肿瘤细胞治疗研究
英文原题：Aponermin-Based Bridging Therapy Prior to CAR-T Infusion in Relapsed/Refractory Multiple Myeloma Patients With Extramedullary Disease
⚠ 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。
这是一项分期未标注的注册临床试验，评估 BCMACAR-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 20 例。试验地点：中国 · 北京、天津（共 2 个中心，其中中国 2 个）。登记号：NCT06793475。
不限性别 · ≥ 18 Years
纳入标准： 1. 被告知并自愿签署知情同意书（ICF）。 2. 年龄≥18岁。 3. 确诊为多发性骨髓瘤（MM）（IMWG共识指南）。 4. 根据IMWG标准诊断为复发或难治性髓外多发性骨髓瘤，且既往至少接受过1线治疗。髓外疾病（EMD）定义为非起源于骨骼病变的软组织浆细胞瘤。通过体格检查检测并经负重CT/MRI/PET-CT和/或活检确认（必要时），髓外病灶最大直径应≥2cm。 5. ECOG评分≤2。 6. 无活动性感染。 7. HBV-DNA、HCV-RNA和HIV阴性。 8. 肝功能符合以下标准：总胆红素<1.5×ULN（Gilbert综合征患者总胆红素必须<3×ULN），ALT和AST<3×ULN。 9. 肾功能符合以下标准：肌酐清除率≥30mL/min（使用Cockcroft-Gault公式计算）。 10. 筛选前7天内进行的血液检查必须符合以下标准：WBC计数≥1.0×10⁹/L，血红蛋白≥70g/L，血小板计数≥75×10⁹/L或≥50×10⁹/L（若骨髓中浆细胞≥50%）；或由研究者判定为合适。 11. 接受造血生长因子（如促红细胞生成素、粒细胞集落刺激因子[G-CSF]、粒细胞-巨噬细胞集落刺激因子[GM-CSF]，以及血小板刺激因子如促血小板生成素[TPO]或白介素-11）的患者必须在筛选前至少2周停止此类治疗。 12. 未怀孕的女性患者必须在筛选时确认妊娠阴性（通过β-hCG血清检测或尿妊娠试验）。 13. 男性患者、有生育能力的女性患者及其伴侣必须同意在治疗期间及CAR-T细胞输注后至少3个月内使用有效避孕措施。 14. 男性患者必须同意不自体捐献精子，从初始筛选期开始至末次给药后90天。 15. 患者必须同意遵守研究程序和随访访视。 排除标准： 1. 浆细胞白血病或孤立性浆细胞瘤。 2. 既往同时接受过BCMA和GPRC5D靶向治疗（仅接受过其中一种靶向治疗的患者可入组）。 3. 有证据表明对elotuzumab、carfilzomib或沙利度胺存在原发性或继发性耐药。 4. 妊娠或哺乳期女性，或未来六个月内计划怀孕的女性。 5. 感染性疾病（如HIV、活动性结核等）。 6. 活动性乙型肝炎或丙型肝炎感染。 7. 生命体征异常或无法配合检查。 8. 精神或心理障碍导致无法遵守治疗或治疗评估。 9. 严重过敏体质或严重过敏史，特别是对aponermin、carfilzomib、thalidomide、dexamethasone或相关药物的其他有效成分或辅料过敏。 10. 主要器官如心、肺或脑的显著功能障碍。 9) 患有严重自身免疫性疾病的患者。11) 研究者认为存在任何其他不适合参加本研究的理由。
Inclusion Criteria: 1. Be informed and voluntarily sign the Informed Consent Form (ICF). 2. Age ≥18 years. 3. Confirmed diagnosis of Multiple Myeloma(MM) (IMWG consensus guidelines) 4. Subjects with diagnosed relapsed or refractory extramedullary multiple myeloma according to IMWG criteria and have had at least 1 prior lines of therapy. Extramedullary disease (EMD) is defined as soft-tissue plasmacytomas NOT arising from skeletal lesions. The maximum diameter of extramedullary lesions should ≥2cm detected by physical exam and confirmed (when required) by Weight Bearing CT/MRI/PET-CT and/or biopsy. 5. ECOG score is ≤ 2 6. No active infections. 7. Negative for HBV-DNA, HCV-RNA, and HIV. 8. Liver function meeting the following criteria: Total bilirubin \<1.5 × ULN (patients with Gilbert's syndrome must have total bilirubin \<3 × ULN), ALT and AST \<3 × ULN. 9. Renal function meeting the following criteria: Creatinine clearance ≥30mL/min (calculated using the Cockcroft-Gault formula). 10. Blood tests conducted within 7 days before screening must meet the following standards: WBC count ≥1.0×10⁹/L, Hemoglobin ≥70g/L, Platelet count ≥75×10⁹/L or ≥50×10⁹/L (if ≥50% plasma cells are present in bone marrow); Or as determined appropriate by the investigator. 