Safety and Efficacy of S103 Cells in Progressive/Refractory Multiple Sclerosis (RESET-MS)
2026年8月3日 更新者:Xijing Hospital
Safety and Efficacy of S103 Cells in Patients With Progressive or Refractory Multiple Sclerosis:An Open-Label, Single-Arm, Exploratory Clinical Study
This study is a single-center, open-label, single-arm, exploratory clinical study to evaluate the safety, tolerability, and preliminary efficacy of S103(BCMA-CAR T )cells in the treatment of progressive or refractory multiple sclerosis.
The study is a dose escalation trial in adult progressive and refractory MS patients.
A standard "3+3" design will be used to perform dose escalation to explore the safety profile and dose-limiting toxicities (DLTs).
A total of 9 MS patients who meet the inclusion criteria are expected to be recruited.
研究概览
地位
招聘中
干预/治疗
研究类型
介入性
注册 (估计的)
9
阶段
- 阶段1
联系人和位置
本节提供了进行研究的人员的详细联系信息,以及有关进行该研究的地点的信息。
学习联系方式
- 姓名:Wen Jiang Dr.
- 电话号码:86 29 84771319
- 邮箱:jiangwen@fmmu.edu.cn
研究联系人备份
- 姓名:Xiaodan Shi Dr.
- 电话号码:+86 29 84771319
- 邮箱:Shixd999@163.com
学习地点
-
-
Shaanxi
-
Xi'an、Shaanxi、中国、710032
- 招聘中
- Xijing Hospital
-
接触:
- Wen Jiang, Dr.
- 电话号码:+86 29 84771319
- 邮箱:jiangwen@fmmu.edu.cn
-
接触:
- Xiaodan Shi, Dr.
- 电话号码:+86 29 84771319
- 邮箱:Shixd999@163.com
-
-
参与标准
研究人员寻找符合特定描述的人,称为资格标准。这些标准的一些例子是一个人的一般健康状况或先前的治疗。
资格标准
适合学习的年龄
- 成人
- 年长者
接受健康志愿者
不
描述
Inclusion Criteria:
- Age ≥18 years and ≤75 years;
- The subject signs the informed consent form, is willing and able to comply with the protocol, complete the research assessments and return for follow-up;
- To be diagnosed with Multiple Sclerosis (MS) according to the 2017 McDonald criteria, specifically including Progressive MS (Primary Progressive MS [PPMS] or Secondary Progressive MS [SPMS]) or Relapsing-Remitting MS (RRMS);
- The subject must be assessed by the investigator as having progressive or refractory MS for which no effective standard therapy is available. For subjects with Relapsing MS (RMS), refractory/progressive disease is defined as having experienced at least 2 clinical relapses within the past 2 years, or at least 1 clinical relapse within the past 1 year accompanied by at least one gadolinium-enhancing lesion on MRI within the past year, despite treatment with standard disease-modifying therapies (DMTs). In addition, the subject must have an Expanded Disability Status Scale (EDSS) score between 2.0 and 7.0, inclusive, at both screening and baseline;
- Male study participants must agree to take contraceptive measures during the treatment period and within 1 year after receiving study treatment, and are prohibited from donating sperm throughout the study period;
- Women of childbearing potential (WOCBP) must agree to take contraceptive measures during treatment and for at least 1 year after receiving study treatment. Participants must have a negative serum pregnancy test result during screening; a negative urine pregnancy test result must be confirmed before receiving CAR-T for the first time.
Exclusion Criteria:
Subjects will be ineligible for inclusion in the study if they meet any of the following criteria prior to screening or at the baseline visit:
- The subject has any medical, psychological, or social condition that, in the investigator's opinion, may harm the subject, interfere with their ability to participate in the study, or result in poor protocol compliance;
- Female subjects who are pregnant or lactating, plans to become pregnant at any time within 12 months after receiving CAR-T cell treatment, or has a history of spontaneous or induced abortion within 4 weeks prior to screening;
- The subject has a clinically relevant active infection, such as sepsis, pneumonia, or a severe local abscess, or a serious infection requiring hospitalization or intravenous antibiotic treatment within 4 weeks prior to screening. Subjects are also excluded if they have a known immunodeficiency disorder including Human Immunodeficiency Virus (HIV), test positive for Hepatitis B surface antigen (HBsAg) or detectable Hepatitis B virus (HBV) DNA, test positive for Hepatitis C virus (HCV) antibody with detectable HCV RNA, test positive for syphilis during the screening period, or have an active or high-risk history of tuberculosis infection;
