注册号：
Registration number：
ChiCTR2600122132 最近更新日期：
Date of Last Refreshed on：
2026-04-09 10:59:02 注册时间：
Date of Registration：
2026-04-09 00:00:00 注册号状态：
预注册Registration Status：
Prospective registration注册题目：
人自体多克隆调节性T细胞（NP001细胞注射液）治疗多系统萎缩患者安全性和有效性的Ⅰ期临床研究Public title：
An Phase Ⅰ clinical study evaluating the safety and efficacy of Autologous Human Polyclonal Regulatory T Cell Injection (NP001 Cell Injection) in patients with Multiple System Atrophy注册题目简写：
人自体多克隆调节性T细胞注射液治疗多系统萎缩患者安全性和有效性的Ⅰ期临床研究English Acronym：
An Phase Ⅰ clinical study evaluating the safety and efficacy of Autologous Human Polyclonal Regulatory T Cell Injection in patients with Multiple System Atrophy研究课题的正式科学名称：
人自体多克隆调节性T细胞（NP001细胞注射液）治疗多系统萎缩患者安全性和有效性的Ⅰ期临床研究Scientific title：
An Phase Ⅰ clinical study evaluating the safety and efficacy of Autologous Human Polyclonal Regulatory T Cell Injection (NP001 Cell Injection) in patients with Multiple System Atrophy研究课题代号(代码)：
Study subject ID：在二级注册机构或其它机构的注册号：
The registration number of the Partner Registry or other
register：申请注册联系人：
吕明启
研究负责人：
王伊龙 Applicant：
Mingqi Lu
Study leader：
Yilong Wang 申请注册联系人电话：
Applicant telephone：
+86 158 0084 3507
研究负责人电话：
Study leader's telephone：
+86 178 6076 5756申请注册联系人传真 ：
Applicant Fax：
研究负责人传真：
Study leader's fax：申请注册联系人电子邮件：
Applicant E-mail：
lumingqi@novabiotx.com
研究负责人电子邮件：
Study leader's E-mail：
yilong528@gmail.com申请单位网址(自愿提供)：
Applicant website(voluntary supply)：
研究负责人网址(自愿提供)：
Study leader's website(voluntary supply)：申请注册联系人通讯地址：
上海市浦东新区华夏东路333弄10B楼5层
研究负责人通讯地址：
北京市丰台区南四环西路119号Applicant address：
5th Floor, Unit 10B, Lane 333, Huaxia East Road,
Pudong New Area, Shanghai, China
Study leader's address：
No. 119, South 4th Ring West Road, Fengtai
District, Beijing, China申请注册联系人邮政编码：
Applicant postcode：
研究负责人邮政编码：
Study leader's postcode：申请人所在单位：
上海赛尔欣⽣物医疗科技有限公司Applicant's institution：
Shanghai Saierxin Biomedical Technology Co., Ltd研究负责人所在单位：
⾸都医科⼤学附属北京天坛医院Affiliation of the Leader：
Beijing Tiantan Hospital, Capital Medical University是否获伦理委员会批准：
是Approved by ethic committee：
Yes伦理委员会批件文号：
Approved No. of ethic committee：
YW2026-006-02
伦理委员会批件附件：
Approved file of Ethical Committee：
查看附件View批准本研究的伦理委员会名称：
⾸都医科⼤学附属北京天坛医院医学伦理委员会Name of the ethic committee：
IRB of Beijing Tiantan Hospital, Capital Medical University伦理委员会批准日期：
Date of approved by ethic committee：
2026-03-12 00:00:00伦理委员会联系人：
梁晓珊Contact Name of the ethic committee：
Xiaoshan Liang伦理委员会联系地址：
北京市丰台区南四环西路119号Contact Address of the ethic committee：
No. 119, South 4th Ring West Road, Fengtai District, Beijing, China伦理委员会联系人电话：
Contact phone of the ethic committee：
+86 10 5997 6269
伦理委员会联系人邮箱：
Contact email of the ethic committee：研究实施负责（组长）单位：
⾸都医科⼤学附属北京天坛医院Primary sponsor：
