Autoimmune DiseasesEarly Phase 1CARTinNSNCT04561557
Safety and Efficacy of CT103A Cells for Relapsed/Refractory Antibody-associated Inflammatory Diseases of the Nervous System
A Early Phase 1 study testing CT103A cells and Cyclophosphamide and fludarabine in people with Autoimmune Diseases and Autoimmune Diseases of the Nervous System, run by Tongji Hospital at 1 site in China.
Trial summary
Antibody-mediated inflammatory diseases of the nervous system (also known as autoimmune diseases of the nervous system) are autoimmune diseases in which autoimmune cells and immune molecules attack the nervous system as the main pathogenic mechanism. Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Autoimmune Diseases, Autoimmune Diseases of the Nervous System, Neuromyelitis Optica Spectrum Disorder
- What is being tested?
- CT103A cells and Cyclophosphamide and fludarabine
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 36 participants
- Where
- 1 siteChina
- Study dates
- 22 Sep 2020to 31 May 2027
- Phase
- Early Phase 1
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Male or female subjects aged 18-75 years (including 18 and 75 years);
- Subjects with Relapsing/refractory Antibody-mediated inflammatory diseases of the nervous system without effective treatment, including:
- Subjects must be diagnosed as AQP4-IgG-positive NMOSD defined by 2015 criteria of IPND NMOSD and meet the following requirements: i. At least one kind of immunosuppressant has been used for more than one year with poorly-controlled symptoms; ii. Clinical evidence of at least two relapses in the last 12 months or three relapses in the last 24 months and one relapse in the preceding 12 months before screening.
- Subjects with MG with positive abnormal antibody, MG-ADL total score ≥ 6 points, MGFA classification II-IV defined by 2020 MGFA diagnostic criteria and meet the following requirement: i. At least one kind of immunosuppressant for standardized treatment for more than 1 year, and have one of the following poor control conditions: 1) continuous inability to affect daily life; 2) Exacerbation of MG symptoms and/or crisis attacks still occur despite standard treatment; 3) Inability to tolerate immunosuppressive therapy ii. Requires plasma exchange or maintenance therapy with IV gamma globulin
- Subjects with CIDP with positive abnormal antibodies, INCAT disability scale with total score of 2-9 defined by 2021 EAN/PNS diagnostic criteria and meet the following requirement: i. Standardized use of at least one first-line therapy for more than 3 months (cortisol hormone therapy, gamma globulin or plasma exchange therapy) with poorly-controlled symptoms. ii. Inability to tolerate cortisol hormones, gamma globulin, and plasmapheresis because of side effects or other conditions
- Subjects were diagnosed with IIM defined by 2017 European League against Rheumatism/American Rheumatology (EULAR/ACR) conference Class criteria; At least one kind of cardiac enzymes (CK, AST, ALT, ALD, LDH) ≥1.5×ULN during the screening period, or Cutaneous Dermatomyositis Disease Area and Severity Index (CDASI) ≥6, or at least one other sign of active disease within the last 6 months: MRI, EMG, or muscle biopsy; positive serological tests for myositis-specific antibodies (MSA) or myositis-associated autoantibodies (MAA), or antinuclear antibody (ANA). and meet one of the following requirements:
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- After at least 1 month of corticosteroid therapy and standardized use of at least one immunosuppressant/modulator (eg, azathioprine, methotrexate, mycophenolate mofetil, cyclosporine, tacrolimus, Cyclophosphamide, leflunomide, intravenous gamma globulin, etc.) for more than 3 months with poorly-controlled symptoms.
- ii. Inability to tolerate the above traditional regimens due to side effects or other conditions;
- Subjects were diagnosed with PMS (including PPMS and SPMS) or RMS according to the 2017 revision of the McDonald diagnostic criteria；EDSS score between 2 to 7 points inclusive, at screening. Subjects with RMS should meet one of the following requirements after standard therapy: i. at least two relapses in the last two years before screening. ii. at least one relapse in the last one year before screening. iii. positive Gd-enhancing MRI in the last one year before screening.
