Draft-Version-001-Safety and Efficacy of CD19 CAR-T Therapy for Recurrent/Refractory Autoimmune Diseases

  • STATUS
    Recruiting
  • End date
    Sep 30, 2027
  • participants needed
    12
  • sponsor
    Beijing Boren Hospital
Updated on 30 May 2025

Summary

Draft-Version-001- 

This study is a single center, open label exploratory clinical trial aimed at evaluating the safety and efficacy of universal CD19 CAR-T therapy in subjects with autoimmune diseases.

The study will adopt the traditional dose escalation model "3+3" design, setting up three dose groups with a starting dose of 0.5 × 106 CAR+T cells/kg for incremental DLT observation. Safety and efficacy follow-up will be conducted for 24 months after cell infusion to observe the safety of the universal CD19 CAR-T and conduct preliminary efficacy evaluation.

Description

Draft-Version-001-

Details
Condition Autoimmune Disease
Age 18years - 65years
Clinical Study IdentifierNCT06983964
SponsorBeijing Boren Hospital
Last Modified on30 May 2025

Eligibility

Yes No Not Sure

Inclusion Criteria

Participants must meet all of the following inclusion criteria in order to be included in this study
Age range of 18 to 65 years old (including 18 and 65 years old), gender is not limited
Special criteria for different indications
Subjects with relapsed/refractory moderate to severe SLE must meet the following criteria
According to the 2019 European League Against Rheumatism (EULAR)/American College of Rheumatology (ACR) SLE classification criteria, the diagnosis is SLE
Positive for anti nuclear antibody (ANA) (titer ≥ 1:80), and/or positive for anti dsDNA antibody, and/or positive for anti Sm antibody during screening
The definition of moderate to severe activity is: SLEDAI-2000 score ≥ 8 during screening; If there is a low complement and/or anti dsDNA antibody score, the clinical symptom score for SLEDAI-2000 (excluding low complement and/or anti dsDNA antibodies) must be ≥ 6 points
At least 6 months of stable standard treatment have been used in the SLE history prior to screening, and the condition remains active for at least 2 months prior to screening. The standard treatment plan refers to the stable use of any of the following drugs (used alone or in combination): glucocorticoids (≤ 20mg/day prednisone or equivalent), antimalarials (hydroxychloroquine ≤ 400mg/day, chloroquine ≤ 500mg/day), nonsteroidal anti-inflammatory drugs (NSAIDs), biologics (rituximab, belimumab, tacept), and other immunosuppressants or immunomodulators, including mycophenolate mofetil (≤ 2g/day), azathioprine (≤ 2mg/kg/day), methotrexate (≤ 20mg/week), etc
Recurrent/refractory dcSSc subjects must meet the following criteria
According to the 2013 EULAR/ACR classification criteria for systemic sclerosis (SSc), the diagnosis is SSc; 2) According to the definition criteria of LeRoy et al. in 1988, it meets the diffuse skin type presentation, which refers to extensive skin fibrosis and involvement of the skin near the elbow and/or knee
When screening, interstitial lung disease (ILD) should be included, and 45% of the predicted values should be ≤ forced vital capacity (FVC) ≤ 70% of the predicted values, or 40% of the predicted values should be ≤ diffusion capacity of carbon monoxide (DLCO) ≤ 70% of the predicted values
Relapse/refractory is defined as: recurrence after previous ineffective conventional treatment or disease remission. Conventional treatment refers to the use of glucocorticoids, cyclophosphamide, and at least one immunosuppressive/regulatory drug for at least 6 months. Immunosuppressive/regulatory drugs include azathioprine, mycophenolate mofetil, methotrexate, leflunomide, tacrolimus, cyclosporine, rituximab, belimumab, tacrolizumab, etc
Activity is defined as having at least one of the following
There is evidence of skin progression during screening, that is, an increase
of ≥ 10% in mRSS scores within the past 6 months
During screening, there is evidence of any of the following ILD activities
Newly diagnosed ILD within the past 6 months; Previous ILD subjects had a 10%
decrease in FVC or a 5% decrease in FVC accompanied by a 15% decrease in DLCO
within the past 6 months
Recurrent/refractory AAV subjects must meet the following criteria
According to the definition of the 2012 Chapel Hill Consensus Conference (CHCC), clinical diagnosis is granulomatous vasculitis (GPA) and microscopic polyangitis (MPA)
In Birmingham Vasculitis Activity Score (BVAS) version 3, at least one primary item or at least three other items