Beijing Tiantan Hospital, Capital Medical University研究实施负责（组长）单位地址：
北京市丰台区南四环西路119号Primary sponsor's address：
No. 119, South 4th Ring West Road, Fengtai District, Beijing, China试验主办单位(项目批准或申办者)：
Secondary sponsor：
国家：
中国
省(直辖市)：
上海市
市(区县)：
上海市
Country：
China
Province：
Shanghai
City：
Shanghai
单位(医院)：
上海赛尔欣生物医疗科技有限公司
具体地址：
上海市浦东新区华夏东路333弄10B楼
Institution
hospital：
Shanghai Saierxin Biomedical Technology Co., Ltd
Address：
5th Floor, Unit 10B, Lane 333, Huaxia East Road, Pudong New Area, Shanghai, China经费或物资来源：
上海赛尔欣生物医疗科技有限公司Source(s) of funding：
Shanghai Saierxin Biomedical Technology Co., Ltd研究疾病：
多系统萎缩 Target disease：
Multiple System Atrophy研究疾病代码：Target disease code：研究类型：
干预性研究Study type：
Interventional study研究所处阶段：
I期临床试验 Study phase：
1研究设计：
随机平行对照 Study design：
Parallel 研究目的：
这是一项评价Treg细胞治疗MSA受试者安全性、耐受性和有效性的Ⅰ期临床研究，分为SAD期单剂量递增、MAD期多剂量递增和剂量扩展三个部分：SAD期和MAD期为单臂、开放性研究；扩展期分两部分，首先采用随机、双盲、安慰剂对照的初始阶段，随后进入长期开放标签扩展研究。
主要目的：1.评估NP001细胞注射液治疗MSA受试者的安全性和耐受性（时间范围：至末次给药后6个月（SAD）/12个月（MAD/扩展期））。
次要目的：1.、评估NP001细胞注射液治疗对疾病状态的影响（时间范围：至末次给药后6个月（SAD）/12个月（MAD/扩展期））。2.通过生存随访，评估NP001细胞注射液治疗对MSA受试者生存期（OS）的影响（时间范围：直至受试者满足退出研究标准或整个研究结束/提前终止或生存随访期满2年[从安全性随访期结束开始计算]）。3.评估NP001细胞注射液治疗对血液中免疫系统参数的影响（时间范围：至末次给药后6个月（SAD）/12个月（MAD/扩展期））。4.评估NP001细胞注射液治疗对脑脊液中免疫系统参数的影响（时间范围：至末次给药后6个月（SAD）/12个月（MAD/扩展期））。 Objectives of Study：
This is a Phase I clinical study evaluating the safety, tolerability, and efficacy of Treg cell therapy in subjects with MSA, consisting of three parts: a single ascending dose (SAD) phase, a multiple ascending dose (MAD) phase, and a dose expansion phase. The SAD and MAD phases are single-arm, open-label studies. The expansion phase is divided into two parts: first, a randomized, double-blind, placebo-controlled initial stage, followed by a long-term open-label extension study.
Primary Objective:
1.To evaluate the safety and tolerability of NP001 cell injection in subjects with MSA (timeframe: up to 6 months after the last dose in the SAD phase / 12 months after the last dose in the MAD/expansion phase).
Secondary Objectives:
1.To evaluate the effect of NP001 cell injection on disease status (timeframe: up to 6 months after the last dose in the SAD phase / 12 months after the last dose in the MAD/expansion phase).
2.To assess the impact of NP001 cell injection on overall survival (OS) in subjects with MSA through survival follow-up (timeframe: until subjects meet study withdrawal criteria, the study ends/terminates early, or the 2-year survival follow-up period is completed [starting from the end of the safety follow-up period]).
3.To evaluate the effect of NP001 cell injection on immune system parameters in blood (timeframe: up to 6 months after the last dose in the SAD phase / 12 months after the last dose in the MAD/expansion phase).