- Subjects were diagnosed with POEMS syndrome according to the 2021 revised IMWG diagnostic criteria and meet all of the following requirements: i. bone marrow involvement; ii. no response to traditional regimens treatment including corticosteroid, chemotherapy, protease inhibitor or inability to tolerate the above traditional regimens; iii. Have measurable lesions (refer to the 2021 revised IMWG standard) iv. VEGF > 2 ULN; v. ECOG score ≥1; vi. ONLS score ≥1.
- Subjects were diagnosed with autoimmune encephalitis according to the 2016 International Diagnostic Criteria for Autoimmune Encephalitis and meet all of the following requirements: i. at least one pathogenic antibody positive; ii. previously standardized use of corticosteroid, at least one immunosuppressant/modulator, including CD20 monoclonal antibody with poorly-controlled symptoms or intolerance; iii. onset of autoimmune encephalitis within 3 months prior to screening; iv. mRS Score ≥2 or CASE score ≥4.
- Subjects were diagnosed with MOGAD according to the 2023 International MOGAD Diagnostic criteria and meet all of the following requirements: i. a documented positive serum MOG Ab test using a cell-based assay (CBA); ii mRS Score ≥2; iii previously standardized use of corticosteroid, at least one immunosuppressant/modulator, including CD20 monoclonal antibody with poorly-controlled symptoms or intolerance.
- All acute toxic reactions resolved to baseline or ≤ grade 1 assessed using NCI-CTCAE v5.0 except the ones adjudicated by the investigator to pose no risks on subjects.
- Enrolled subjects must have satisfactory organ function and laboratory findings as defined by the following:i. Blood tests: absolute neutrophil count ≥ 2×109/L (or normal lower limit set by the central lab of the institution), platelets ≥ 100 × 109/L, and hemoglobin ≥ 100 g/L; ii. Liver function: total serum bilirubin, alanine aminotransferase (ALT) and aspartate aminotransferase (AST) must be ≤ 1.5x the institutional normal upper limit (ULN); iii. Kidney function: CrCl ≥ 60 ml/min/1.73m2 (according to the following Cockcroft-Gault formula); iv. Electrolytes: blood potassium ≥ 3.0 mmol/L; blood calcium ≥ 2.0 mmol/L, blood magnesium ≥ 0.5 mmol/L; v. Coagulation function: fibrinogen ≥ 1.0 g/L; APTT ≤ ULN + 10s; PT ≤ ULN + 3s.
- Blood oxygen saturation > 91% in resting state.
- Echocardiography suggests LVEF≥ 50%.
- Expected life expectancy ≥ 12 weeks as assessed by the investigator.
- After signing the informed consent form, subjects and their partners must be willing to use effective and reliable method of contraception, devices or medicines, within one year after CT103A cells infusion (excluding contraception safety periods).
- Subjects must provide written informed consent before the study begin.
Who cannot join
- Patients do not have adequate mononuclear cells without mobilization for CAR-T cell manufacturing.
- History of autoimmune hemolytic disease.
- History of solid organ transplantation.
- Patients were treated with alemtuzumab within 6 months prior to apheresis. Patients were treated with fludarabine or cladribine within 3 months prior to apheresis.
- Patients with Papovaviruses infection.
- Patients have been diagnosed with malignancies in the last 2 years prior to screening except for non-melanoma skin cancer, stage I cancers with complete resection and low risk of relapse, localized prostate cancer post-treatments, biopsy-confirmed in situ cervical cancer, or squamous epithelial lesion by PAP smear.
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- Chronic and active hepatitis B (HBV), hepatitis C (HCV), Human Immunodeficiency Virus (HIV) infection, CMV or syphilis infections concurrently.
- MG crisis was not effectively controlled within 2 weeks before enrollment.
- Known history of primary immunodeficiency (innate or acquired).
- Patients with severe impaired cardiac function, including but not limited to the following: unstable angina, myocardial infarction (within 6 months before enrollment), congestive heart failure (≥Grade III by NYHA), severe ventricular arrhythmia.
- Cerebrovascular accidents, including transient ischemic attack or stroke history, occurred within 6 months before enrollment.
- Major operation or surgical treatment caused by any reason within 4 weeks before enrollment.