During screening, antibodies against PR3-ANCA or MPO-ANCA were positive
The definition of recurrence/refractory is
Recurrent AAV subjects: those who have received initial efficacy (BVAS score 0
and glucocorticoid dose ≤ 7.5mg/d prednisone or other equivalent
glucocorticoid drugs) after at least 3 months of combined glucocorticoid and
immunosuppressive therapy (cyclophosphamide, rituximab, azathioprine
methotrexate, mycophenolate mofetil, etc.), and have experienced at least 1
disease recurrence (recurrence defined as the appearance of at least one
important item in BVAS assessment, or at least 3 other items, or 1-2 new items
in two consecutive assessments), and have experienced disease recurrence
within 12 weeks before screening; Difficult to treat AAV subjects: Despite at
least 3 months of previous treatment with corticosteroids combined with
immunosuppressants (cyclophosphamide, rituximab, azathioprine, methotrexate
mycophenolate mofetil, etc.), no therapeutic effect has been achieved (BVAS
score 0 and glucocorticoid dose ≤ 7.5mg/d prednisone or other equivalent
glucocorticoid drugs)
Recurrent/refractory IIM subjects must meet the following criteria
According to the 2017 EULAR/ACR IIM classification criteria, the probability of being diagnosed with IIM is ≥ 55%, and based on factors such as age of first onset, skin and muscle strength, laboratory tests, and muscle biopsy characteristics, it is classified as dermatomyositis (DM), polymyositis (PM), or immune-mediated necrotizing myopathy (IMNM)
Disease activity/severity meets the following criteria
Manual muscle strength check-8 (MMT-8) score ≤ 141 (total score of 150)
Meet at least 2 of the anomalies in other CSMs
The overall disease activity evaluated by the patient [based on Visual Analog
Scale (VAS)] score is ≥ 2 points (range 0-10 points); The overall disease
activity VAS score evaluated by physicians is ≥ 2 points (range 0-10 points)
The overall extramuscular disease activity VAS score evaluated by the
physician is ≥ 2 points (range 0-10 points); The Health Assessment
Questionnaire Disability Index (HAQ-DI) score evaluated by physicians is ≥
25 (range 0-3 points)
At least one muscle enzyme level is greater than 1.5 times the upper limit of
normal (ULN)
Recurrent/refractory SS subjects must meet the following criteria
According to the 2016 EULAR/ACR SS classification criteria, the diagnosis is SS
Positive for anti Ro/SSA antibodies during screening
Dynamic saliva total flow rate ≥ 0.05 mL/min or static saliva total flow rate ≥ 0.01 mL/min during screening
Activity is defined as a Dry Syndrome Disease Activity Index (ESSDAI) score of ≥ 5 across 8 sub items
For autoimmune diseases that are intolerant to or have insufficient efficacy in the treatment of glucocorticoids and at least one other immunosuppressant or modulator in the past, it is required to be treated with glucocorticoids and at least two immunosuppressants (azathioprine, methotrexate, mycophenolate mofetil, etc.) at known effective doses for at least 3 months
Having good organ function
Blood routine: Absolute neutrophil count (ANC) ≥ 0.5 × 109/L, platelet count (PLT) ≥ 20 × 109/L, hemoglobin ≥ 60 g/L
Coagulation function: International normalized ratio (INR) ≤ 1.5 times ULN, and activated partial thromboplastin time (APTT) ≤ 1.5 times ULN
Liver function: serum aspartate aminotransferase (AST), alanine aminotransferase (ALT) ≤ 3 times the upper limit of normal (ULN), total bilirubin ≤ 1.5 times ULN
Renal function: serum creatinine ≤ 1.5 × ULN or creatinine clearance rate (Cockcroft Gault formula) ≥ 30 mL/min
Cardiac function: NYHA grade I or II, with an echocardiogram (ECHO) left ventricular ejection fraction (LVEF) ≥ 50% and no pericardial effusion; There were no clinically significant abnormal findings in the 12 lead electrocardiogram (ECG)
Pulmonary function: Under indoor ventilation conditions, the oxygen saturation in non oxygenated state is ≥ 92%; No clinically significant pleural effusion
Expected survival period greater than 6 months
Subjects with reproductive partners agree to use effective contraceptive measures throughout the entire treatment period and for 24 months after treatment, and are not allowed to donate eggs/sperm for assisted reproduction during this period; Female subjects of childbearing age (those who have undergone sterilization surgery or have been menopausal for ≥ 12 months are not considered fertile) with negative urine pregnancy or blood pregnancy tests during the screening period; Voluntarily participate in this experiment and sign the informed consent form