4.To evaluate the effect of NP001 cell injection on immune system parameters in cerebrospinal fluid (timeframe: up to 6 months after the last dose in the SAD phase / 12 months after the last dose in the MAD/expansion phase).药物成份或治疗方案详述：
Description for medicine or protocol of treatment in
detail：
纳入标准：
受试者必须符合以下所有标准，才有资格入组本研究：
1. 30岁至70岁（包括边界值）的男性或女性受试者。
2. 根据国际运动障碍学会（MDS）和中国专家共识（2022版）的定义，诊断为MSA的患者，包括神经病理确诊的、临床确诊的和临床很可能的MSA。
3. 受试者的MSA病程（从首次症状发作至筛选访视）≤5年。
4. 根据研究者的判断，估计生存时间≥3年。
5. 受试者现有针对MSA核心症状治疗控制不佳的。
6. 在无需他人协助的情况下可以自主行走至少10步，允许使用辅助设备（如助行器或拐杖）。
7. 筛选期呼吸功能：%FVC≥65%。
8. 首次给药前14天未接受过且研究期间也无使用计划，或至少接受稳定剂量的抗帕金森药物治疗至少4周，且研究期间剂量保持不变。
9. 受试者必须有适当的器官功能，符合下列所有检查结果：
• 肌酐清除率>30mL/min（根据Cockcroft-Gault公式）；
• 血清总胆红素≤1.5×ULN（对于吉尔伯特综合征受试者，血清总胆红素≤3×ULN）；
• ALT或AST≤2×ULN；
• 凝血酶原时间≤1.5×ULN、活化部分凝血酶原时间（APTT）<1.5×ULN、国际标准化比值（INR）<1.5×ULN，且无异常出血病史；
• 中性粒细胞绝对计数（ANC）≥1.0×10^9/L（筛选期实验室检查前7天内未接受过粒细胞集落刺激因子[G-CSF]等生长因子）支持治疗；
• 淋巴细胞计数≥0.3×10^9/L；
• 血小板计数≥50×10^9/L（筛选期实验室检查前7天内未接受血小板注射）。
10. 受试者无外周血单细胞采集禁忌症（如：红细胞压积<25%、血小板<50×10^9/L等）。
11. 对于有生育能力的女性受试者或伴侣有生育能力的男性受试者同意在签署知情同意后至末次给药后至少12个月采取医学上认可的有效避孕措施，男性受试者禁止捐精，女性受试者禁止捐卵；有生育能力的受试者在基线血妊娠试验必须为阴性。
注：有生育能力的女性是指经历过初潮、未接受过绝育手术（子宫切除术或双侧卵巢切除术或双侧输卵管结扎术）、不处于绝经状态（绝经是指无其他医学原因下，持续自然停经≥12个月）的女性。
12. 在执行不属于标准医疗护理的任何与研究相关的程序之前，必须获得ICF，并理解受试者可以随时撤回ICF，而不会影响未来的护理。Inclusion criteria
Subjects must meet all of the following criteria to be eligible for enrollment in this study:
1. Male or female subjects aged 30 to 70 years (inclusive).
2. Patients diagnosed with MSA according to the International Movement Disorder Society (MDS) criteria and the Chinese expert consensus (2022 edition), including neuropathologically confirmed, clinically established, and clinically probable MSA.
3. Duration of MSA (from first symptom onset to screening visit) <= 5 years.
4. Estimated life expectancy >= 3 years, as judged by the investigator.
5. Inadequate control of current core MSA symptoms with existing therapies.
6. Able to walk at least 10 steps independently without assistance, with or without aids (such as a walker or cane).
7. Respiratory function at screening: %FVC >= 65%.
8. No use of antiparkinsonian medications within 14 days prior to the first dose and no plan to use during the study, or have been on a stable dose of antiparkinsonian medications for at least 4 weeks with the dose to remain unchanged during the study.
9. Subjects must have adequate organ function, meeting all of the following laboratory results:
(1) Creatinine clearance > 30 mL/min (calculated by Cockcroft-Gault formula);
(2) Serum total bilirubin <= 1.5 × ULN (for subjects with Gilbert's syndrome, <= 3 × ULN);
(3) ALT or AST <= 2 × ULN;
(4) Prothrombin time <= 1.5 × ULN, activated partial thromboplastin time (APTT) < 1.5 × ULN, and international normalized ratio (INR) < 1.5 × ULN, with no history of abnormal bleeding;
(5) Absolute neutrophil count (ANC) >= 1.0 × 10^9/L (no granulocyte colony-stimulating factor [G-CSF] or similar growth factor support within 7 days prior to screening laboratory tests);
(6) Lymphocyte count >= 0.3 × 10^9/L;
(7) Platelet count >= 50 × 10^9/L (no platelet transfusion within 7 days prior to screening laboratory tests).
10. No contraindications to peripheral blood mononuclear cell collection (e.g., hematocrit < 25%, platelets < 50 × 10^9/L, etc.).
11. For female subjects of childbearing potential or male subjects with partners of childbearing potential, agreement to use medically approved effective contraception from signing the informed consent until at least 12 months after the last dose. Male subjects must refrain from sperm donation, and female subjects must refrain from egg donation. Subjects of childbearing potential must have a negative blood pregnancy test at baseline.