- Any serious and/or uncontrolled comorbidities which may interfere with the evaluation during the study in the opinion of the investigator
- Previous treatments: History of thymectomy within 12 months prior to CT103A infusion;
- History of psychoactive drug abuse and failed to withdraw, or have a history of psychiatric disorders.
- Prone to allergies or history of serious allergy.
- Pregnant or lactating women.
- Patients with other conditions adjudicated by the investigator as unsuitable for enrollment.
- Criteria for lymphodepletion and CAR-T cells infusion:
- Before lymphocyte depletion and CAR-T cells infusion, patients are evaluated and those meeting the following criteria cannot be included:
- Blood tests: neutrophil count < 2 × 109/L, platelet count < 50 × 10^9/L;
- Oxygen inhalation is required to maintain blood oxygen saturation ≥ 91%;
- Patients have the following conditions, including but not limited to: new arrhythmia cannot be controlled by drugs; hypotension requiring pressor drugs; bacterial, fungal or viral infection requiring intravenous antibiotic treatment; creatinine clearance rate < 50 ml/min ;
- Patients require maintenance support treatment within one week to meet the criteria for lymphodepletion or CAR T cell infusion.
- Cell infusion is delayed > 7 days after lymphodepletion for any reason;
- Patients with other conditions adjudicated by the investigator as unsuitable for lymphodepletion or cell infusion.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site, 1 recruiting
- Recruiting
Tongji Hospital affiliated to Tongji Medical College of Huazhong University of Science and Technology
Wuhan, Hubei, 430000
Chuan Qin, PhD, 86-27-83663337, qinchuan712@126.com
Chuan Qin, MD, 86-27-83663333, chuanqin@tjh.tjmu.edu.cn
About this study
Official title: An Open Label Clinical Trial to Evaluate the Safety and Efficacy of CT103A Cells for the Treatment of Relapsed/Refractory Antibody-associated Inflammatory Diseases of the Nervous System
Antibody-mediated inflammatory diseases of the nervous system (also known as autoimmune diseases of the nervous system) are autoimmune diseases in which autoimmune cells and immune molecules attack the nervous system as the main pathogenic mechanism. In the immune response, pathogenic antibodies acting on autoantigens of the nervous system are collectively referred to as autoantibodies of the nervous system, and antibody-mediated inflammatory diseases of the nervous system can occur in the central nervous system, peripheral nervous system, and neuromuscular junctions, and muscles. In this study, we will recruit eight kinds of autoimmune diseases of nervous system including Neuromyelitis Optica Spectrum Disorder (NMOSD), Myasthenia Gravis (MG), Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP), idiopathic inflammatory myopathyand (IIM), multiple sclerosis (MS), autoimmune encephalitis (AE), Myelin Oligodendrocyte Glycoprotein Antibody-Associated Disease (MOGAD) and POEMS Syndrome. B-cell maturation antigen (BCMA) is expressed on the surface of plasma cells, thus making it an ideal target for targeted therapies. Chimeric antigen receptor (CAR) T cells against BCMA offers another potential therapeutic option to eliminate plasma cells in patients with neurological autoimmune diseases driven by abnormal antibody who still suffer recurrent attacks from conventional treatments. In the current study, the safety and efficacy of a novel CAR-T cell therapy using CT103A cells, are evaluated in patients with relapsed/refractory antibody-mediated idiopathic inflammatory diseases.
- Conditions
- Autoimmune Diseases, Autoimmune Diseases of the Nervous System, Neuromyelitis Optica Spectrum Disorder, Myasthenia Gravis, Chronic Inflammatory Demyelinating Polyradiculoneuropathy, Idiopathic Inflammatory Myopathies
- Being tested
- CT103A cells, Cyclophosphamide and fludarabine
- Keywords
- Adoptive T Cell Therapy, Chimeric antigen receptor, B-cell maturation antigen (BCMA), Autoimmune Diseases of the Nervous System
- Medical subject headings
- Autoimmune Diseases, Autoimmune Diseases of the Nervous System, Neuromyelitis Optica, Myasthenia Gravis, Polyradiculoneuropathy, Chronic Inflammatory Demyelinating, Myositis, Multiple Sclerosis, Myelin Oligodendrocyte Glycoprotein Antibody-Associated Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CT103A cellsBiological
Subjects will undergo leukapheresis to isolate peripheral blood mononuclear cells (PBMCs) to manufacture CT103A cells, during which cyclophosphamide will be administered for the purpose of lymphocytes depletion. After lymphodepletion, subjects will receive one dose treatment with CT103A cells by intravenous (IV) infusion. The initial dose of 0.5×10^6 CAR+ T cells/kg will be infused on day 0.
