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Paediatric B Cell-related Autoimmune DiseasesEarly Phase 1NCT07283315

Exploratory Clinical Study of Second-generation CD19/BCMA Chimeric Antigen Receptor NK Cell Injection in Treating Paediatric B Cell-related Autoimmune Diseases

A Early Phase 1 study testing anti-CD19/BCMA CAR NK cells (KN5601-K) in people with Paediatric B Cell-related Autoimmune Diseases and Autoimmune Diseases, run by The Children's Hospital of Zhejiang University School of Medicine at 1 site in China.

Recruiting

Looking for participants now. Contact the study team or a site below to ask about taking part.

Sites and contacts
Official record
Search all trials

Trial summary

Evaluating the efficacy and safety of anti-CD19/BCMA CAR-NK Cells in Paediatric B cell-related autoimmune diseases Read more about this study

Who is it for?
Ages 3 years and older
What is being tested?
anti-CD19/BCMA CAR NK cells (KN5601-K)
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
12 participants
Where
1 siteChina
Study dates
3 Dec 2025to 30 Dec 2029
Phase
Early Phase 1
Are there results?
Not yet: the study is under way

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 3 years and older
  • All sexes
  • No healthy volunteers
  • Child, Adult, Older adult

Who may be able to join

  • Common Inclusion Criteria:
  • Informed consent: The participant (and guardian if the participant is a minor) must provide written informed consent, indicating understanding of the study purpose and procedures and willingness to participate.
  • Cardiac Function: Echocardiography shows no significant structural abnormalities and left ventricular ejection fraction (LVEF) ≥ 55%.
  • Hepatic Function: ALT ≤ 3 × ULN (except when elevation is attributable to inflammatory myopathy); AST ≤ 3 × ULN (except when elevation is attributable to inflammatory myopathy); Total bilirubin ≤ 2.0 × ULN (≤ 3.0 × ULN allowed for Gilbert syndrome).
  • Renal Function: eGFR ≥ 30 mL/min/1.73 m².(Participants with eGFR < 30 mL/min/1.73 m² and/or those receiving renal replacement therapy may be considered eligible if, in the investigator's judgment, the potential benefit outweighs the risk and the participant or guardian provides fully informed consent.)
  • Pulmonary Function: No severe pulmonary disease, and SpO₂ ≥ 92%.
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  • Women of childbearing potential must use medically acceptable contraception or practice abstinence during the study treatment period and for at least 12 months after the end of study treatment.
  • Disease-Specific Inclusion Criteria
  • SLE:
  • Age ≥ 5 years.
  • Meets the 2012 SLICC or 2019 EULAR/ACR classification criteria for SLE.
  • Must meet at least one of the following adequate treatment conditions:
  • Treated with glucocorticoids (≥1 mg/kg/day prednisone or equivalent) plus one or more immunomodulatory agents (including cyclophosphamide, MMF, azathioprine, methotrexate, cyclosporine, tacrolimus, sirolimus, leflunomide, thalidomide, belimumab, or rituximab) for at least 3 months.
  • Patients unable to tolerate conventional therapy may be eligible if, in the investigator's judgment, the potential benefit outweighs the risk and the participant (or guardian if minor) provides fully informed consent.
  • Patients who cannot taper glucocorticoids to ≤5 mg/day after 6 months of conventional therapy.
  • SLE disease activity: SLEDAI-2K score ≥ 6.
  • No occurrence of macrophage activation syndrome within 1 month prior to screening.
  • MDR-SRNS
  • Age ≥3 years old, male or female.
  • Diagnosed with SRNS according to the 2021 Kidney Disease: Improving Global Outcomes #KDIGO# Guidelines;
  • Must meet at least one of the following adequate treatment conditions:
  • 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 replacement drugs include Mycophenolate Mofetil, cyclophosphamide, Taitacept or rituximab).
  • if no remission has been achieved after 3 to 6 months of adequate treatment with one calcineurin- inhibitor. 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.
