- ICH GCP
- Registro de ensayos clínicos de EE. UU.
- Ensayo clínico NCT07587125
Safety and Efficacy of S103 Cells in Progressive/Refractory Multiple Sclerosis (RESET-MS)
3 de agosto de 2026 actualizado por: 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.
Descripción general del estudio
Estado
Reclutamiento
Condiciones
Intervención / Tratamiento
Tipo de estudio
Intervencionista
Inscripción (Estimado)
9
Fase
- Fase 1
Contactos y Ubicaciones
Esta sección proporciona los datos de contacto de quienes realizan el estudio e información sobre dónde se lleva a cabo este estudio.
Estudio Contacto
- Nombre: Wen Jiang Dr.
- Número de teléfono: 86 29 84771319
- Correo electrónico: jiangwen@fmmu.edu.cn
Copia de seguridad de contactos de estudio
- Nombre: Xiaodan Shi Dr.
- Número de teléfono: +86 29 84771319
- Correo electrónico: Shixd999@163.com
Ubicaciones de estudio
-
-
Shaanxi
-
Xi'an, Shaanxi, Porcelana, 710032
- Reclutamiento
- Xijing Hospital
-
Contacto:
- Wen Jiang, Dr.
- Número de teléfono: +86 29 84771319
- Correo electrónico: jiangwen@fmmu.edu.cn
-
Contacto:
- Xiaodan Shi, Dr.
- Número de teléfono: +86 29 84771319
- Correo electrónico: Shixd999@163.com
-
-
Criterios de participación
Los investigadores buscan personas que se ajusten a una determinada descripción, denominada criterio de elegibilidad. Algunos ejemplos de estos criterios son el estado de salud general de una persona o tratamientos previos.
Criterio de elegibilidad
Edades elegibles para estudiar
- Adulto
- Adulto Mayor
Acepta Voluntarios Saludables
No
Descripción
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.
Plan de estudios
Esta sección proporciona detalles del plan de estudio, incluido cómo está diseñado el estudio y qué mide el estudio.
¿Cómo está diseñado el estudio?
Detalles de diseño
- Propósito principal: Tratamiento
- Asignación: N / A
- Modelo Intervencionista: Asignación Secuencial
- Enmascaramiento: Ninguno (etiqueta abierta)
Armas e Intervenciones
Grupo de participantes/brazo |
Intervención / Tratamiento |
|---|---|
|
Experimental: 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
|
¿Qué mide el estudio?
Medidas de resultado primarias
Medida de resultado |
Medida Descripción |
Periodo de tiempo |
|---|---|---|
|
Incidence and type of Dose-Limiting Toxicities (DLTs)
Periodo de tiempo: 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.
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From Day 1 up to Day 28 post S103 infusion
|
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Incidence and severity of AEs, including changes in vital signs, physical examination, laboratory parameters, Electrocardiograms and Echocardiograms.
Periodo de tiempo: From Day 1 up to week 26 post S103 infusion
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To evaluate the AEs occurring within 6 months after S103 infusion
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From Day 1 up to week 26 post S103 infusion
|
Medidas de resultado secundarias
Medida de resultado |
Medida Descripción |
Periodo de tiempo |
|---|---|---|
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PD-soluble BCMA
Periodo de tiempo: From Day 1 up to week 26 post S103 infusion
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The changes in concentration of BCMA in the peripheral blood after S103 infusion
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From Day 1 up to week 26 post S103 infusion
|
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PK-BCMA CAR-T cells
Periodo de tiempo: From Day 1 up to Day 28 post S103 infusion
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The concentration of BCMA-CAR T cells(cells/mL) in peripheral blood after administration was detected by flow cytometry
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From Day 1 up to Day 28 post S103 infusion
|
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PD-NFL
Periodo de tiempo: From Day 1 up to week 26 post S103 infusion
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The changes in peripheral blood neurofilament light chain(NfL) concentration in patients with MS
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From Day 1 up to week 26 post S103 infusion
|
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PD-OB
Periodo de tiempo: From Day 1 up to week 26 post S103 infusion
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The changes in CSF Oligoclonal Bands index in patients with MS
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From Day 1 up to week 26 post S103 infusion
|
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Changes of Expanded Disability Status Scale(EDSS)scores
Periodo de tiempo: 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.