Note: A female of childbearing potential is defined as one who has experienced menarche, has not undergone sterilization (hysterectomy, bilateral oophorectomy, or bilateral tubal ligation), and is not postmenopausal (menopause is defined as the absence of menses for >= 12 consecutive months without other medical causes).
12. Before performing any study-related procedures that are not part of standard medical care, a signed informed consent form (ICF) must be obtained. Subjects must understand that they may withdraw the ICF at any time without affecting future medical care.排除标准：
符合以下任一标准的受试者均将从本研究中排除：
1. 筛查时头MRI显示其他中枢神经系统病变的证据，提示除了MSA之外的其他神经系统退行性疾病的诊断。
2. 筛查时头MRI显示其他重要的病理发现，包括但不限于：脑出血，急性期脑梗死，动脉瘤，血管畸形，感染性病变，脑肿瘤或其他占位性病变（最大直径<1cm的脑膜瘤或蛛网膜囊肿不需要排除）。
3. 符合以下任何一项表明病情已较为严重的标准的受试者：
• 根据UMSARS-I第1题得分为≥3的情况判定的言语障碍；
• 根据UMSARS-I第2题得分为≥3的情况判定的吞咽障碍；
• 根据UMSARS-I第7题得分为≥3的情况判定的行走障碍；
• 根据UMSARS-I第8题得分为≥3的情况判定的每周发生跌倒次数超过一次的情况；
4. 受试者对NP001细胞注射液中任何一种成分有过敏史。
5. 筛选期存在未控制的活动性感染。
6. 患有严重的伴随情况或疾病，研究者认为会使受试者处于不适当的风险或干扰研究，包括但不限于：
• 签署ICF前6个月内记录有肺栓塞病史；
• 签署ICF前6个月患有慢性阻塞性肺病（COPD）；
• 已知中度或重度持续性哮喘，或过去2年内有哮喘病史，或目前有任何分类的不受控制的哮喘（需注意，目前病情可控的间歇性哮喘或可控的轻度持续性哮喘的受试者允许参与本研究），需要吸氧才能维持充分的血氧饱和度。
7. 严重心脑血管疾病，包括但不限于签署ICF前6个月内有：
• 不稳定型心绞痛、脑血管意外或短暂性脑缺血；
• 严重心律失常；
• 严重非缺血性心肌病；
• 活动传导系统异常的心电图证据；
• 充血性心力衰竭（纽约心脏病学会III级或IV级）；
• 需要机械维持（如心脏起搏器）的其他心脏疾病；
• 高血压且经≤2种降压药治疗无法下降到以下范围内（收缩压<140 mmHg且舒张压<90 mmHg）；
• 低血压且经过治疗后仍低于正常范围（收缩压<90 mmHg或舒张压<60 mmHg）；
• 有冠状动脉搭桥术或血管成形术病史的受试者将接受心脏病学评估，并由研究者根据具体情况进行考虑。
8. 受试者患有经研究者判断为不稳定的系统性疾病：包括但不限于需要药物治疗的严重肝脏、肾脏、呼吸系统疾病或代谢性疾病。
9. 筛选期人类免疫缺陷病毒（HIV）、乙型肝炎病毒（HBV）、丙型肝炎病毒（HCV）、梅毒、EB病毒、巨细胞病毒、戊型肝炎病毒（HEV）、甲型肝炎病毒（HAV）、人类嗜T淋巴细胞病毒（HTLV）血清学检测提示感染。
10. 妊娠或哺乳期受试者。
11. 单采前4周内或5个半衰期（以较长者为准）内使用过另一种研究药物、生物制剂或装置进行过治疗。受试者参与观察性/非介入性临床研究将与医学监察员及研究者讨论。
12. 既往接受过目标适应症为MSA的基因或细胞疗法治疗。
13. 既往治疗导致的任何非血液学毒性尚未恢复到CTCAE v6.0分级≤1级或基线。
14. 单采前4周内接受过大手术或在研究期间有手术计划。
15. 单采前4周内，有活疫苗接种史（包括减毒活疫苗）。
16. 患有除MSA外的其他自身免疫性疾病、免疫缺陷或其它需要免疫抑制剂治疗的疾病。
17. 单采前1周内，系统性使用超过5 mg/d的强的松（或等量的其他皮质类固醇激素）。
18. 存在饲管。
19. 不耐受基本医疗操作的，如单采、鞘内注射、采血等。
20. 正在使用抗精神病药、抗癫痫药（苯二氮卓类药物、加巴丁、普瑞巴林除外）或1类（例如氟卡尼）或3类（例如胺碘酮）抗心律失常药物。
21. 合并酒精、药物滥用史或2年内存在酒精、药物依赖的患者。
22. 经研究者判断，具有重大自杀风险的受试者。
23. 研究者认为不适合入组的其他情况。Exclusion criteria：
Subjects who meet any of the following criteria will be excluded from the study:
1. Head MRI at screening shows evidence of other central nervous system lesions suggesting a diagnosis of neurodegenerative diseases other than MSA.
