Rheumatic DiseasesEarly Phase 1Cell therapyNCT07490041
Exploratory Clinical Study on the Safety and Efficacy of Anti- CD19/BCMA CAR-NK Cell Injection for the Treatment of Refractory Pediatric Rheumatic Diseases
A Early Phase 1 study testing KN5601 in people with Rheumatic Diseases and Pediatric Rheumatological Condition (i.e., Arthritis, SLE, Kawasaki's Disaese), run by The Children's Hospital of Zhejiang University School of Medicine at 1 site in China.
Trial summary
A single arm, open-label pilot study is designed to determine the safety and effectiveness of anti-CD19/BCMA CAR-NK cell injection in patients with refractory pediatric rheumatic diseases. Read more about this study
- Who is it for?
- Ages 5 years and older
- Conditions the study is for
- Rheumatic Diseases, Pediatric Rheumatological Condition (i.e., Arthritis, SLE, Kawasaki's Disaese), Systemic Lupus Erythematosus (SLE)
- What is being tested?
- KN5601
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 36 participants
- Where
- 1 siteChina
- Study dates
- 19 Mar 2026to 19 Dec 2027
- Phase
- Early Phase 1
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 5 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
None listed.
Who cannot join
- . History of malignancy (except for basal cell or squamous cell skin cancer or carcinoma in situof the cervix that has been excised and cured for at least 5 years), or current malignancy.
- . Known allergy, hypersensitivity, intolerance, or contraindication to CD19/BCMA CAR-NK cells or any component of the drugs that may be used in the study (including fludarabine, cyclophosphamide, and tocilizumab), or subjects who have experienced a severe allergic reaction in the past.
- . Evidence of severe active viral or bacterial infection, or uncontrolled systemic fungal infection at screening or baseline visits, or subjects with active or uncontrolled infection requiring parenteral antimicrobial therapy.
- . Subjects with cardiac insufficiency classified as Class III or IV according to the New York Heart Association (NYHA) functional classification (see Appendix).
- . Subjects with congenital heart disease, or history of acute myocardial infarction within 6 months prior to screening, or severe arrhythmia (including multifrequent ventricular premature beats, supraventricular tachycardia, ventricular tachycardia, etc.); or combined with moderate to large pericardial effusion, severe myocarditis, etc.; or unstable vital signs requiring vasopressors to maintain blood pressure.
- . Positive hepatitis B surface antigen (HBsAg) or hepatitis B core antibody (HBcAb) with detectable hepatitis B virus (HBV) DNA levels above the normal reference range in peripheral blood at screening; OR positive hepatitis C virus (HCV) antibody with detectable HCV RNA levels above the normal reference range; OR positive human immunodeficiency virus (HIV) antibody; OR positive syphilis test; OR positive cytomegalovirus (CMV) DNA test.
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- . History of severe herpes infection, such as herpes encephalitis, ocular herpes, or disseminated herpes; signs of herpes or varicella-zoster virus infection (particularly varicella, herpes zoster) within 12 weeks prior to screening.
- . Current active tuberculosis or history of active tuberculosis, or subjects whose interferon-gamma release assay for tuberculosis infection cannot yield a negative result during the screening period.
- . Subjects with interstitial lung disease (ILD), meeting any of the following conditions are excluded: Forced vital capacity (FVC) <50% of predicted value at screening, OR diffusing capacity of the lungs for carbon monoxide (DLCO) <40% of predicted value; Requiring long-term oxygen therapy or non-invasive ventilation; Acute exacerbation of interstitial pneumonia/acute respiratory failure within the past 6 months, or hospitalization due to ILD requiring intravenous pulse corticosteroid therapy; Rapidly progressive ILD as judged by the investigator.
- . Subjects with pulmonary arterial hypertension (PAH), meeting any of the following conditions are excluded: Results from right heart catheterization or echocardiography (non-invasive estimation) meeting any of the following: a. Estimated systolic pulmonary artery pressure (sPAP) >50 mmHg (assessed in conjunction with tricuspid regurgitation velocity); b. Diagnosis of WHO functional class III or IV PAH.
- Hospitalization within the past 6 months due to acute exacerbation of PAH or right heart failure; Requiring intravenous prostacyclin analog therapy during the screening period; Presence of signs of right ventricular dysfunction, including but not limited to ascites, hepatic congestion, peripheral edema, accompanied by significantly elevated BNP/NT-proBNP levels and clinically unstable symptoms.
- . History of epilepsy or other active central nervous system diseases.
- . Subjects with acquired or congenital immunodeficiency diseases.
- . History of any clinically significant cardiac, endocrine, hematological, hepatic, immunological, metabolic, urological, pulmonary, neurological, dermatological, psychiatric, renal disease, or other major condition that, in the investigator's judgment, precludes the administration of KN5601.