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From Day 1 up to week 26 post S103 infusion
|
Otras medidas de resultado
Medida de resultado |
Medida Descripción |
Periodo de tiempo |
|---|---|---|
|
Changes in proportion of peripheral blood immune cell subsets and inflammatory markers levels
Periodo de tiempo: From Day 1 up to week 26 post S103 infusion
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Changes in proportion of peripheral blood immune cell subsets and inflammatory markers levels of subjects after infusion of S103
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From Day 1 up to week 26 post S103 infusion
|
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Changes in Cerebrospinal Fluid (CSF) parameters
Periodo de tiempo: From Day 1 up to week 26 post S103 infusion
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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
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From Day 1 up to week 26 post S103 infusion
|
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MS: Annualized Relapse Rate (ARR)
Periodo de tiempo: From Day 1 up to 1 year post S103 infusion
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Annualized relapse rate (ARR): Number of MS relapses divided by person-years of observation
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From Day 1 up to 1 year post S103 infusion
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Time to first relapse
Periodo de tiempo: From Day 1 up to 1 year post S103 infusion
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Time from S103 infusion to the first relapse of MS
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From Day 1 up to 1 year post S103 infusion
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Imaging prognostic evaluation
Periodo de tiempo: At baseline (pre-infusion), Day 28, and week 26 post S103 infusion
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Neurological imaging assessments before intervention and at 26 weeks after intervention, including: gadolinium-enhancing lesions, T2, SWI, and other relevant MRI sequences.
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At baseline (pre-infusion), Day 28, and week 26 post S103 infusion
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Percent of NEDA-3
Periodo de tiempo: From Day 1 up to 1 year post S103 infusion
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The proportion of patients achieving no evidence of disease activity-3 (NEDA-3), defined as no relapse, no disability worsening, and no MRI activity
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From Day 1 up to 1 year post S103 infusion
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9-Hole Peg Test (9-HPT)
Periodo de tiempo: At baseline (pre-infusion), Month 1, Month 3, and Month 6 post S103 infusion
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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.
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At baseline (pre-infusion), Month 1, Month 3, and Month 6 post S103 infusion
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Change in Timed 25-Foot Walk (T25-FW) Time
Periodo de tiempo: At Baseline (pre-infusion), Month 1, Month 3, and Month 6 post S103 infusion
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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.
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At Baseline (pre-infusion), Month 1, Month 3, and Month 6 post S103 infusion
|
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Change in Mini-Mental State Examination (MMSE) Score
Periodo de tiempo: At Baseline (pre-infusion), Month 1, Month 3, and Month 6 post S103 infusion
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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.
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At Baseline (pre-infusion), Month 1, Month 3, and Month 6 post S103 infusion
|
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Change in Montreal Cognitive Assessment (MoCA) Score
Periodo de tiempo: 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
|
Colaboradores e Investigadores
Aquí es donde encontrará personas y organizaciones involucradas en este estudio.
Patrocinador
Colaboradores
Fechas de registro del estudio
Estas fechas rastrean el progreso del registro del estudio y los envíos de resultados resumidos a ClinicalTrials.gov. Los registros del estudio y los resultados informados son revisados por la Biblioteca Nacional de Medicina (NLM) para asegurarse de que cumplan con los estándares de control de calidad específicos antes de publicarlos en el sitio web público.
Fechas importantes del estudio
Inicio del estudio (Actual)
8 de mayo de 2026
Finalización primaria (Estimado)
1 de noviembre de 2027
Finalización del estudio (Estimado)
1 de noviembre de 2029
Fechas de registro del estudio
Enviado por primera vez
28 de abril de 2026
Primero enviado que cumplió con los criterios de control de calidad
7 de mayo de 2026
Publicado por primera vez (Actual)
14 de mayo de 2026
Actualizaciones de registros de estudio
Última actualización publicada (Actual)
5 de agosto de 2026
Última actualización enviada que cumplió con los criterios de control de calidad
3 de agosto de 2026
Última verificación
1 de mayo de 2026
Más información
Términos relacionados con este estudio
Palabras clave
Términos MeSH relevantes adicionales
- Enfermedades del Sistema Nervioso
- Procesos Patológicos
- Enfermedad crónica
- Atributos de la enfermedad
- Enfermedades autoinmunes
- Enfermedades del sistema inmunológico
- Enfermedades Autoinmunes Desmielinizantes, SNC
- Enfermedades Autoinmunes del Sistema Nervioso
- Enfermedades desmielinizantes
- Condiciones Patológicas, Signos y Síntomas
- Esclerosis múltiple
- Esclerosis Múltiple Crónica Progresiva
Otros números de identificación del estudio
- KY20262101JIANGWEN
Plan de datos de participantes individuales (IPD)
¿Planea compartir datos de participantes individuales (IPD)?
INDECISO
Información sobre medicamentos y dispositivos, documentos del estudio
Estudia un producto farmacéutico regulado por la FDA de EE. UU.
No
Estudia un producto de dispositivo regulado por la FDA de EE. UU.
No
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