Cyclophosphamide and fludarabineDrug
Subjects will receive one 3-day cycle of lymphodepletion starting 4 days prior to CT103A infusion on Day 0.
Subjects will be given IV infusion of cyclophosphamide 300 mg/m2/day on day -4, -3 and -2, and fludarabine 30 mg/m2 over 30 minutes administered immediately after cyclophosphamide.
| Study group | Role | What they receive |
|---|---|---|
| CAR T cells therapy，Dose level 1: 0.5 × 10^6 CAR-T cells/Kg | Experimental | The tolerability and safety of CT103A cells will be assessed in an initial dose of 0.5×10^6 CAR-T cells/Kg and three subjects will be enrolled firstly. If no dose-limiting toxicity (DLT) occurs and at least one subject benefits from the treatment, there will be two options for the investigator based on the available data: 1) three more subjects will be enrolled in the 0.5 × 10^6 CAR-T cells/Kg group and DLT will be evaluated in a total of six subjects; 2) another three subjects will be treated with 1 × 10^6 CAR-T cells/Kg instead of 0.5 × 10^6 CAR-T cells/Kg.
If DLT occurs in one of the first three subjects, three more subjects will be enrolled in this cohort to reach the total subjects of six. Receives: Biological: CT103A cells; Drug: Cyclophosphamide and fludarabine |
| CAR T cells therapy，Dose level 2: 1 × 10^6 CAR-T cells/Kg | Experimental | If neither DLT nor efficacy is shown in the first three subjects, the dose of CAR-T cells will be increased to 1 × 10^6 CAR-T cells/kg to assess DLT. Receives: Biological: CT103A cells; Drug: Cyclophosphamide and fludarabine |
| CAR T cells therapy，Dose level 3: 0.25 × 10^6 CAR-T cells/Kg | Experimental | If DLT occurs in two subjects, whether to test the safety and efficacy in 0.25 × 10^6 CAR-T cells/kg group will be determined by the investigator based on the initial data of efficacy, PK and PD. Receives: Biological: CT103A cells; Drug: Cyclophosphamide and fludarabine |
What is being measured
The primary outcome is the main question the study is designed to answer.
Types and incidence of dose-limiting toxicity (DLT) after CT103A cells infusion
To evaluate the DLT occurred within 28 days after CT103A infusion
Measured: Up to 28 days post CT103A infusion
Incidence and severity of AEs, including changes in vital signs, physical examination, laboratory parameters, Electrocardiograms and Echocardiograms.
To evaluate the AEs occurred within 2 years after CT103A infusion
Measured: Up to 2 years post CT103A infusion
Secondary outcomes (5)
PD-Soluble BCMA
The changes of concentration of soluble BCMA in the peripheral blood after CT103A infusion.
Measured: Up to 2 years post CT103A infusion
PD-Pathogenic antibody
The changes of pathogenic antibody titers in peripheral blood or cerebrospinal fluid.
Measured: Up to 2 years post CT103A infusion
PD-Nfl（MS）
The changes of peripheral blood Neurofilament Light chain (NfL) concentration in patients with MS.
Measured: Up to 2 years post CT103A infusion
PK-VCN
The number of BCMA CAR gene copies (VCN，copies/μg DNA) in peripheral blood and cerebrospinal fluid after administration.
Measured: Up to 15 years post CT103A infusion
PK-BCMA CAR-T cells
The concentration of BCMA CAR T cells (cells/mL) in peripheral blood after administration was detected by flow cytometry.