- The subject has previously received any Chimeric Antigen Receptor (CAR) T-cell therapy or other genetically modified cell therapies, or has a history of allogeneic hematopoietic stem cell transplantation (HSCT) or solid organ transplantation. Furthermore, subjects are excluded if they have received intravenous immunoglobulin (IVIG) or plasma exchange (PE) within 4 weeks prior to screening, or have received tocilizumab or eculizumab treatment within 3 months prior to screening;
- The subject is diagnosed with an active, severe autoimmune disease other than Multiple Sclerosis, such as Systemic Lupus Erythematosus or Rheumatoid Arthritis. This also includes the presence of other severe progressive neurodegenerative or central nervous system (CNS) disorders, such as Parkinson's disease, Alzheimer's disease, stroke, or active CNS tumors, that the investigator believes would confound the clinical assessment of Multiple Sclerosis. Additionally, a history of active malignancy within the past 5 years excludes the subject, with the exception of adequately treated basal cell or squamous cell carcinoma of the skin, or carcinoma in situ of the cervix;
- The subject exhibits specific laboratory abnormalities or organ dysfunction during the screening period, including elevated liver enzymes with AST or ALT greater than 1.5 times the upper limit of normal (ULN), total bilirubin greater than 1.5 times the ULN, or a Creatinine Clearance (CrCl) of less than 60 mL/min. Exclusions also apply for an absolute neutrophil count (ANC) of less than 2.0 × 10^9/L, a platelet count of less than 100 × 10^9/L, hemoglobin levels below 100 g/L, or severe cardiovascular or pulmonary diseases, which include a Left Ventricular Ejection Fraction (LVEF) below 50%, unstable angina or myocardial infarction within the past 6 months, or a resting peripheral blood oxygen saturation (SpO2) of 91% or less;
- The subject has a known severe allergy, hypersensitivity, or intolerance to fludarabine, cyclophosphamide, or any excipients contained in the S103 CAR-T cell product, such as human serum albumin. Subjects are also excluded if they have received any live attenuated vaccine within 6 weeks prior to screening, or plan to receive a live vaccine during the study period.
学习计划
本节提供研究计划的详细信息,包括研究的设计方式和研究的衡量标准。
研究是如何设计的?
设计细节
- 主要用途:治疗
- 分配:不适用
- 介入模型:顺序分配
- 屏蔽:无(打开标签)
武器和干预
参与者组/臂 |
干预/治疗 |
|---|---|
|
实验性的:S103 cells
subjects will undergo leukapheresis followed by lymphodepletion conditioning with cyclophosphamide and fludarabine, then receive S103 CAR-T cell infusion.
|
Dose Level 0 (De-escalation dose): 0.5×10^6 CAR-T cells/kg Dose Level 1 (Starting dose): 1.0×10^6 CAR-T cells/kg Dose Level 2 (Maximum dose): 2.0×10^6 CAR-T cells/kg
|
研究衡量的是什么?
主要结果指标
结果测量 |
措施说明 |
大体时间 |
|---|---|---|
|
Incidence and type of Dose-Limiting Toxicities (DLTs)
大体时间:From Day 1 up to Day 28 post S103 infusion
|
A DLTs is defined as any Grade 4 toxicity or Grade 3 toxicity lasting more than 7 days occurring within 28 days after BCMA-CAR T infusion that is related to the treatment (with specific exceptions for manageable CRS, neurotoxicity, and hematologic toxicities as defined in the protocol).
This is used to determine the safety and tolerable dose.
|
From Day 1 up to Day 28 post S103 infusion
|
|
Incidence and severity of AEs, including changes in vital signs, physical examination, laboratory parameters, Electrocardiograms and Echocardiograms.
大体时间:From Day 1 up to week 26 post S103 infusion
|
To evaluate the AEs occurring within 6 months after S103 infusion
|
From Day 1 up to week 26 post S103 infusion
|
次要结果测量
结果测量 |
措施说明 |
大体时间 |
|---|---|---|
|
PD-soluble BCMA
大体时间:From Day 1 up to week 26 post S103 infusion
|
The changes in concentration of BCMA in the peripheral blood after S103 infusion
|
From Day 1 up to week 26 post S103 infusion
|
|
PK-BCMA CAR-T cells
大体时间:From Day 1 up to Day 28 post S103 infusion
|
The concentration of BCMA-CAR T cells(cells/mL) in peripheral blood after administration was detected by flow cytometry
|
From Day 1 up to Day 28 post S103 infusion
|
|
PD-NFL
大体时间:From Day 1 up to week 26 post S103 infusion
|
The changes in peripheral blood neurofilament light chain(NfL) concentration in patients with MS
|
From Day 1 up to week 26 post S103 infusion
|
|
PD-OB
大体时间:From Day 1 up to week 26 post S103 infusion
|
The changes in CSF Oligoclonal Bands index in patients with MS
|
From Day 1 up to week 26 post S103 infusion
|
|
Changes of Expanded Disability Status Scale(EDSS)scores
大体时间:From Day 1 up to week 26 post S103 infusion
|
The EDSS score ranges from 0 to 10, with higher scores indicating worse disability.
A negative change relative to baseline indicates clinical improvement.
