肿瘤细胞治疗研究
英文原题：hUC-MSC-Exo Therapy for Autoimmune Encephalitis
⚠ 该试验的登记信息已有 13 个月未更新， 页面上显示的「尚未开始招募」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 I/II 期注册临床试验，评估人源脐带间充质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：尚未开始招募。计划入组 38 例。登记号：NCT07131683。
不限性别 · ≥ 18 Years 且 ≤ 65 Years
纳入标准： 1. 年龄18-65岁，男女不限； 2. 发病3个月内诊断为自身免疫性脑炎（符合2016年Graus和Dalmau诊断标准），且血清/脑脊液抗NMDAR抗体或抗LGI1抗体阳性； 3. 入组时改良Rankin量表（mRS）评分≥2分； 4. 受试者或其法定授权代表能够签署知情同意书； 5. 有生育能力的受试者必须同意在研究期间采取严格避孕措施。 排除标准： 1. 病前改良Rankin量表（mRS）评分≥2分； 2. 已知对研究产品任何成分过敏或曾有严重过敏反应史； 3. 存在研究者认为可能影响试验参与或研究评估的神经系统或精神疾病（如脑血管病、帕金森病、重度抑郁）； 4. 目前存在或既往有研究者判断不适合入组的任何临床显著全身性疾病，包括但不限于： * 严重心血管疾病（如充血性心力衰竭、严重心律失常、心肌梗死） * 肝脏疾病（如肝硬化） * 肾脏疾病（如需血液透析或腹膜透析） * 血液系统疾病（如伴出血倾向的血友病） * 内分泌疾病（如血糖>16.8 mmol/L或<2.8 mmol/L的未控制糖尿病，或伴有严重并发症） * 免疫系统疾病（活动性或未控制的全身性自身免疫性疾病、原发性/继发性免疫缺陷） * 恶性肿瘤； 5. 鼻腔解剖异常、鼻黏膜损伤、严重鼻炎或其他影响药物给药的鼻部情况； 6. 需要留置胃管； 7. 器官功能符合以下任一标准： 1. 中性粒细胞绝对计数（ANC）<1.5×10⁹/L，血小板（PLT）<100×10⁹/L，血红蛋白（Hb）<90 g/L 2. 天冬氨酸氨基转移酶（AST）>2.5×ULN和/或丙氨酸氨基转移酶（ALT）>2.5×ULN，总胆红素（TBIL）>1.5×ULN 3. 肌酐>1.5×ULN 4. 未接受抗凝/抗血小板治疗：国际标准化比值（INR）>1.7或活化部分凝血活酶时间（APTT）>1.25×ULN 接受抗凝/抗血小板治疗：INR>3.0或APTT>1.5×ULN； 8. 乙型肝炎表面抗原（HBsAg）或乙型肝炎核心抗体（HBcAb）阳性且HBV-DNA可检出；或丙型肝炎抗体（HCVAb）、梅毒螺旋体抗体（TPAb/RPR）或人类免疫缺陷病毒抗体（HIV）阳性； 9. 妊娠或哺乳期患者； 10. MRI禁忌症（如心脏起搏器等金属植入物、幽闭恐惧症）； 11. 给药前3个月内（或末次给药后5个半衰期内，以较长者为准）参加过任何涉及研究药物的临床试验； 12. 给药前3个月内经历重大创伤或手术，或计划在试验期间进行手术（不包括基线前4周以上的腹腔镜检查或小手术；不包括胸腺瘤/畸胎瘤手术）； 13. 给药前1年内有药物滥用或酗酒史； 14. 既往接受过干细胞或其衍生物治疗； 15. 研究者判断的任何其他可能增加患者风险或干扰结果解读的情况。
Inclusion Criteria: 1. Aged 18-65 years, both male and female are eligible; 2. Diagnosis of autoimmune encephalitis within 3 months of onset (meeting the 2016 Graus and Dalmau diagnostic criteria), with positive serum/cerebrospinal fluid anti-NMDAR antibodies or anti-LGI1 antibodies; 3. Modified Rankin Scale (mRS) score ≥ 2 at enrollment; 4. The subject or legally authorized representative is able to sign the informed consent form; 5. Subjects of childbearing potential must agree to practice strict contraception during the study period. Exclusion Criteria: 1. Pre-morbid modified Rankin Scale (mRS) score ≥ 2; 2. Known allergy to any component of the investigational product or history of severe allergic reactions; 3. Presence of neurological or psychiatric disorders (e.g., cerebrovascular disease, Parkinson's disease, severe depression) deemed by the investigator to potentially impair trial participation or study assessments; 4. Current or history of any clinically significant systemic diseases judged by the investigator as unsuitable for inclusion, including but not limited to: * Severe cardiovascular diseases (e.g., congestive heart failure, severe arrhythmia, myocardial infarction) * Hepatic diseases (e.g., cirrhosis) * Renal diseases (e.g., requiring hemodialysis or peritoneal dialysis) * Hematological diseases (e.g., hemophilia with bleeding