Measured: Up to 28 days post CT103A infusion
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Sep 2020
- Study start22 Sep 2020
- Primary completion22 Feb 2027 (estimated)
- Study completion31 May 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (3)Papers the sponsor has linked to this study.
- Other trials for Autoimmune DiseasesStudies of the same condition, open and under way.
- More trials run by Tongji HospitalThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Early Phase 1
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 36 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Tongji Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- Nanjing IASO Biotechnology Co., Ltd.
- Responsible party
- Principal investigator, Wei Wang, Tongji Hospital
- Investigators
- Wei Wang, MD, Principal investigator, Tongji Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04561557
- Sponsor's protocol ID
- CARTinNS
- Acronym
- CARTinNS
- Status last verified
- Oct 2024
- First submitted
- 10 Sep 2020
- Last updated
- 30 Oct 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Qin C, Dong MH, Zhou LQ, Chu YH, Pang XW, He JY, Shang K, Xiao J, Zhu L, Ye H, Cai SB, Wang D, Bu BT, Meyer Zu Horste G, Li CR, Tian DS, Wang W. Anti-BCMA CAR-T therapy in patients with progressive multiple sclerosis. Cell. 2025 Nov 13;188(23):6414-6423.e11. doi: 10.1016/j.cell.2025.09.020. Epub 2025 Oct 15.
PubMed 41101309Dong MH, Mei ZC, Zhou LQ, Heming M, Xu LL, Liu YX, Pang XW, Chu YH, Cai SB, Ye H, Shang K, Xiao J, Meyer Zu Horste G, Wang W, Qin C, Tian DS. Anti-BCMA CAR-T cell therapy in relapsed/refractory chronic inflammatory demyelinating polyneuropathy. Med. 2025 Sep 12;6(9):100704. doi: 10.1016/j.medj.2025.100704. Epub 2025 May 26.
PubMed 40425008Keam SJ. Equecabtagene Autoleucel: First Approval. Mol Diagn Ther. 2023 Nov;27(6):781-787. doi: 10.1007/s40291-023-00673-y. Epub 2023 Sep 2.
PubMed 37658205
Study at a glance
From the registry record, last updated by the sponsor on 30 Oct 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open Label Clinical Trial to Evaluate the Safety and Efficacy of CT103A Cells for the Treatment of Relapsed/Refractory Antibody-associated Inflammatory Diseases of the Nervous System
- ClinicalTrials.gov ID
- NCT04561557
- Acronym / sponsor ID
- CARTinNS / CARTinNS
- Conditions
- Autoimmune Diseases, Autoimmune Diseases of the Nervous System, Neuromyelitis Optica Spectrum Disorder, Myasthenia Gravis, Chronic Inflammatory Demyelinating Polyradiculoneuropathy, Idiopathic Inflammatory Myopathies
- Keywords
- Adoptive T Cell Therapy, Chimeric antigen receptor, B-cell maturation antigen (BCMA), Autoimmune Diseases of the Nervous System
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 36 participants (estimated)
- Lead sponsor
- Tongji HospitalUniversity, hospital or other
- Collaborators
- Nanjing IASO Biotechnology Co., Ltd.
