Recruiting EARLY_PHASE1 Rheumatic Diseases

Tests treatment safety and results for Rheumatic Diseases

Official title Exploratory Clinical Study on the Safety and Efficacy of Anti- CD19/BCMA CAR-NK Cell Injection for the Treatment of Refractory Pediatric Rheumatic Diseases

ClinicalTrials.gov ID: NCT07490041

What this study is testing

What is KN5601?

KN5601 is an investigational medicine, given as an injectable medicine, being studied as a potential treatment for rheumatic diseases.

Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.

What it's testing
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.
  • Phase 1: an early, usually small safety study

A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.

Who can take part

Ages 5 and older

You may be able to join if

  • Common
  • . Gender unrestricted, age ≥5 years;
  • . The patient or their legal guardian agrees to participate in this clinical trial and signs the informed consent form, indicating their...
  • . Peripheral blood B cells confirmed by flow cytometry to express CD19, with a B cell count \>5 cells/uL;
  • . If previously treated with B cell-targeted therapy, peripheral blood B cell count at screening has returned to normal or above the pre-treatment...

You likely can't join if

  • . 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...
  • . Known allergy, hypersensitivity, intolerance, or contraindication to CD19/BCMA CAR-NK cells or any component of the drugs that may be used in the...
  • . Evidence of severe active viral or bacterial infection, or uncontrolled systemic fungal infection at screening or baseline visits, or people with...
  • . people with cardiac insufficiency classified as Class III or IV according to the New York Heart Association (NYHA) functional classification (see...
  • . people with congenital heart disease, or history of acute myocardial infarction within 6 months prior to screening, or severe arrhythmia (including...
  • . Positive hepatitis B surface antigen (HBsAg) or hepatitis B core antibody (HBcAb) with detectable hepatitis B virus (HBV) DNA levels above the...
See the full eligibility criteria
Who can join
  • Common
  • . Gender unrestricted, age ≥5 years;
  • . The patient or their legal guardian agrees to participate in this clinical trial and signs the informed consent form, indicating their understanding of the trial's purpose and procedures and willingness to participate;
  • . Peripheral blood B cells confirmed by flow cytometry to express CD19, with a B cell count \>5 cells/uL;
  • . If previously treated with B cell-targeted therapy, peripheral blood B cell count at screening has returned to normal or above the pre-treatment level;
  • . Echocardiography indicates basically normal cardiac structure and left ventricular ejection fraction (LVEF) ≥55%; electrocardiogram shows no significant abnormalities;
  • . Liver function: aspartate aminotransferase (AST) and alanine aminotransferase (ALT) ≤3.0×ULN, total bilirubin (TBIL) ≤2.0×ULN;
  • . Renal function: estimated glomerular filtration rate (eGFR) ≥30 mL/min/1.73m²; (If eGFR \<30 mL/min/1.73m² and/or undergoing renal replacement therapy, the subject may be considered for enrollment after the...
  • . Pulmonary function: No severe pulmonary lesions, blood oxygen saturation (SpO₂) ≥92%;
  • . Female people of childbearing potential must have a negative urine pregnancy test and agree to use effective contraception during the trial until 1 year after infusion. Polyarticular Juvenile Idiopathic Arthritis...
  • . Onset before age 16, disease duration ≥6 weeks, diagnosed as polyarticular JIA according to the 2001 International League of Associations for Rheumatology (ILAR) classification criteria, and positive for rheumatoid...
  • . Judged by the investigator to have active disease despite adequate standard-dose treatment prior to screening, meeting the following adequacy of treatment conditions: a. Treatment with conventional disease-modifying...
  • . Juvenile Arthritis Disease Activity Score-27 (JADAS-27) \>8.5;
  • . Must have at least 5 active joints (defined as the presence of joint swelling; or in the absence of swelling, the presence of limited range of motion accompanied by pain on motion and/or tenderness) as per the...
  • . No occurrence of macrophage activation syndrome within 1 month prior to screening. Sjögren's Syndrome (SS):
  • . Diagnosed with childhood-onset primary SS at least 24 weeks prior to signing the ICF, according to the 2002 American-European Consensus Group (AECG) classification criteria / 2016 EULAR/ACR classification criteria and...