- . Solid organ or hematopoietic stem cell transplantation within 3 months prior to screening; OR acute graft-versus-host disease (GVHD) of grade 2 or higher within 2 weeks prior to screening.
- . Vaccination with a live vaccine within 4 weeks prior to screening.
- . Having received the following treatments within the specified time frames prior to the baseline visit: B cell depletion therapy within 26 weeks; Within 24 weeks prior to randomization: Anti-CD40 monoclonal antibody, belimumab, abatacept, anti-tumor necrosis factor alpha (anti-TNFα) biologics, immunoglobulin, plasmapheresis; Within 12 weeks prior to randomization: JAK inhibitors or other kinase inhibitors, unless explicitly permitted by the protocol; Use of traditional Chinese medicines, proprietary Chinese medicines, or health products containing Tripterygium wilfordii(Lei Gong Teng), Tripterygium hypoglaucum(Kunming Shan Hai Tang), Colquhounia coccineavar. mollis(Huo Ba Hua Gen), or white peony root (Paeonia lactiflora, Bai Shao) within 4 weeks; Within 3 half-lives of prior therapy, OR within 4 weeks, OR until the expected pharmacodynamic effects have returned to baseline levels (whichever is longer); B cell count below the lower limit of normal or baseline value (whichever is lower) following prior B cell depletion therapy.
- . Participation in any clinical trial within three months.
- . Any other condition that, in the opinion of the investigator, may increase the risk to the subject or interfere with the trial results.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site, 1 recruiting
- Recruiting
The Children's Hospital of Zhejiang University School of Medicine
Hangzhou, Zhejiang
Yiyi Xie, Prof., 86670072, chkjk@zju.edu.cn
About this study
Official title: Exploratory Clinical Study on the Safety and Efficacy of Anti- CD19/BCMA CAR-NK Cell Injection for the Treatment of Relapsed/Refractory Pediatric Rheumatic Diseases
A single arm, open-label pilot study is designed to determine the safety and effectiveness of anti-CD19/BCMA CAR-NK cell injection in patients with refractory pediatric rheumatic diseases.
- Conditions
- Rheumatic Diseases, Pediatric Rheumatological Condition (i.e., Arthritis, SLE, Kawasaki's Disaese), Systemic Lupus Erythematosus (SLE), Connective Tissue Disease-associated Interstitial Lung Disease
- Being tested
- KN5601
- Medical subject headings
- Rheumatic Diseases, Arthritis, Lupus Erythematosus, Systemic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
KN5601Biological
Multiple doses of anti-CD19/BCMA CAR-NK cells (KN5601) will be administered using dose-escalation strategy.
| Study group | Role | What they receive |
|---|---|---|
| Experimental: KN5601 | Experimental | Patients will receive Fludarabine and Cyclophosphamide on day-5, -4, and -3. Multiple doses of anti-CD19/BCMA CAR-NK cells (KN5601) will be administered using dose-escalation strategy. Receives: Biological: KN5601 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of Dose-Limiting Toxicity (DLT)
To characterize the safety of anti-CD19/BCMA CAR NK Cells (KN3601) for patients with Refractory Pediatric Rheumatic Diseases.
Measured: up to 28 days after infusion
Secondary outcomes (2)
The proportion of subjects who achieved clinical remission, or clinical inactive status at months 3, 6, 9, and 12.
To characterize the efficacy of anti-CD19/BCMA CAR NK Cells (KN5601) for patients with Relapsed/Refractory autoimmune diseases
Measured: up to 52 weeks after infusion
B cell depletion rate
To characterize the efficacy of anti-CD19/BCMA CAR NK Cells (KN5601) for patients with Refractory Pediatric Rheumatic Diseases.
Measured: up to 52 weeks after infusion
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Mar 2026
- Study start19 Mar 2026 (estimated)
- Primary completion19 Dec 2026 (estimated)
- Study completion19 Dec 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Rheumatic DiseasesStudies of the same condition, open and under way.
- 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
- Early Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- Single Group Assignment
- Masking
- None (open label)
- Enrolment
- 36 participants (estimated)
Sponsor and investigators
- Lead sponsor
- The Children's Hospital of Zhejiang University School of Medicine
- Sponsor type
- University, hospital or other
- Collaborators
- Rui Therapeutics Co., Ltd
- Responsible party
- Sponsor
- Investigators
- Meiping Lu, Principal investigator, The Children's Hospital of Zhejiang University School of Medicine
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07490041
- Sponsor's protocol ID
- 2025-SC-0006-P-02
- Acronym
- Cell therapy
- Status last verified
- Mar 2026
- First submitted
- 18 Mar 2026
- Last updated
- 24 Mar 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No