- Study start
- 22 Sep 2020 (actual)
- Primary completion
- 22 Feb 2027 (estimated)
- Study completion
- 31 May 2027 (estimated)
- First posted
- 23 Sep 2020 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Antibody-mediated inflammatory diseases of the nervous system (also known as autoimmune diseases of the nervous system) are autoimmune diseases in which autoimmune cells and immune molecules attack the nervous system as the main pathogenic mechanism. In the immune response, pathogenic antibodies acting on autoantigens of the nervous system are collectively referred to as autoantibodies of the nervous system, and antibody-mediated inflammatory diseases of the nervous system can occur in the central nervous system, peripheral nervous system, and neuromuscular junctions, and muscles. In this study, we will recruit eight kinds of autoimmune diseases of nervous system including Neuromyelitis Optica Spectrum Disorder (NMOSD), Myasthenia Gravis (MG), Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP), idiopathic inflammatory myopathyand (IIM), multiple sclerosis (MS), autoimmune encephalitis (AE), Myelin Oligodendrocyte Glycoprotein Antibody-Associated Disease (MOGAD) and POEMS Syndrome. B-cell maturation antigen (BCMA) is expressed on the surface of plasma cells, thus making it an ideal target for targeted therapies. Chimeric antigen receptor (CAR) T cells against BCMA offers another potential therapeutic option to eliminate plasma cells in patients with neurological autoimmune diseases driven by abnormal antibody who still suffer recurrent attacks from conventional treatments. In the current study, the safety and efficacy of a novel CAR-T cell therapy using CT103A cells, are evaluated in patients with relapsed/refractory antibody-mediated idiopathic inflammatory diseases.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| CAR T cells therapy，Dose level 1: 0.5 × 10^6 CAR-T cells/Kg | Experimental | Biological: CT103A cells, Drug: Cyclophosphamide and fludarabineThe tolerability and safety of CT103A cells will be assessed in an initial dose of 0.5×10^6 CAR-T cells/Kg and three subjects will be enrolled firstly. If no dose-limiting toxicity (DLT) occurs and at least one subject benefits from the treatment, there will be two options for the investigator based on the available data: 1) three more subjects will be enrolled in the 0.5 × 10^6 CAR-T cells/Kg group and DLT will be evaluated in a total of six subjects; 2) another three subjects will be treated with 1 × 10^6 CAR-T cells/Kg instead of 0.5 × 10^6 CAR-T cells/Kg. If DLT occurs in one of the first three subjects, three more subjects will be enrolled in this cohort to reach the total subjects of six. |
| CAR T cells therapy，Dose level 2: 1 × 10^6 CAR-T cells/Kg | Experimental | Biological: CT103A cells, Drug: Cyclophosphamide and fludarabineIf neither DLT nor efficacy is shown in the first three subjects, the dose of CAR-T cells will be increased to 1 × 10^6 CAR-T cells/kg to assess DLT. |
| CAR T cells therapy，Dose level 3: 0.25 × 10^6 CAR-T cells/Kg | Experimental | Biological: CT103A cells, Drug: Cyclophosphamide and fludarabineIf DLT occurs in two subjects, whether to test the safety and efficacy in 0.25 × 10^6 CAR-T cells/kg group will be determined by the investigator based on the initial data of efficacy, PK and PD. |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (19 listed)
- Male or female subjects aged 18-75 years (including 18 and 75 years);
- Subjects with Relapsing/refractory Antibody-mediated inflammatory diseases of the nervous system without effective treatment, including:
- All acute toxic reactions resolved to baseline or ≤ grade 1 assessed using NCI-CTCAE v5.0 except the ones adjudicated by the investigator to pose no risks on subjects.
- Enrolled subjects must have satisfactory organ function and laboratory findings as defined by the following:i. Blood tests: absolute neutrophil count ≥ 2×109/L (or normal lower limit set by the central lab of the institution), platelets ≥ 100 × 109/L, and hemoglobin ≥ 100 g/L; ii. Liver function: total serum bilirubin, alanine aminotransferase (ALT) and aspartate aminotransferase (AST) must be ≤ 1.5x the institutional normal upper limit (ULN); iii. Kidney function: CrCl ≥ 60 ml/min/1.73m2 (according to the following Cockcroft-Gault formula); iv. Electrolytes: blood potassium ≥ 3.0 mmol/L; blood calcium ≥ 2.0 mmol/L, blood magnesium ≥ 0.5 mmol/L; v. Coagulation function: fibrinogen ≥ 1.0 g/L; APTT ≤ ULN + 10s; PT ≤ ULN + 3s.
- Blood oxygen saturation > 91% in resting state.
Key exclusion criteria (26 listed)
- Patients do not have adequate mononuclear cells without mobilization for CAR-T cell manufacturing.
- History of autoimmune hemolytic disease.
- History of solid organ transplantation.
- Patients were treated with alemtuzumab within 6 months prior to apheresis. Patients were treated with fludarabine or cladribine within 3 months prior to apheresis.
