肿瘤细胞治疗研究
英文原题：ASCT Combined With BCMA CAR-T and GPRC5D/CD3 BiTEs Maintenance for Transplant-Eligible Ultra-High-Risk Multiple Myeloma
⚠ 该试验的登记信息已有 14 个月未更新， 页面上显示的「招募中」可能已经失效。联系研究中心之前，建议先到 ClinicalTrials.gov 核对登记原文的最新状态与联系方式。
这是一项 II 期注册临床试验，评估自体造血干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 30 例。试验地点：中国 · 天津（共 1 个中心，其中中国 1 个）。登记号：NCT07106710。
不限性别 · ≥ 18 Years 且 ≤ 70 Years
纳入标准：年龄≥18岁且≤70岁；按IMWG诊断标准确诊新发多发性骨髓瘤；筛查时存在可测量疾病：血清M蛋白≥1.0 g/dL或尿M蛋白≥200 mg/24小时；无血清或尿液可测量疾病的轻链型骨髓瘤患者，血清免疫球蛋白游离轻链（FLC）≥10 mg/dL且血清κ/λ FLC比值异常；适合接受大剂量化疗及自体造血干细胞移植；至少有一项超高危特征：双重打击型骨髓瘤（至少存在以下两项高危细胞遗传学异常：t(4;14)、t(14;16)、1p缺失、1q增加、MYC重排、17p缺失或TP53突变）；髓外软组织浆细胞瘤；外周血循环浆细胞≥2%；肿瘤细胞BCMA和GPRC5D阳性；血清总胆红素<正常值上限2倍，AST和ALT<正常值上限3倍，肌酐清除率≥30 mL/min（Cockcroft-Gault公式）；能够理解并愿意签署书面知情同意书。排除标准：活动性淀粉样变；中枢神经系统受累；既往接受BCMA靶向治疗或CAR-T治疗；活动性乙肝或丙肝病毒感染；已知HIV感染；预期寿命<6个月；妊娠或哺乳；有心、肺、脑或其他重要器官未控制的功能障碍证据；研究主要研究者判断不适合入组的其他情况。
Inclusion Criteria: 1. Age ≥ 18 years and ≤ 70 years. 2. Participants with documented newly-diagnosed multiple myeloma according to IMWG diagnostic criteria. 3. Measurable disease at screening, defined as: Serum M-protein level ≥1.0 g/dL or urine M-protein level ≥200 mg/24 hours; or Light chain MM without measurable disease in serum or urine: serum Ig free-light chain (FLC) ≥10 mg/dL and abnormal serum Ig kappa lambda FLC ratio. 4. Patients deemed eligible for high-dose chemotherapy with ASCT. 5. Presence of at least one of the following ultra-high-risk features: a. Double-hit multiple myeloma, defined as the presence of at least two of the following high-risk cytogenetic abnormalities: t(4;14), t(14;16), deletion 1p, gain 1q, MYC rearrangement, deletion 17p, or TP53 mutation; b. Presence of extramedullary soft tissue plasmacytomas; c. Circulating plasma cells ≥2% in peripheral blood. 6. Tumor cells were BCMA and GPRC5D positive. 7. Serum total bilirubin \<2 x upper limit of normal (ULN), serum AST and ALT \<3 x ULN, creatinine clearance ≥ 30mL/min (Cockroft-Gault formula). 8. Informed Consent/Assent: All subjects have the ability to understand and the willingness to sign a written informed consent. Exclusion Criteria: 1. Active amyloidosis. 2. Central nervous system involvement. 3. Prior BCMA-targeted therapy or CAR-T therapy. 4. Active hepatitis B or hepatitis C virus infection. 5. Known HIV infection. 6. Life expectancy \<6 months. 7. Woman who are pregnant or breastfeeding. 8. Evidence of uncontrolled dysfunction of heart, lung, brain, and other important organs. 9. Any other conditions that are not eligible for the trial in the judgement of the principal investigator.
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：Sustained MRD-negative rate · Rate of patients achieving sustained MRD negativity for more than 12 months · Up to 2 year；MRD negativity rate · Rate of patients achieving MRD negativity · Up to 2 years
次要终点：Safety and Tolerability；Complete response rate (CRR)；Progression free survival (PFS)；Overall survival (OS)
患者接受自体造血干细胞移植，随后输注BCMA CAR-T。CAR-T输注3个月后，开始GPRC5D/CD3双特异性抗体维持治疗，疗程≥2年。
前瞻性、单臂II期研究，评估自体造血干细胞移植联合BCMA CAR-T治疗，随后使用GPRC5D/CD3双特异性抗体维持治疗适合移植的超高危多发性骨髓瘤患者的疗效和安全性。
This is a prospective, single-arm, phase II study to evaluate the efficacy and safety of autologous stem cell transplantation combined with BCMA CAR-T therapy followed by GPRC5D/CD3 bispecific antibody maintenance in transplant-eligible patients with ultra-high-risk multiple myeloma.
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