2. Head MRI at screening shows other significant pathological findings, including but not limited to: cerebral hemorrhage, acute cerebral infarction, aneurysm, vascular malformation, infectious lesions, brain tumors, or other space-occupying lesions (meningiomas or arachnoid cysts with a maximum diameter <1 cm do not require exclusion).
3. Subjects meeting any of the following criteria indicating more severe disease:
(1) Speech impairment as determined by a score of >=3 on Unified Multiple System Atrophy Rating Scale (UMSARS) Part I, Item 1;
(2) Swallowing impairment as determined by a score of >=3 on UMSARS Part I, Item 2;
(3) Walking impairment as determined by a score of >=3 on UMSARS Part I, Item 7;
(4) Experiencing falls more than once per week as determined by a score of >=3 on UMSARS Part I, Item 8.
4. History of allergy to any component of the NP001 cell injection.
5. Presence of uncontrolled active infection at screening.
6. Presence of severe concurrent conditions or diseases that, in the investigator's judgment, would place the subject at undue risk or interfere with the study, including but not limited to:
(1) History of pulmonary embolism documented within 6 months prior to signing the ICF;
(2) History of chronic obstructive pulmonary disease (COPD) within 6 months prior to signing the ICF;
(3) Known moderate or severe persistent asthma, or history of asthma within the past 2 years, or currently having uncontrolled asthma of any classification (Note: subjects with currently controlled intermittent or mild persistent asthma are allowed to participate). Requires supplemental oxygen to maintain adequate blood oxygen saturation.
7. Severe cardiovascular or cerebrovascular disease, including but not limited to any of the following within 6 months prior to signing the ICF:
(1) Unstable angina, cerebrovascular accident, or transient ischemic attack;
(2) Severe arrhythmia;
(3) Severe non-ischemic cardiomyopathy;
(4) Electrocardiographic evidence of active conduction system abnormalities;
(5) Congestive heart failure (New York Heart Association Class III or IV);
(6) Other cardiac diseases requiring mechanical support (e.g., pacemaker);
(7) Hypertension that cannot be controlled to the following range (systolic blood pressure <140 mmHg and diastolic blood pressure <90 mmHg) with <=2 antihypertensive medications;
(8) Hypotension that remains below the normal range (systolic blood pressure <90 mmHg or diastolic blood pressure <60 mmHg) despite treatment;
(9) Subjects with a history of coronary artery bypass grafting or angioplasty will undergo cardiology evaluation and be considered by the investigator on a case-by-case basis.
8. Subjects with systemic diseases judged by the investigator as unstable, including but not limited to severe hepatic, renal, respiratory, or metabolic diseases requiring pharmacologic treatment.
9. Screening serology tests positive for human immunodeficiency virus (HIV), hepatitis B virus (HBV), hepatitis C virus (HCV), syphilis, Epstein-Barr virus (EBV), cytomegalovirus (CMV), hepatitis E virus (HEV), hepatitis A virus (HAV), or human T-lymphotropic virus (HTLV), indicating infection.
10. Pregnant or breastfeeding subjects.
11. Treatment with another investigational drug, biologic, or device within 4 weeks or 5 half-lives (whichever is longer) prior to apheresis. Participation in observational/non-interventional clinical studies will be discussed with the Medical Monitor and investigator.
12. Prior treatment with gene or cell therapy targeted for MSA.
13. Any non-hematologic toxicity from prior therapy that has not recovered to CTCAE v6.0 Grade <=1 or baseline.
14. Major surgery within 4 weeks prior to apheresis or planned surgery during the study period.
15. History of live vaccination (including attenuated live vaccines) within 4 weeks prior to apheresis.
16. Presence of autoimmune diseases other than MSA, immunodeficiency, or other conditions requiring immunosuppressive therapy.
