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Study of CD19 CAR-T Therapy for Refractory SLE
Bu Faz 1 (Phase 1), araştırmacı tarafından başlatılan çalışma, dirençli (refractory) sistemik lupus eritematozus (systemic lupus erythematosus, SLE) tedavisinde CD19 hedefli CAR-T hücre ürünü PTOC1 enjeksiyonunun güvenliğini ve etkinliğini değerlendiriyor. Katılımcıların en az 5 yaşında olması, 2019 EULAR/ACR kriterlerine göre SLE tanısı alması ve en az 3 ay yüksek doz kortikosteroid (glucocorticoid), hidroksiklorokin ve en az 2 bağışıklık baskılayıcı ya da biyolojik ilaç tedavisine rağmen hastalığın orta-ağır düzeyde aktif kalması gerekmektedir.
Yukarıdaki özet, resmî kaydın sade dile aktarılmış halidir. Orijinal İngilizce başlık referans için gösterilir.
Kriterler ClinicalTrials.gov kaydından orijinal İngilizce haliyle alınmıştır. Uygunluk kararını yalnızca deneyi yürüten ekip verebilir.
Inclusion Criteria: * Age ≥ 5 years, no gender limitation; * Diagnosed with SLE according to the 2019 EULAR/ACR classification criteria, and still in moderate to severe disease activity despite ≥ 3 months of high dose glucocorticoids(prednisone≥1mg/kg/d or other equivalent amount of other steroid), combined with hydroxychloroquine, and at least 2 Immunosuppressants or biologics (including cyclophosphamide, mycophenolate mofetil, azathioprine, methotrexate, cyclosporin, tacrolimus, sirolimus, leflunomide, telitacicept, belimumab, and rituximab) or intolerant to standard treatments; * SLEDAI-2K score ≥ 8 points; * The functions of vital organs must meet the following requirements: 1. cardiac function: left ventricular ejection fraction (LVEF) ≥ 50%, with no obvious abnormalities on electrocardiogram (ECG); 2. renal function: eGFR ≥ 30 mL/min/1.73m2; 3. hepatic function: AST and ALT ≤ 3.0×ULN, total bilirubin ≤ 2.0×ULN; 4. pulmonary function: no severe pulmonary lesions; blood oxygen saturation ≥ 92% under non-oxygen supplementation conditions. * Meet the criteria of leukapheresis or intravenous blood collection, and no contraindication for leukapheresis; * Negative pregnancy test for female subjects of childbearing age, and agree to take effective contraceptive measures until one year after infusion; * Participant or his/her guardians agree to participate in the clinical trial and sign the informed consent form indicating that he/she understands the purpose and procedure of the clinical trial and is willing to participate in the study. Exclusion Criteria: * Central nervous system (CNS) diseases: presence of CNS lupus symptoms requiring intervention within 60 days, including epilepsy, confusion, cerebrovascular events, etc; * Congenital heart disease or severe arrhythmia before screening: Including multifocal and frequent supraventricular tachycardia, ventricular tachycardia, etc.; or complicated with moderate to large pericardial effusion, severe myocarditis, etc.; or patients with unstable vital signs who require vasopressors to maintain blood pressure; * Presence of active infections requiring systemic treatment or uncontrolled infections within 3 months prior to screening; * Having received solid organ transplantation or hematopoietic stem cell transplantation within 3 months prior to screening; or having grade 2 or above acute graft-versus-host disease (GVHD) within 2 weeks prior to screening; * Positive for hepatitis B surface antigen (HBsAg) or hepatitis B core antibody (HBcAb) with peripheral blood hepatitis B virus (HBV) DNA level exceeding the normal range; positive for hepatitis C virus (HCV) antibody with peripheral blood hepatitis C virus (HCV) RNA level exceeding the normal range; positive for human immunodeficiency virus (HIV) antibody; positive for treponema pallidum antibody; * History of macrophage activation syndrome within 1 month prior to screening (except for those for whom the investigator has determined that safety risks are excluded after treatment); * History of previous CAR-T therapy (except for those for whom the investigator has determined that safety risks are excluded after treatment); * Presence of active pulmonary tuberculosis at the time of screening; * Having received any vaccination within 4 weeks prior to screening; * Positive result of blood pregnancy test; * A confirmed diagnosis of malignant diseases such as tumors prior to screening; * Participation in other clinical trials within 3 months prior to enrollment; * Other circumstances that the investigator deems inappropriate for participation in this study.