tendency) * Endocrine disorders (e.g., poorly controlled diabetes with blood glucose \>16.8 mmol/L or \<2.8 mmol/L, or with severe complications) * Immune system disorders (active or uncontrolled systemic autoimmune diseases, primary/secondary immunodeficiency) * Malignancies; 5. Anatomical nasal abnormalities, nasal mucosal damage, severe rhinitis, or other nasal conditions affecting drug administration; 6. Requiring nasogastric tube placement; 7. Organ function meeting any of the following criteria: 1. Absolute neutrophil count (ANC) \<1.5×10⁹/L, platelets (PLT) \<100×10⁹/L, hemoglobin (Hb) \<90 g/L 2. Aspartate aminotransferase (AST) \>2.5×ULN and/or alanine aminotransferase (ALT) \>2.5×ULN, total bilirubin (TBIL) \>1.5×ULN 3. Creatinine \>1.5×ULN 4. Without anticoagulant/antiplatelet therapy: International normalized ratio (INR) \>1.7 or activated partial thromboplastin time (APTT) \>1.25×ULN With anticoagulant/antiplatelet therapy: INR \>3.0 or APTT \>1.5×ULN; 8. Positive hepatitis B surface antigen (HBsAg) or hepatitis B core antibody (HBcAb) with detectable HBV-DNA; or positive for hepatitis C antibody (HCVAb), Treponema pallidum antibody (TPAb/RPR), or human immunodeficiency virus antibody (HIV); 9. Pregnant or lactating patients; 10. Contraindications for MRI (e.g., metal implants such as pacemakers, claustrophobia); 11. Participation in any clinical trial involving investigational drugs within 3 months prior to dosing (or within 5 half-lives of last dose, whichever is longer); 12. Major trauma or surgery within 3 months prior to dosing, or planned surgery during the trial (excluding laparoscopy or minor procedures \>4 weeks before baseline; excluding thymoma/teratoma surgery); 13. History of drug abuse or alcoholism within 1 year prior to dosing; 14. Previous treatment with stem cells or derivatives; 15. Any other condition that may increase patient risk or interfere with result interpretation, 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：DLT events · Drug-related dose limiting toxicity (DLT) · Within 24 weeks after drug administration；mRS · modified Ranking Scale (mRS), which ranges from 0 to 6, with higher scores indicating worse outcome, and a score of 6 representing death. · 24 weeks ±10 days
次要终点：AE and SAE；mRS；Percentage of mRS 0-2；Percentage of mRS improvement ≧ 1；CASE score change compared to baseline；ADL score change compared to baseline；MMSE score change compared to baseline；MoCA score change compared to baseline
这是一项I/IIa期研究，旨在探讨人脐带间充质干细胞来源外泌体（hUC-MSC-Exo）经鼻给药用于自身免疫性脑炎患者的安全性和初步疗效。
This is a phase I/IIa study to investigate the safety and preliminary efficacy of intranasal admnistration of human umbilical mesenchymal stem cell-derived exosome (hUC-MSC-Exo) for patients with autoimmune encephalitis.
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