17. Systemic use of >5 mg/day prednisone (or equivalent dose of other corticosteroids) within 1 week prior to apheresis.
18. Presence of a feeding tube.
19. Intolerance to basic medical procedures, such as apheresis, intrathecal injection, blood draws, etc.
20. Concurrent use of antipsychotics, antiepileptic drugs (except benzodiazepines, gabapentin, pregabalin), or Class I (e.g., flecainide) or Class III (e.g., amiodarone) antiarrhythmic drugs.
21. History of alcohol or drug abuse, or alcohol/drug dependence within the past 2 years.
22. Subjects judged by the investigator to be at significant risk of suicide.
23. Any other condition deemed by the investigator to make the subject unsuitable for enrollment.研究实施时间：
Study execute time：
从
From
2026-04-15 00:00:00至
To
2029-12-31 00:00:00
征募观察对象时间：
Recruiting time：
从
From
2026-04-15 00:00:00
至
To
2029-12-31 00:00:00干预措施：
Interventions：
组别：
SAD
样本量：
9
Group：
SAD
Sample size：
干预措施：
NP001：鞘内回输，单次给药，3个剂量组（1.0、3.0、9.0×10^7个Treg细胞）。如经SRC判断某个预设剂量组满足剂量递增终止标准，经SRC确认后可能额外增加其他备降剂量组（0.5、2.0、6.0×10^7个Treg细胞）。
干预措施代码：
Intervention：
NP001: Intrathecal re-infusion, single administration, with three dose cohorts (1.0, 3.0, and 9.0 × 10^7 regulatory T cells). If the Study Review Committee (SRC) determines that a pre-specified dose cohort meets the dose escalation stopping criteria, additional lower alternative dose cohorts (0.5, 2.0, and 6.0 × 10^7 regulatory T cells) may be added upon SRC confirmation.
Intervention code：
组别：
MAD
样本量：
6
Group：
MAD
Sample size：
干预措施：
NP001:鞘内回输。MAD期重复给药，每月1次，共三次。剂量根据SAD期选择。
干预措施代码：
Intervention：
NP001: Intrathecal infusion. Multiple-ascending dose phase with repeated administration once monthly for a total of three doses. Dosage to be selected based on the single-ascending dose phase.
Intervention code：
组别：
扩展期-试验组-MSA-C
样本量：
9
Group：
Expansion Phase - Treatment Group - MSA-C
Sample size：
干预措施：
NP001:鞘内回输。扩展期重复给药，每月1次，共三次。剂量根据MAD期选择。
干预措施代码：
Intervention：
NP001: Intrathecal infusion. The expansion phase with repeated administration once monthly for a total of three doses. Dosage to be selected based on the MAD phase.
Intervention code：
组别：
扩展期-安慰剂组-MSA-C
样本量：
3
Group：
Expansion Phase - Placebo Group - MSA-C
Sample size：
干预措施：
NP001:鞘内回输。扩展期的双盲阶段给予与试验药物等体积的0.9%生理盐水，扩展期的开放标签阶段给予试验药物。每个阶段的给药均为每月1次，共三次。剂量根据MAD期选择。
干预措施代码：
Intervention：
NP001: Intrathecal re-infusion. During the double-blind phase of the expansion period, an equal volume of 0.9% saline (placebo) will be administered. During the open-label phase of the expansion period, the investigational product will be administered. In each phase, dosing will occur once monthly for a total of three administrations.
Intervention code：
组别：
扩展期-试验组-MSA-P
样本量：
9
Group：
Expansion Phase - Treatment Group - MSA-P
Sample size：
干预措施：
NP001:鞘内回输。扩展期重复给药，每月1次，共三次。剂量根据MAD期选择。
干预措施代码：
Intervention：
NP001: Intrathecal infusion. The expansion phase with repeated administration once monthly for a total of three doses. Dosage to be selected based on the MAD phase.
Intervention code：
组别：
扩展期-安慰剂组-MSA-P
样本量：
3
Group：
Expansion Phase - Placebo Group - MSA-P
Sample size：
干预措施：
NP001:鞘内回输。扩展期的双盲阶段给予与试验药物等体积的0.9%生理盐水，扩展期的开放标签阶段给予试验药物。每个阶段的给药均为每月1次，共三次。
干预措施代码：
Intervention：
NP001: Intrathecal re-infusion. During the double-blind phase of the expansion period, an equal volume of 0.9% saline (placebo) will be administered. During the open-label phase of the expansion period, the investigational product will be administered. In each phase, dosing will occur once monthly for a total of three administrations.