Exclusion Criteria

Subjects who meet any of the following criteria are not eligible for inclusion in this study
Patients with previous or concurrent active malignant tumors, including tumor associated polymyositis/dermatomyositis. Cervical carcinoma in situ that has been cured or has not recurred for at least 2 years, non-invasive basal cell or squamous cell skin cancer, locally treated prostate cancer, and ductal carcinoma in situ after radical surgery are excluded
Within the past 3 months, severe pulmonary diseases such as moderate to severe pulmonary arterial hypertension (average pulmonary arterial pressure detected by echocardiography>60 mmHg) require oxygen mask therapy or non-invasive or invasive ventilator assisted breathing during screening; During screening, IgA, IgG, and IgM levels were below the lower limit of normal (LLN)
Patients who have used any of the following medications or treatments within the specified time
Individuals who have used B-cell depletion therapy within the previous month and have not failed treatment as assessed by clinical physicians, including those targeting CD20, CD22, CD52, CD38, BCMA monoclonal antibodies, or bispecific antibodies
Screening for excessive use of human intravenous immunoglobulin within the previous month
Use therapeutic doses of corticosteroids (prednisone ≥ 20mg/day or equivalent doses of other corticosteroids) within 24 hours prior to Qinglin pretreatment
Received corticosteroid shock therapy within 2 weeks (defined as a dose ≥ 500mg/d of prednisone or equivalent dose of other corticosteroids)
Received tacrolizumab within 2 weeks prior to screening, or received belimumab within 3 weeks
Patients with a history of severe central nervous system diseases or related symptoms (excluding simple trigeminal nerve disease) within the past 6 months, including but not limited to: lupus encephalopathy, cerebrovascular disease, encephalitis, brain injury, aneurysm, cerebellar disease, organic brain syndrome, Parkinson's disease and other central nervous system diseases, as well as epilepsy, seizures, aphasia, dementia and other symptoms
Individuals who have experienced lupus crisis within the previous 3 months, such as active central nervous system lupus, severe hemolytic anemia, severe thrombocytopenic purpura, severe neutropenia, severe myocardial injury, severe lupus pneumonia or pulmonary hemorrhage, severe lupus hepatitis, severe vasculitis, etc
Concurrent severe kidney disease: severe lupus nephritis (defined as urinary protein>4g/24 hours or serum creatinine>1.5 × ULN or creatinine clearance rate (Cockcroft Gault formula)<30 mL/min) within the first 8 weeks of screening, Or active nephritis that requires treatment with drugs prohibited by the protocol, or requires treatment with prednisone greater than 500mg/day or equivalent corticosteroids for ≥ 14 days
Individuals who have severe allergies to any components of the Qinglin pre-treatment drug and the universal CD19 CAR-T used in this study
hepatitis B surface antigen (HBsAg) positive and peripheral blood HBV DNA positive; Individuals with positive hepatitis C virus (HCV) antibodies and positive HCV RNA; Positive for Treponema pallidum antibody; HIV antibody positive individuals
There are uncontrolled fungi, bacteria, viruses, or other infections that researchers have assessed as unsuitable for participation in the study
History of major organ transplantation (such as heart and lung)
During screening, there is active tuberculosis or latent tuberculosis (referring to whether the tuberculin skin test or interferon test is positive, and there are no clinical symptoms or imaging evidence)
If any of the following cardiovascular diseases (including but not limited to) have occurred within the 6 months prior to screening
Congestive heart failure, myocardial infarction, unstable angina, coronary angioplasty, stent implantation, coronary artery/peripheral artery bypass grafting
Serious arrhythmias that require treatment (such as persistent ventricular tachycardia, ventricular fibrillation, apical torsion ventricular tachycardia, etc.); Congenital long QT syndrome, left anterior half block (double bundle block), allowing asymptomatic right bundle branch block to be included in the study
Uncontrolled hypertension (systolic blood pressure greater than 160 mmHg and/or diastolic blood pressure greater than 100 mmHg), history of hypertensive crisis or hypertensive encephalopathy
In addition to the target indication, patients with a history of other autoimmune diseases (including but not limited to eosinophilic granulomatous polyangitis, cryoglobulinemia vasculitis, inclusion body myositis, anti glomerular basement membrane disease, Behcet's disease or Takayasu arteritis, etc.) who require systematic treatment
Individuals with a family history of non IIM such as drug-induced myopathy or human immunodeficiency virus related myopathy
Pregnant or lactating women
Those who have received a live vaccine within 6 weeks before the pre-treatment of Qinglin
Participants who have participated in other interventional clinical studies, received active investigational drug treatment, or intended to participate in another clinical trial or receive treatment for autoimmune diseases outside of the protocol within 3 months prior to signing the informed consent form during the entire study period
Mental illness patients with depression or suicidal tendencies; The researchers believe that there are other factors that are not suitable for inclusion or affect the participation or completion of the study by the subjects
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