0×ULN);
    3. Renal function: eGFR ≥30 ml/(min.1.73m2) (Schwartz formula, except abnormal renal function by SLE);

    4. Coagulation function:

      • International standardized ratio (INR) ≤1.5×ULN,
      • prothrombin time (PT) ≤1.5×ULN;
    5. Heart function: hemodynamic stability;
  10. Anti-nuclear antibody (ANA) ≥1:80;
  11. Eastern Cancer Cooperation Group (ECOG) physical status score 0 to 2.

Exclusion Criteria:

  1. Received kidney transplant previously;
  2. Serious drug allergy history or allergy;
  3. Presence or suspicion of fungal, bacterial, viral or other infections that cannot be controlled or require treatment;
  4. Complicated with severe organ dysfunction of heart, liver, lung or coagulation dysfunction;
  5. Complicated with congenital immunoglobulin deficiency;

  6. Participants with infectious diseases:

    1. Hepatitis B surface antigen (HBsAg) or hepatitis B core antibody (HBc Ab) positive and peripheral blood hepatitis B virus (HBV) DNA titer greater than the normal reference value range;
    2. Hepatitis C virus (HCV) antibody positive and peripheral blood hepatitis C virus (HCV) RNA titer greater than the normal reference value range;
    3. Human immunodeficiency virus (HIV) antibody positive;
    4. Syphilis positive;
  7. Diagnosed with malignant tumors in the last five years.
  8. Suffer from severe central nervous system disease, mental illness and severe cognitive dysfunction;
  9. Participated in other clinical trials within 3 months before enrollment;
  10. Received CAR-T therapy previously;
  11. Other situations that the researcher considers unsuitable for inclusion.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Experimental: CAR-T
Children who met the inclusion criteria were given transfusions of CAR-T cells
Biological: Low-dose CAR-T cells group
This group of patients received low dose novel structure of Chimeric Antigen Receptors T (CAR-T) cells therapy with an infusion dose of approximately 5×100,000 cells/Kg.
Biological: High-dose CAR-T cells group
This group of patients received high dose novel structure of Chimeric Antigen Receptors T (CAR-T) cells therapy with an infusion dose of approximately 1×1000,000 cells/Kg.

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
Number of participants with CAR-T cells treatment-related adverse events(AE)
Time Frame: 2 years
Incidence and severity of treatment-related AE, including adverse event of special interest (AESI), as assessed by CTCAE v5.0.
2 years

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Overall renal response (ORR) rete
Time Frame: 2 years
ORR rate: Number of participants meet the complete renal response (CRR) or partial renal response (PRR) as a percentage of total participants. Follow-up for 2 years after CAR-T cells reinfusion, monitored and recorded ORR at study set time points (Month 3, Month 6, Month 12, Month 24, or participant withdrawal from the study).
2 years
Number of participants with SRI-4 response
Time Frame: 2 years
Number of participants with SLE response Index 4(SRI-4) response: including Systemic Lupus Erythematosus Disease Activity Index(SLEDAI) 2000 scores decreased by ≥4 points from baseline, PGA with no worsening (VAS increased by <0.30 points from baseline), British Isles Lupus Assessment Group(BILAG) 2004 with no new A domain score and no more than 1 new B domain scores. Follow-up for 2 years after CAR-T cells reinfusion, monitored and recorded SRI-4 response at study set time points (Month 3, Month 6, Month 12, Month 24, or participant withdrawal from the study).
2 years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Guangzhou Women and Children's Medical Center

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Estimated)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
May 1, 2025

Primary Completion (Estimated)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
April 30, 2029

Study Completion (Estimated)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
April 30, 2029

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
February 26, 2025

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
March 27, 2025

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
April 1, 2025

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
April 1, 2025

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
March 27, 2025

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
February 1, 2025

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • [2025]040A01

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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