Exploratory Clinical Study on the Safety and Efficacy of Anti- CD19/BCMA U CAR-T Cell Injection for the Treatment of Relapsed/Refractory Autoimmune Diseases

NCT07246096 | Recruiting Early Phase 1

• 1 other identifier: CHEC2025-363
• Study Type: interventional
• Target: 60
• Locations: 1 country
• Sites: 1

Brief Summary

A single arm, open-label pilot study is designed to determine the safety and effectiveness of anti-CD19/BCMA U CAR T cells in patients with autoimmune diseases. 60 patients are planned to be enrolled in the dose-escalation trial.

Conditions

Autoimmune Diseases; Systemic Lupus Erythematosus; Systemic Sclerosis; Primary Sjögren Syndrome; Rheumatoid Arthritis; Anti-Neutrophil Cytoplasmic Antibody-Associated Vasculitis

Outcome Measures

Primary Outcomes (2)

• Incidence of Dose-Limiting Toxicity (DLT)

  To characterize the safety of anti-CD19/BCMA U CAR T Cells (KN3601) for patients with Relapsed/Refractory autoimmune diseases

  up to 52 weeks after infusion

• Incidence of Treatment Emergent Adverse Events (TEAEs)

  To characterize the safety of anti-CD19/BCMA U CAR T Cells (KN3601) for patients with Relapsed/Refractory autoimmune diseases

  up to 52 weeks after infusion

Secondary Outcomes (4)

• The overall response rate (ORR)

  up to 52 weeks after infusion

• Disease control rate (DCR)

  up to 52 weeks after infusion

• B cell depletion rate

  up to 52 weeks after infusion

• B cell reconstitution

  up to 52 weeks after infusion

Study Arms (1)

KN3601

EXPERIMENTAL

Biological: anti-CD19/BCMA U CAR T cells

Interventions

anti-CD19/BCMA U CAR T cells BIOLOGICAL

Patients will receive Fludarabine and Cyclophosphamide on day-5, -4, and -3. Dose escalation will be performed for the single dose injection of anti-CD19/BCMA CAR T cells (KN3601)

KN3601

Eligibility Criteria

• Age: 18 Years - 70 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Age: ≥ 18 years old and ≤ 70 years old, male or female;
• The functions of critical organs meet the following requirements:
• a )Neutrophil count ≥ 1 x 10\^9/L, Hemoglobin ≥60g/L; b) Liver function: ALT ≤ 3 x ULN,AST≤3 x ULN, TBIL≤1.5 x ULN; c) Coagulation function: International standardized ratio (INR) ≤1.5x ULN, prothrombin time (PT) ≤1.5 x ULN; d) Cardiac function: good hemodynamic stability, left ventricular ejection fraction (LVEF) ≥50%; 3. Female subjects of childbearing potential and male subjects whose partner is a female of childbearing potential are required to use medically approved contraception or abstain from sex for at least 6 months during and at least 6 months after the end of the study treatment period; female subjects of childbearing potential have had a negativeserum HCG test within 7 days prior to study enrollment and are not lactating; 4. Voluntarily participate in this clinical study, sign an informed consent form, have good compliance, and cooperate with follow-up.
• Relapsed/Refractory Systemic lupus erythematosus
• Subject have previous diagnosis of systemic lupus erythematosus (SLE) (according to the 1997 American College of Rheumatology revised SLE classification criteria, the 2012 Systemic Lupus International Collaborating Clinics classification criteria, or the 2019 European League Against Rheumatism/American College of Rheumatology joint classification criteria);
• Subjects had a modified SLEDAI-2K score of ≥8 at screening;
• Subject has ≥ 1 organ system with BILAG-2004 Class A mobility score or ≥ 2 organ systems with BILAG-2004 Class B mobility score at screening;
• Based on the results of the central laboratory tests at screening, the subject meets one of the following: a. ANA by immunofluorescence ≥ 1:80 b. Anti-dsDNA antibodies above the normal level c. Anti-Smith antibodies above the normal level.
• Relapsed/Refractory Systemic Sclerosis
• Meets 2013 ACR classification criteria for systemic sclerosis;
• If combined with interstitial pneumonia, interstitial changes suggestive of ground-glass exudates on chest HRCT and FVC or DLCO \<70% predictive value on pulmonary function tests;
• Ineffective conventional treatment or relapse of disease activity after remission. Definition of routine treatment: Use of glucocorticoids (above 1mg/Kg/d) and cyclophosphamide, as well as any of the following immunomodulatory drugs for more than 6 months: antimalarials,
• methotrexate, leflunomide, cyclophosphamide, azathioprine, mertiomate, tacrolimus, cyclosporine, and biologics, including rituximab, belimumab and tetracycline;
• Definition of progressiveness; 1) Definition of cutaneous progression: increase in mRSS \>10%; 2) Definition of lung disease progression: 10% decrease in FVC or 5% decrease in FVC with 15% decrease in DLCO (OMERACT progression).
• Relapsed/Refractory Primary Sjögren's Syndrome