- Patients with Papovaviruses infection.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| CT103A cellsSubjects will undergo leukapheresis to isolate peripheral blood mononuclear cells (PBMCs) to manufacture CT103A cells, during which cyclophosphamide will be administered for the purpose of lymphocytes depletion. After lymphodepletion, subjects will receive one dose treatment with CT103A cells by intravenous (IV) infusion. The initial dose of 0.5×10^6 CAR+ T cells/kg will be infused on day 0. | Biological | CAR T cells therapy，Dose level 1: 0.5 × 10^6 CAR-T cells/Kg, CAR T cells therapy，Dose level 2: 1 × 10^6 CAR-T cells/Kg, CAR T cells therapy，Dose level 3: 0.25 × 10^6 CAR-T cells/Kg |
| Cyclophosphamide and fludarabineSubjects will receive one 3-day cycle of lymphodepletion starting 4 days prior to CT103A infusion on Day 0. Subjects will be given IV infusion of cyclophosphamide 300 mg/m2/day on day -4, -3 and -2, and fludarabine 30 mg/m2 over 30 minutes administered immediately after cyclophosphamide. | Drug | CAR T cells therapy，Dose level 1: 0.5 × 10^6 CAR-T cells/Kg, CAR T cells therapy，Dose level 2: 1 × 10^6 CAR-T cells/Kg, CAR T cells therapy，Dose level 3: 0.25 × 10^6 CAR-T cells/Kg |
Primary outcome
Types and incidence of dose-limiting toxicity (DLT) after CT103A cells infusion
Time frame: Up to 28 days post CT103A infusion
To evaluate the DLT occurred within 28 days after CT103A infusion
Incidence and severity of AEs, including changes in vital signs, physical examination, laboratory parameters, Electrocardiograms and Echocardiograms.
Time frame: Up to 2 years post CT103A infusion
To evaluate the AEs occurred within 2 years after CT103A infusion
5 secondary outcomes
PD-Soluble BCMA
Time frame: Up to 2 years post CT103A infusion
PD-Pathogenic antibody
Time frame: Up to 2 years post CT103A infusion
PD-Nfl（MS）
Time frame: Up to 2 years post CT103A infusion
PK-VCN
Time frame: Up to 15 years post CT103A infusion
PK-BCMA CAR-T cells
Time frame: Up to 28 days post CT103A infusion
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 Oct 2024.
- Derived from this study: PubMed 41101309Qin C, Dong MH, Zhou LQ, Chu YH, Pang XW, He JY, Shang K, Xiao J, Zhu L, Ye H, Cai SB, Wang D, Bu BT, Meyer Zu Horste G, Li CR, Tian DS, Wang W. Anti-BCMA CAR-T therapy in patients with progressive multiple sclerosis. Cell. 2025 Nov 13;188(23):6414-6423.e11. doi: 10.1016/j.cell.2025.09.020. Epub 2025 Oct 15.
- Derived from this study: PubMed 40425008Dong MH, Mei ZC, Zhou LQ, Heming M, Xu LL, Liu YX, Pang XW, Chu YH, Cai SB, Ye H, Shang K, Xiao J, Meyer Zu Horste G, Wang W, Qin C, Tian DS. Anti-BCMA CAR-T cell therapy in relapsed/refractory chronic inflammatory demyelinating polyneuropathy. Med. 2025 Sep 12;6(9):100704. doi: 10.1016/j.medj.2025.100704. Epub 2025 May 26.
- Derived from this study: PubMed 37658205Keam SJ. Equecabtagene Autoleucel: First Approval. Mol Diagn Ther. 2023 Nov;27(6):781-787. doi: 10.1007/s40291-023-00673-y. Epub 2023 Sep 2.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Wei Wang, MD principal investigator
Tongji Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Early Phase 1 study testing CT103A cells and Cyclophosphamide and fludarabine in people with Autoimmune Diseases and Autoimmune Diseases of the Nervous System, run by Tongji Hospital at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 19 inclusion and 26 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 30 Oct 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Chuan Qin, MD (86-27-83663337, qinchuan712@126.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Types and incidence of dose-limiting toxicity (DLT) after CT103A cells infusion (time frame: Up to 28 days post CT103A infusion). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04561557, which also lists 3 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 30 Oct 2024.