  • . Meet the classification criteria for SS, and Intolerance or inadequate response to glucocorticoids (prednisone 1-2 mg/kg/day or equivalent doses of other corticosteroids) and at least 2 immunosuppressants, with a...
  • . EULAR Sjögren's Syndrome Disease Activity Index (ESSDAI) score ≥5 in at least 1 of the following 8 domains at screening: constitutional, lymphadenopathy, glandular, articular, cutaneous, renal, hematological, and...
  • . EULAR Sjögren's Syndrome Patient Reported Index (ESSPRI) score ≥5 at screening;
  • . Positive for anti-SSA/Ro antibody. Juvenile Dermatomyositis (JDM):
  • . Diagnosed with JDM at least 24 weeks prior to signing the ICF according to the 2017 EULAR/ACR classification criteria;
  • . Meet the classification criteria for Refractory JDM (RJDM), and had Intolerance or inadequate response to glucocorticoids (prednisone 1-2 mg/kg/day or equivalent doses of other corticosteroids) and at least 2...
  • . Patients with anti-synthetase syndrome who are anti-synthetase antibody positive and meet the criteria for RJDM can be directly enrolled;
  • . Patients with immune-mediated necrotizing myopathy who are signal recognition particle (SRP) or 3-hydroxy-3-methylglutaryl-coenzyme A reductase (HMGCR) antibody positive and meet the criteria for RJDM can be directly...
  • . Meet the 2013 ACR/EULAR classification criteria for SSc, with first non-Raynaud's phenomenon occurring at age \<18 years and disease duration ≤60 months;
  • . Positive for antinuclear antibody (ANA) or any SSc-specific antibody;
  • . Modified Rodnan Skin Score (mRSS) ≥15 (total score 51);
  • . Meet the definition of treatment-refractory disease: inadequate response to glucocorticoids (≥0.5 mg/kg/day) and cyclophosphamide and at least 1 other immunomodulatory drug for over 3 months;
  • . Diagnosed with refractory UCTD-ILD: UCTD typically refers to patients with symptoms and signs suggestive of CTD and serologic evidence of autoimmunity, but not fulfilling classification criteria for any defined CTD...
  • After treatment with glucocorticoids (≥1 mg/kg/day prednisone or equivalent) and one or more immunomodulators (including cyclophosphamide, MMF, azathioprine, methotrexate, cyclosporine, tacrolimus, sirolimus...
  • Patients intolerant to conventional therapy may be considered for enrollment after the investigator judges benefit outweighs risk and with full informed consent from the patient/guardian;
  • OR inability to taper glucocorticoids to ≤5 mg/day after 6 months (6M) of conventional therapy; (3). Systemic Lupus Erythematosus Disease Activity Index 2000 (SLEDAI-2K) score \>6; (4). No occurrence of macrophage...
What rules you out
  • . 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...
  • . Evidence of severe active viral or bacterial infection, or uncontrolled systemic fungal infection at screening or baseline visits, or people with active or uncontrolled infection requiring parenteral antimicrobial...
  • . people with cardiac insufficiency classified as Class III or IV according to the New York Heart Association (NYHA) functional classification (see Appendix).
  • . people 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...
  • . 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...
  • . 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...
  • . Current active tuberculosis or history of active tuberculosis, or people whose interferon-gamma release assay for tuberculosis infection cannot yield a negative result during the screening period.
  • . people 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...
  • . people 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...
  • . History of epilepsy or other active central nervous system diseases.
  • . people 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...
  • . 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...
  • . 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.

The study team makes the final eligibility decision.

Where it's taking place

  • Hangzhou, Zhejiang, China

Questions & answers

Do participants get paid in this trial?

This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.

Is it free to join, and do I need insurance?

Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.

How long does this study last?

The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.

Who can join this trial?

This study is enrolling all sexes, 5 years and older. The study team makes the final eligibility decision.

Where is this trial taking place?

Study sites include Hangzhou, Zhejiang, China. Enter your location above to see the nearest site and check your eligibility.

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BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.