Intervention code：研究实施地点：
Countries of recruitment and research settings：
国家：
中国
省(直辖市)：
北京市
市(区县)：
北京市
Country：
China
Province：
Beijing
City：
Beijing
单位(医院)：
首都医科大学附属北京天坛医院
单位级别：
三甲
Institution
hospital：
Beijing Tiantan Hospital, Capital Medical University
Level of the institution：
Tertiary A测量指标：
Outcomes：
指标中文名：
不良事件（AE）及严重不良事件（SAE）的类型、严重程度和发生率，包括注射NP001细胞注射液后生命体征、体格检查、12-ECG、实验室检查（血常规、血生化、凝血功能、尿常规）等有显著临床意义的变化。
指标类型：
主要指标
Outcome：
Type, severity, and incidence of adverse events (AEs) and serious adverse events (SAEs), including clinically significant changes in vital signs, physical examination, 12-lead ECG, and laboratory tests (complete blood count, blood biochemistry, coagulation function, urinalysis) following the administration of NP001 cell injection.
Type：
Primary indicator
测量时间点：
测量方法：
Measure time point of outcome：
Measure method：
指标中文名：
剂量限制性毒性（DLT）的发生率与特征。
指标类型：
主要指标
Outcome：
Incidence and characteristics of dose-limiting toxicities (DLTs).
Type：
Primary indicator
测量时间点：
测量方法：
Measure time point of outcome：
Measure method：
指标中文名：
NP001细胞注射液的MTD。
指标类型：
主要指标
Outcome：
Maximum tolerated dose (MTD) of NP001 cell injection.
Type：
Primary indicator
测量时间点：
测量方法：
Measure time point of outcome：
Measure method：
指标中文名：
NP001细胞注射液对疾病状态的影响：UMSARS、MoCA、%FVC、ATLIS、手握力、步态功能、COMPASS、3T MRI影像学特征、脑多巴胺代谢功能活性等较基线的变化
指标类型：
次要指标
Outcome：
Effect of NP001 cell injection on disease status: Changes from baseline in UMSARS, MoCA, %FVC, ATLIS, handgrip strength, gait function, COMPASS, 3T MRI imaging characteristics, and cerebral dopamine metabolic activity.
Type：
Secondary indicator
测量时间点：
测量方法：
Measure time point of outcome：
Measure method：
指标中文名：
生存期（OS）
指标类型：
次要指标
Outcome：
Overall Survival (OS)
Type：
Secondary indicator
测量时间点：
测量方法：
Measure time point of outcome：
Measure method：
指标中文名：
血液中免疫系统参数：Th2、Th1、Treg细胞的数量较基线的变化；血清炎症因子和NfL、GFAP和TREM-2较基线的变化。
指标类型：
次要指标
Outcome：
Immune system parameters in blood: Changes from baseline in the counts of Th2, Th1, and Treg cells; and changes in serum inflammatory cytokines and levels of NfL, GFAP, and TREM-2.
Type：
Secondary indicator
测量时间点：
测量方法：
Measure time point of outcome：
Measure method：
指标中文名：
脑脊液中免疫系统参数：Treg细胞的数量较基线的变化；炎症因子和NfL、GFAP和TREM-2较基线的变化。
指标类型：
次要指标
Outcome：
Immune system parameters in cerebrospinal fluid: Changes from baseline in the count of Treg cells; and changes in inflammatory cytokines and levels of NfL, GFAP, and TREM-2.