11. Patients receiving hematopoietic growth factors (e.g., erythropoietin, granulocyte colony-stimulating factor \[G-CSF\], granulocyte-macrophage colony-stimulating factor \[GM-CSF\], and platelet-stimulating factors such as thrombopoietin \[TPO\] or interleukin-11) must stop such treatments at least 2 weeks prior to screening. 12. Non-pregnant female patients must confirm pregnancy negativity at screening (via β-hCG serum test or urine pregnancy test). 13. Male patients, female patients of childbearing potential, and their partners must agree to use effective contraception during the treatment period and for at least 3 months after CAR-T cell infusion. 14. Male patients must agree not to donate sperm, starting from the initial screening period until 90 days after the last dose. 15. Patients must agree to comply with study procedures and follow-up visits. Exclusion Criteria: 1. Plasma cell leukemia or solitary plasmacytoma. 2. Prior exposure to both BCMA- and GPRC5D-targeted therapies (patients who have received only one of these targeted therapies are eligible for enrollment). 3. Evidence of primary or secondary resistance to elotuzumab, carfilzomib, or thalidomide. 4. Pregnant or breastfeeding women, or women with pregnancy plans within the next six months. 5. Infectious diseases (e.g., HIV, active tuberculosis, etc.). 6. Active hepatitis B or hepatitis C infection. 7. Abnormal vital signs or inability to cooperate with examinations. 8. Mental or psychological disorders preventing compliance with treatment or treatment evaluation. 9. Severe allergic constitution or severe allergic history, particularly to aponermin, carfilzomib, thalidomide, dexamethasone or other effective components or excipients of related drugs. 10. Significant dysfunction of major organs, such as the heart, lungs, or brain. 9\) Patients with severe autoimmune diseases. 11) Any other reasons deemed unsuitable for participation in this study as determined by the 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：Overall response rate (ORR) · The definition of ORR is the proportion of participants who achieve a PR or better as the best response according to the IMWG criteria. · within 1 months after BCMA/GPRC5D CAR-T infusion
次要终点：ORR before CAR-T cell infusion；Progression free survival(PFS)；Overall Survival (OS)；Adverse events and serious adverse events
患者将接受以Aponermin为基础的桥接治疗，随后接受基于Fc的预处理和CAR-T细胞输注。CAR-T细胞治疗后一个月，患者将开始维持治疗，最长6个月，或直至疾病进展、死亡、不耐受、因其他原因退出，或研究终止/完成。
这是一项前瞻性、单臂、多中心、开放标签研究，旨在评估以aponermin为基础的桥接治疗在伴有髓外病变的复发/难治性多发性骨髓瘤患者中，于CAR-T输注前的疗效和安全性。
This is a prospective, single-arm, multicenter, open-label study to evaluate the efficacy and safety of aponermin-based bridging therapy prior to CAR-T infusion in relapsed/refractory multiple myeloma patients with extramedullary disease.
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