  • Patients unable to tolerate conventional therapy may be eligible if, in the investigator's judgment, the potential benefit outweighs the risk and the participant (or guardian if minor) provides fully informed consent.
  • Renal biopsy was performed and the pathological type was determined to be minimal lesion nephropathy(MCD) or focal segmental glomerulosclerosis (FSGS);
  • IgA nephropathy
  • Age: ≥ 5 years old,male or female;
  • IgA nephropathy pathologically confirmed by renal biopsy;
  • Angiotensin-Converting Enzyme Inhibitors (ACE) or angiotensin receptor blocker (ARB) treated for at least 3 months and meet at least one of the following requirements:
  • Combination or sequential treatment with steroids and at least one immunosuppressant or biologic for ≥ 3 months; and 24-hour urine protein quantification ≥500mg or UPCR≥0.5mg/mg;
  • >50% decline in eGFR within 3 months;
  • Patients who are unable to tolerate conventional treatment and for whom the investigator determines the benefits outweigh the risks and who have obtained fully informed consent from the patient or guardian may be considered for inclusion;
  • Exclude subjects with other secondary causes; exclude patients with uncontrolled blood pressure.
  • Refractory/Relapsed/Progressive Systemic Sclerosis:
  • Age: ≥ 5 years old, male or female;
  • Scleroderma fulfilling the 2013 ACR classification criteria
  • Positive scleroderma-related antibodies.
  • Modified Rodnan Skin Score (mRSS) ≥ 15 (total score 51).
  • Must meet at least one of the following adequate treatment conditions:
  • Treated with glucocorticoids (≥0.5 mg/kg/day) plus at least one immunomodulatory agent (including antimalarials, cyclophosphamide, azathioprine, mycophenolate mofetil, methotrexate, leflunomide, tacrolimus, cyclosporine, or biologics such as rituximab, belimumab, thalidomide) for more than 3 months without significant improvement.
  • Meets criteria for rapidly progressive disease (see Appendix 1) and is unresponsive to standard therapy; participant may be enrolled if the investigator determines that potential benefit outweighs risk and informed consent is obtained.
  • Patients unable to tolerate conventional therapy may be eligible if the investigator determines that potential benefit outweighs risk and informed consent is obtained.
  • Presence of severe pulmonary arterial hypertension (PAH), defined as a mean pulmonary arterial pressure >45 mmHg, or other severe involvement of major organs will be exclude.
  • Refractory/Relapsed ANCA-Associated Vasculitis:
  • Age: ≥ 5 years old, male or female;
  • Meets the diagnostic criteria for ANCA-associated vasculitis according to the 2007 European Medicines Agency (EMA) algorithm or the 2022 ACR/EULAR classification criteria, including microscopic polyangiitis (MPA) and granulomatosis with polyangiitis (GPA).
  • Has received glucocorticoids (≥1 mg/kg/day) in combination with at least one immunosuppressant or biologic agent (including cyclophosphamide, rituximab, azathioprine, mycophenolate mofetil, methotrexate, leflunomide, tacrolimus, cyclosporine, belimumab, telitacicept, etc.) for at least 3 months without achieving sustained remission, or has experienced relapse after remission;or meets the criteria for severe vasculitis (Appendix 1), with inadequate response to standard-of-care therapy, and may be considered for enrollment if the investigator determines that the potential benefits outweigh the risks and the patient or legal guardian provides fully informed consent.
  • Evidence of active vasculitis meeting the following criteria: For participants <18 years of age: Pediatric Vasculitis Activity Score (PVAS) ≥10 (total score 63); For participants ≥18 years of age: Birmingham Vasculitis Activity Score (BVAS) ≥10 (total score 63); indicating active vasculitis.
  • Exclusion Criterion: Presence of alveolar hemorrhage requiring ventilatory support for more than one week.