Disability worsening is defined as follows: an increase of at least 2.0 points if the baseline EDSS score is 0; an increase of at least 1.0 point if the baseline EDSS score is 1.0 to 5.0; and an increase of at least 0.5 points if the baseline EDSS score is 5.5 or higher.
|
From Day 1 up to week 26 post S103 infusion
|
其他结果措施
结果测量 |
措施说明 |
大体时间 |
|---|---|---|
|
Changes in proportion of peripheral blood immune cell subsets and inflammatory markers levels
大体时间:From Day 1 up to week 26 post S103 infusion
|
Changes in proportion of peripheral blood immune cell subsets and inflammatory markers levels of subjects after infusion of S103
|
From Day 1 up to week 26 post S103 infusion
|
|
Changes in Cerebrospinal Fluid (CSF) parameters
大体时间:From Day 1 up to week 26 post S103 infusion
|
Evaluation of central nervous system (CNS) microenvironment changes through lumbar puncture.
Parameters include CSF cytology, biochemistry, cytokines, lymphocyte subsets, free kappa light chains, and oligoclonal band (OB) index, and multi-omics analysis of cerebrospinal fluid immune microenvironment
|
From Day 1 up to week 26 post S103 infusion
|
|
MS: Annualized Relapse Rate (ARR)
大体时间:From Day 1 up to 1 year post S103 infusion
|
Annualized relapse rate (ARR): Number of MS relapses divided by person-years of observation
|
From Day 1 up to 1 year post S103 infusion
|
|
Time to first relapse
大体时间:From Day 1 up to 1 year post S103 infusion
|
Time from S103 infusion to the first relapse of MS
|
From Day 1 up to 1 year post S103 infusion
|
|
Imaging prognostic evaluation
大体时间:At baseline (pre-infusion), Day 28, and week 26 post S103 infusion
|
Neurological imaging assessments before intervention and at 26 weeks after intervention, including: gadolinium-enhancing lesions, T2, SWI, and other relevant MRI sequences.
|
At baseline (pre-infusion), Day 28, and week 26 post S103 infusion
|
|
Percent of NEDA-3
大体时间:From Day 1 up to 1 year post S103 infusion
|
The proportion of patients achieving no evidence of disease activity-3 (NEDA-3), defined as no relapse, no disability worsening, and no MRI activity
|
From Day 1 up to 1 year post S103 infusion
|
|
9-Hole Peg Test (9-HPT)
大体时间:At baseline (pre-infusion), Month 1, Month 3, and Month 6 post S103 infusion
|
Change from baseline in the 9-Hole Peg Test (9-HPT), measured in seconds.
A higher value indicates worse upper extremity function.
A negative change from baseline indicates improvement.
|
At baseline (pre-infusion), Month 1, Month 3, and Month 6 post S103 infusion
|
|
Change in Timed 25-Foot Walk (T25-FW) Time
大体时间:At Baseline (pre-infusion), Month 1, Month 3, and Month 6 post S103 infusion
|
Change from baseline in the Timed 25-Foot Walk (T25-FW), measured in seconds.
A higher value indicates worse ambulatory function.
A negative change from baseline indicates improvement.
|
At Baseline (pre-infusion), Month 1, Month 3, and Month 6 post S103 infusion
|
|
Change in Mini-Mental State Examination (MMSE) Score
大体时间:At Baseline (pre-infusion), Month 1, Month 3, and Month 6 post S103 infusion
|
Change from baseline in the Mini-Mental State Examination (MMSE) score.
Scores range from 0 to 30, with higher scores indicating better cognitive function.
A positive change from baseline indicates improvement.
|
At Baseline (pre-infusion), Month 1, Month 3, and Month 6 post S103 infusion
|
|
Change in Montreal Cognitive Assessment (MoCA) Score
大体时间:At Baseline (pre-infusion), Month 1, Month 3, and Month 6 post S103 infusion
|
Change from baseline in the Montreal Cognitive Assessment (MoCA) score.
Scores range from 0 to 30, with higher scores indicating better cognitive function.
A positive change from baseline indicates improvement.
|
At Baseline (pre-infusion), Month 1, Month 3, and Month 6 post S103 infusion
|
合作者和调查者
在这里您可以找到参与这项研究的人员和组织。
研究记录日期
这些日期跟踪向 ClinicalTrials.gov 提交研究记录和摘要结果的进度。研究记录和报告的结果由国家医学图书馆 (NLM) 审查,以确保它们在发布到公共网站之前符合特定的质量控制标准。
研究主要日期
学习开始 (实际的)
2026年5月8日
初级完成 (估计的)
2027年11月1日
研究完成 (估计的)
2029年11月1日
研究注册日期
首次提交
2026年4月28日
首先提交符合 QC 标准的
2026年5月7日
首次发布 (实际的)
2026年5月14日
研究记录更新
最后更新发布 (实际的)
2026年8月5日
上次提交的符合 QC 标准的更新
2026年8月3日
最后验证
2026年5月1日
更多信息
与本研究相关的术语
关键字
其他相关的 MeSH 术语
其他研究编号
- KY20262101JIANGWEN
计划个人参与者数据 (IPD)
计划共享个人参与者数据 (IPD)?
未定
药物和器械信息、研究文件
研究美国 FDA 监管的药品
不
研究美国 FDA 监管的设备产品
不
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