Type：
Secondary indicator
测量时间点：
测量方法：
Measure time point of outcome：
Measure method：采集人体标本：
Collecting sample(s)
from participants：
标本中文名：
血液
组织：
Sample Name：
Blood
Tissue：
人体标本去向
使用后销毁
说明
Fate of sample：
Destruction after use
Note：
标本中文名：
脑脊液
组织：
Sample Name：
Cerebrospinal fluid
Tissue：
人体标本去向
使用后销毁
说明
Fate of sample：
Destruction after use
Note：征募研究对象情况：
Recruiting status：
尚未开始
Not yet
recruiting
年龄范围：
Participant age：
最小
Min age
30
岁
years
最大
Max age
70
岁
years性别：
男女均可
Gender：
Both随机方法（请说明由何人用什么方法产生随机序列）：
扩展期受试者筛选成功的受试者将采⽤交互式⽹络应答系统（IWRS）分配随机号。受试者的随机号由统计师应⽤SAS 9.4或更⾼版本软件产⽣，同样亚型的受试者按照3：1的比例随机分配⾄试验药物组或空⽩组，。 对于因任何原因退出临床试验的已随机分组的受试者，将保留其随机号，不可被替换，已退出的受试者也不能再参加本研究。Randomization Procedure (please state who
generates the
random number sequence and by what method)：
Subjects who successfully pass screening for the expansion phase will be assigned a randomization number via an Interactive Web Response System (IWRS). The randomization numbers for subjects will be generated by a statistician using SAS software version 9.4 or higher. Subjects of the same subtype will be randomly assigned to either the investigational drug group or the blank control group in a 3:1 ratio. For any randomized subject who withdraws from the clinical trial for any reason, their assigned randomization number will be retained and cannot be replaced. Subjects who have withdrawn are also not permitted to re-enter the study.是否公开试验完成后的统计结果:
Calculated Results after the Study Completed public access:
公开/Public盲法：
本研究方案分三个阶段：SAD和MAD为单臂、开放性研究；扩展期分两部分，首先采用随机、双盲、安慰剂对照的初始阶段，随后进入一项长期的开放标签扩展研究，在初始阶段，合格受试者被随机分配至活性药物组或安慰剂组。Blinding：
This study protocol is divided into three phases: the SAD and MAD phases are single-arm, open-label studies. The expansion phase consists of two parts: first, a randomized, double-blind, placebo-controlled initial stage, followed by a long-term open-label extension study. During the initial stage, eligible subjects are randomly assigned to either the active drug group or the placebo group.试验完成后的统计结果（上传文件）：
点击下载Calculated Results after
the Study Completed(upload file)：
download是否共享原始数据：
IPD sharing
否No共享原始数据的方式（说明：请填入公开原始数据日期和方式，如采用网络平台，需填该网络平台名称和网址）：
否The way of sharing IPD”(include metadata and
protocol,
If use web-based public database, please provide
the
url)：
None数据采集和管理（说明：数据采集和管理由两部分组成，一为病例记录表(Case
Record Form, CRF)，二为电子采集和管理系统(Electronic Data
Capture, EDC)，如ResMan即为一种基于互联网的EDC：
本研究采用电子数据采集系统（EDC）进行数据采集。
数据管理计划：由数据管理（DM）撰写，作为整个数据管理过程的指导性文件，数据管理所有过程均应按照其中定义的时间、内容及方法进行操作。
本研究数据管理由申办者或申办者委托的CRO数据部负责，以确保临床研究数据的真实性、完整性、私密性和可溯源性。
eCRF：根据方案要求设计数据采集表格，定义研究流程、数据表单名称及其收集的数据项，同时形成相应的eCRF填写说明，经申办者审阅后，供研究中心填写eCRF使用。
eCRF的数据均来源于原始病历，由研究者或者研究者指定人员填写，需确保信息的完整性和准确性。如果出现任何需要更正的错误，修改应按照eCRF填写说明进行规范操作，EDC系统将自动记录数据修改者的姓名及修改日期。
EDC系统中数据经原始数据核查（SDV）、DM审核、质疑等处理确认无疑问后，在数据锁定前，研究者需要进行电子签名确认。Data collection and Management (A
standard data collection and management system
include a CRF and an electronic data capture：
This study utilizes an Electronic Data Capture (EDC) system for data collection.
Data Management Plan: A Data Management (DM) Plan will be authored by the Data Management team to serve as the guiding document for the entire data management process. All data management procedures must be conducted in accordance with the timelines, content, and methods defined therein.
The data management for this study is the responsibility of the Sponsor or a CRO's Data Management department commissioned by the Sponsor, ensuring the authenticity, integrity, confidentiality, and traceability of the clinical study data.
eCRF: Data collection forms will be designed according to the protocol requirements, defining the study workflow, data form names, and the data items to be collected. Corresponding eCRF completion guidelines will also be developed. These will be reviewed by the Sponsor before being provided to the study sites for eCRF completion.
All data in the eCRF originate from source documents. They are to be entered by the Investigator or a designee, who must ensure the completeness and accuracy of the information. If any errors require correction, modifications must be made following the standardized procedures outlined in the eCRF completion guidelines. The EDC system will automatically record the name of the person making the modification and the date.
Before data lock, and after data in the EDC system have undergone processing such as Source Data Verification (SDV), DM review, and query resolution with no outstanding issues, the Investigator is required to provide an electronic signature for confirmation.数据与安全监察委员会：
Data and Safety Monitoring Committee：
暂未确定/Not yet注册人：
Name of Registration：
2026-04-09 10:58:57