Who cannot join

  • Subjects with known severe allergic reactions, hypersensitivity, contraindication to any medications during the trial (cyclophosphamide, tocilizumab), or subjects with a history of severe allergic reactions.
  • Uncontrollable infection, or active infection that requires systemic treatment at screening.
  • Subjects with grade III or IV heart failure (NYHA classification).
  • 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.
  • 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.
  • History of severe herpes infection prior to screening, such as herpetic encephalitis, ocular herpes, or disseminated herpes; Signs of herpes or varicella-zoster virus infection (especially chickenpox, shingles) within 12 weeks prior to screening.
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  • Patients with active central nervous system disease.
  • Patients with malignant diseases such as tumors before screening.
  • Secondary or congenital immunodeficiency.
  • History of any cardiac, endocrine, hematologic, hepatic, immunologic, metabolic, urologic, pulmonary, neurologic, dermatologic, psychiatric, and renal disease or other major medical condition that would prevent the administration of KN5601-K, except for lupus.
  • 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.
  • Received live vaccine within 4 weeks before screening.
  • Tested positive in Blood pregnancy test.
  • Patients who had participated in other clinical trials and received any intervention within 3 months before enrollment.
  • Any abnormal laboratory test results judged by the investigator to be clinically significant and prevent the subject from participating in the study. Laboratory test values that are out of range and not of clinical significance will not be considered as exclusion criteria.
  • Any situation judged by the investigators that may increase the risk of the subjects or interfere with the clinical trial outcome

Trial locations and contacts

1 site in 1 country. Contact the study team or a site near you to ask about taking part.

Jianhua Mao, Ph.D contact

13516819071

maojh88@zju.edu.cn

Qiuyu Li, Ph.D contact

17794588355

liqiuyu1992@zju.edu.cn

China1 site, 1 recruiting

  • Children's Hospital of Zhejiang University School of Medicine

    Hangzhou, Zhejiang, 310052

    Jianhua Mao, 0571-56555983, maojh88@zju.edu.cn

    Recruiting

Other trials in China

About this study

Official title: Exploratory Clinical Study on the Safety and Efficacy of Second-generation CD19/BCMA Chimeric Antigen Receptor NK Cell Injection in Treating Paediatric B Cell-related Autoimmune Diseases

Evaluating the efficacy and safety of anti-CD19/BCMA CAR-NK Cells in Paediatric B cell-related autoimmune diseases

Being tested
anti-CD19/BCMA CAR NK cells (KN5601-K)
Medical subject headings
Autoimmune Diseases

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • anti-CD19/BCMA CAR NK cells (KN5601-K)Biological

    The study adopted a dose-escalation and expansion study design. All subjects will receive fludarabine/ cyclophosphamide lymphodepletion followed by anti-CD19/BCMA CAR NK cells infusion.

Study groups in NCT07283315
Study groupRoleWhat they receive
anti-CD19/BCMA CAR NK cellsExperimentalNot described
Receives: Biological: anti-CD19/BCMA CAR NK cells (KN5601-K)

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Incidence and severity of dose-limiting toxicity (DLT) and treatment-emergent adverse events (TEAEs)

    The safety of CAR-NK cell therapy in patients with B cell mediated autoimmune disease

    Measured: 1 month

  2. response rate

    The Efficacy of CAR-NK cell therapy in patients with SLE, MDR-SRNS, IgA nephropathy, or ANCA-AAV

    Measured: 3 month

  3. CRISS score

    The Efficacy of CAR-NK cell therapy in patients with Systemic Sclerosis

    Measured: 3 month

Secondary outcomes (1)

  1. Complete response or partial response rate

    The Efficacy of CAR-NK cell therapy in patients with SLE, MDR-SRNS, IgA nephropathy, or ANCA-AAV, SSC

    Measured: 6 month

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted15 Dec 2025
  2. Study start3 Dec 2025
  3. Primary completion30 Dec 2028 (estimated)
  4. Study completion30 Dec 2029 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Early Phase 1
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
12 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT07283315
Sponsor's protocol ID
2025-SC-0007-P-01
Status last verified
Jan 2026
First submitted
1 Dec 2025
Last updated
3 Feb 2026
Results posted
No
Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A Early Phase 1 study testing anti-CD19/BCMA CAR NK cells (KN5601-K) in people with Paediatric B Cell-related Autoimmune Diseases and Autoimmune Diseases, run by The Children's Hospital of Zhejiang University School of Medicine at 1 site in China.

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 50 inclusion and 16 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: looking for participants now, as of the sponsor's last update on 3 Feb 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.

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?

Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

Contact Jianhua Mao, Ph.D (13516819071, maojh88@zju.edu.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Incidence and severity of dose-limiting toxicity (DLT) and treatment-emergent adverse events (TEAEs) (time frame: 1 month). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT07283315.

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 3 Feb 2026.

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