You may not qualify if:

• Subjects with known severe allergic reactions, hypersensitivity, contraindication to any medications during the trial (cyclophosphamide, fludarabine, tozumabs), or subjects with a history of severe allergic reactions;
• Existence or suspicion of uncontrollable or treatable fungal, bacterial, viral or other infections;
• Subjects with central nervous system disorders caused by ADs or not caused by ADs (including epilepsy, psychiatric disorders, organic encephalopathy syndromes, cerebrovascular accidents, encephalitis, central nervous system vasculitis);
• Subjects s with relatively serious heart diseases, such as angina pectoris, myocardial infarction, heart failure, and arrhythmia;
• Subjects with congenital immunoglobulin deficiency;
• Subjects with malignant tumors (except for non-melanoma skin cancer and in situ cervical, bladder, and breast cancers that have been disease free for more than 5 years);
• Subjects with end-stage renal failure;
• Subjects with positive hepatitis B surface antigen (HBsAg) or hepatitis,B core antibody (HBcAb) and HBV DNA titer in peripheral blood higher than the upper limit of detection; Patients with positive hepatitis C virus (HCV) antibodies and positive peripheral blood HCV RNA; People who are positive for human immunodeficiency virus (HIV) antibodies; Those who have tested positive for syphilis;
• Subjects with mental illness and severe cognitive impairment;
• Subjects who have received other clinical trial treatment within 6 months;
• Pregnant or intending to conceive women;
• Subjects with hypertension or diabetes that cannot be controlled by medication;
• In the opinion of the investigator, there are other reasons that prevent some subjects from being included in this study.
• Relapsed/Refractory Systemic lupus erythematosus
• Except for SLE, any clinically significant history of cardiac, endocrine, blood, liver, immune, metabolic, urinary, pulmonary, neurological, skin, psychiatric, or renal disorders, or other major diseases that may interfere with the administration of KN3601 (as determined by the investigator);

Sponsors & Collaborators

• Changhai Hospital: lead
• Rui Therapeutics Co., Ltd: collaborator

Study Sites (1)

Changhai Hospital

Shanghai, China

RECRUITING

MeSH Terms

Conditions

Autoimmune Diseases; Lupus Erythematosus, Systemic; Scleroderma, Systemic; Arthritis, Rheumatoid; Anti-Neutrophil Cytoplasmic Antibody-Associated Vasculitis

Condition Hierarchy (Ancestors)

Immune System Diseases; Connective Tissue Diseases; Skin and Connective Tissue Diseases; Skin Diseases; Arthritis; Joint Diseases; Musculoskeletal Diseases; Rheumatic Diseases; Systemic Vasculitis; Vasculitis; Vascular Diseases; Cardiovascular Diseases; Skin Diseases, Vascular

Central Study Contacts

Dongbao Zhao

CONTACT

+86-15921061314; dongbaozhao@163.com

Study Design

• Study Type: interventional
• Phase: early phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 14, 2025
• First Posted: November 24, 2025
• Study Start: February 20, 2026
• Primary Completion (Estimated): July 20, 2027
• Study Completion (Estimated): July 20, 2028
• Last Updated: February 25, 2026

Record last verified: 2025-09

Locations

CN (1)