肿瘤细胞治疗研究
英文原题：Clinical Study on the Safety and Efficacy of BCMA-CART±ASCT in Treating Young Multiple Myeloma Patients
Clinical Study on the Safety and Efficacy of BCMA-CART±ASCT in Treating Young Multiple Myeloma Patients
这是一项 I/II 期注册临床试验，评估 CAR-T 细胞治疗多发性骨髓瘤的安全性、可行性及初步疗效。当前状态：招募中。计划入组 50 例。试验地点：中国 · 天津（共 1 个中心，其中中国 1 个）。登记号：NCT07169500。
不限性别 · ≥ 18 Years 且 ≤ 50 Years
纳入标准：年轻女性，18–55岁。 1. 受试者本人或通过法定监护人自愿参加研究并签署知情同意书（ICF）； 2. 流式细胞术或免疫组化确诊多发性骨髓瘤； 3. 器官功能充分并符合以下全部检测标准：总胆红素（TBIL）≤1.5×ULN；血清ALT和AST≤2.5×ULN；按Cockcroft-Gault公式计算的肌酐清除率（CrCl）≥40 mL/min；PT≤1.5×ULN，APTT<1.5×ULN，INR<1.5×ULN；血红蛋白≥60 g/L；中性粒细胞绝对计数（ANC）≥1.0×10⁹/L（筛查实验室检查前7天内未接受G-CSF或其他生长因子）；淋巴细胞绝对计数（ALC）≥0.5×10⁹/L；血小板≥50×10⁹/L（筛查实验室检查前7天内未输注血小板）；左心室射血分数（LVEF）≥45%；血氧饱和度（SpO₂）≥92%； 4. ECOG评分0–1分（见附录5）； 5. 预期生存期≥3个月； 6. 有生育能力的女性受试者妊娠检测须阴性且不哺乳；有生育能力的女性或男性须在细胞输注后24个月内使用有效避孕方法或器具。 排除标准： 1. 对细胞产品任一成分有过敏史； 2. 严重心脏病，包括但不限于：签署ICF前6个月内发生心肌梗死、接受冠状动脉成形术或支架植入；不稳定型心绞痛；严重心律失常；严重非缺血性心肌病史；充血性心力衰竭（NYHA III或IV级，评分见附录2）； 3. 签署ICF前6个月内发生卒中或癫痫； 4. 自身免疫性疾病、免疫缺陷，或其他需要免疫抑制治疗的情况； 5. 签署ICF前3年内患有多发性骨髓瘤以外的恶性肿瘤，以下情况除外：完全治疗的宫颈原位癌、皮肤基底细胞癌或鳞状细胞癌、根治术后的局限性前列腺癌、根治术后的乳腺导管原位癌，以及其他部位根治术后满1年的原位癌；筛查期间不得接受持续治疗且不得有复发迹象； 6. 存在未控制的活动性感染； 7. 研究者判断存在不稳定的全身性疾病，包括但不限于需药物治疗的严重肝、肾或代谢性疾病； 8. 淋巴细胞采集前1周内，出现以下任一结果：外周血HBV DNA高于检测限；HCV抗体及外周血HCV RNA阳性；HIV抗体阳性；梅毒抗原或抗体阳性；CMV-DNA阳性； 9. 淋巴细胞采集前1周内使用泼尼松>5 mg/日或等效剂量其他皮质类固醇； 10. 既往接受过任何CAR-T产品或其他基因修饰T细胞治疗； 11. 既往接受过BCMA靶向治疗； 12. 签署ICF前4周内接种活疫苗； 13. 有酗酒、药物滥用或精神障碍史；研究者认为不适合参加研究的其他情况。
Inclusion Criteria： Young female, 18-55 years old; 1. Subjects voluntarily participate in the study and sign the informed consent form (ICF) themselves or through their legal guardian; 2. Confirmed diagnosis of multiple myeloma through flow cytometry or immunohistochemistry; 3. Subjects must have adequate organ function and meet all of the following test results: * Serum total bilirubin (TBIL) ≤ 1.5 times the upper limit of normal (ULN) * Serum alanine aminotransferase (ALT) and aspartate aminotransferase (AST) ≤ 2.5 × ULN * Creatinine clearance (CrCl) (Cockcroft-Gault formula) ≥ 40 ml/min * Prothrombin time (PT) ≤ 1.5 × ULN, activated partial thromboplastin time (APTT) \< 1.5 × ULN, international normalized ratio (INR) \< 1.5 × ULN * Hemoglobin (Hb) ≥ 60 g/L * Absolute neutrophil count (ANC) ≥ 1.0 × 10\^9/L (no granulocyte colony-stimulating factor \[G-CSF\] or other growth factors received within 7 days prior to screening laboratory tests) * Absolute lymphocyte count (ALC) ≥ 0.5 × 10\^9/L * Platelets (PLT) ≥ 50 × 10\^9/L (no platelet transfusion received within 7 days prior to screening laboratory tests) * Left ventricular ejection fraction (LVEF) ≥ 45% * Blood oxygen saturation (SpO2) ≥ 92% 4. ECOG score of 0-1, see Appendix 5 for ECOG scoring; 5. Expected survival ≥ 3 months; 6. Female participants of childbearing potential must have a negative pregnancy test and not be breastfeeding; female or male participants of childbearing potential must use effective contraceptive methods or devices for 24 months after cell infusion. Exclusion Criteria: 1. History of allergy to any component of the cellular product; 2. Severe heart disease, including but not limited to: * Myocardial infarction, coronary angioplasty, or stent implantation within 6 months prior to signing the ICF * Unstable angina * Severe arrhythmia * History of severe non-ischemic cardiomyopathy * Congestive heart failure (New York Heart Association \[NYHA\] class III or IV), NYHA scores are in Appendix 2 3. Stroke or seizure within 6 months prior to signing the ICF; 4. Autoimmune diseases, immunodeficiency, or other conditions requiring immunosuppressive therapy; 5. Malignant tumors other than multiple myeloma within 3 years prior to signing the ICF, except fully treated cervical carcinoma in situ, basal cell or squamous cell skin cancer, localized prostate cancer after radical surgery, ductal carcinoma in situ of the breast after radical surgery, and other in situ cancers at other sites one year after radical surgery, provided there is no ongoing treatment and no signs of recurrence during the screening period; 6. Presence of uncontrolled active infection; 7. Unstable systemic diseases as judged by the investigator, including but not limited to severe liver, kidney, or metabolic diseases requiring medication. 8. Within one week before lymphocyte collection, the storage device falls under any of the following conditions: * Peripheral blood hepatitis B virus (HBV) DNA test value is above the detection limit * Hepatitis C virus (HCV) antibody positive and peripheral HCV-RNA positive * Human immunodeficiency virus (HIV) antibody positive * Syphilis antigen or antibody positive * CMV-DNA positive 9. Within one week before lymphocyte collection, use of more than 5 mg/day of prednisone (or an equivalent dose of other corticosteroids); 10. Prior use of any CAR-T cell products or other genetically modified T cell therapies; 11. Prior BCMA-targeted therapy; 12. Vaccination with a live vaccine within 4 weeks before signing the ICF; 13. History of alcoholism, drug abuse, or psychiatric disorders; Other conditions that the investigator deems unsuitable for participation in this study.
以上为辅助阅读译文。是否适合入组须由主治医生判断，最终以登记平台与研究者确认为准。
主要终点：Assessment and comparison of MRD negativity rate 3 months after BCMA-CART±ASCT · MRD by flow cytometry · 3 month
次要终点：Progression-free Survival (PFS)；Minimal Residual Disease (MRD) negetive rate；Overall response rate (ORR) evaluated by the investigators；Duration of Response (DOR)；Time to Response (TTR)；Overall Survival (OS)；PK:Cmax,Tmax,AUC(0-28days),Tlast
所有研究参与者根据治疗开始前的疾病状态非随机分组。适合移植者进入移植（ASCT CART）治疗组，不适合移植者进入非移植组。移植组患者在适当时间先接受ASCT，之后接受BCMA-CART治疗；非移植组不接受ASCT，可在适当时间直接接受BCMA-CART治疗。
评估并比较BCMA-CART±ASCT治疗年轻新诊断多发性骨髓瘤（NDMM）患者的安全性和有效性。
Evaluate and compare the safety and efficacy of BCMA-CART ± ASCT in the treatment of newly diagnosed multiple myeloma (NDMM) in young patients
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