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CD19/BCMA-Targeted UCAR-T for Patients With Neurological Autoimmune Diseases

RecruitingEARLY Phase 1Ages 18 Years - 70 YearsAllTongji Hospital
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In plain words

What the trial is, and whether it could suit you

This single-arm, open-label investigator-initiated trial (IIT) evaluates the safety, tolerability, pharmacokinetics, pharmacodynamics, and efficacy of RD06-05 in patients with autoimmune neurological diseases, including Multiple Sclerosis (MS), Myasthenia Gravis (MG), Chronic Inflammatory Demyelinating Polyneuropathy (CIDP), Autoimmune Encephalitis (AE), and other B-cell-mediated neuroautoimmune disorders. In this study, the dose of CAR-T cells administered is 10×10⁶ CAR⁺T cells per kilogram of body weight. Investigators may decide whether to add other dose groups based on the subjects' safety data, pharmacokinetic (PK) data, pharmacodynamic (PD) data, and preliminary efficacy data. For each indication, 6 to 9 subjects will be enrolled, with a total of 24 to 36 subjects planned for enrollment in the entire study.

Sign in to get the eligibility rules as simple questions to take to your doctor.

📋 Eligibility rules (as published)
General Inclusion Criteria for All Participants: 1. Patients voluntarily agree to participate in this trial and sign the informed consent form. 2. Aged ≥ 18 years and ≤ 70 years, regardless of gender. 3. Organ function and laboratory test requirements: 1. Liver function: Alanine Aminotransferase (ALT) and Aspartate Aminotransferase (AST) ≤ 3 × Upper Limit of Normal (ULN); Total Bilirubin (TBIL) ≤ 2 × ULN (except for patients with Gilbert's syndrome). 2. Renal function: Serum creatinine ≤ 1.5 × ULN OR creatinine clearance rate ≥ 40 ml/min. 3. Complete blood count: Neutrophil count ≥ 1 × 10⁹/L; hemoglobin ≥ 60 g/L; platelet count ≥ 20 × 10⁹/L; lymphocyte count \> 0.3 × 10⁹/L. 4. Coagulation function: International Normalized Ratio (INR) ≤ 1.5 × ULN OR Prothrombin Time (PT) ≤ 1.5 × ULN. 5. Oxygen saturation (SpO₂) ≥ 92% at rest while breathing room air. 6. Echocardiography shows Left Ventricular Ejection Fraction (LVEF) ≥ 50%. 4. For female patients of childbearing potential at screening, the result of serum or urine pregnancy test is negative. 5. Female of childbearing potential must use effective contraception from at least 28 days before apheresis until 12 months after RD06-05 infusion. Male of reproductive potential must use effective barrier contraception during the same period and must not donate semen or sperm throughout the study. Specific Inclusion Criteria for Patients with MS: 1. Diagnosed as Relapsing-Remitting Multiple Sclerosis (RRMS), Primary Progressive Multiple Sclerosis (PPMS), or Secondary Progressive Multiple Sclerosis (SPMS) by a neurologist with diagnostic and treatment qualifications in accordance with the 2017 Revised McDonald Criteria, and relevant diagnostic documents must be provided. 2. Expanded Disability Status Scale (EDSS) score ranging from 3.0 to 7.5 (inclusive of the cutoff values). 3. Having undergone a brain MRI examination that meets the 2017 McDonald Criteria within 12 months prior to screening (must include T2/FLAIR and gadolinium-enhanced T1 sequences), showing spatial multiplicity (≥ 2 typical MS lesion regions) and/or temporal multiplicity (new T2 or gadolinium-enhanced \[Gd+\] lesions). 4. Previous cerebrospinal fluid (CSF) examination or CSF examination report during the screening period indicating at least one of the following conditions: 1. Elevated IgG index 2. Detection of one or more IgG oligoclonal bands (OCB) 5. Having received high-efficacy disease-modifying therapy (DMT) for at least 6 months, with the occurrence of any of the following conditions during the treatment period: 1. Clinically confirmed relapse by a neurologist (new or recurrent persistent neurological deficit lasting ≥ 24 hours, excluding other causes such as fever/infection) 2. EDSS progression (defined as: an increase of ≥ 1.0 point if EDSS ≤ 5.5; or an increase of ≥ 0.5 point if EDSS \> 5.5) High-efficacy DMTs include but are not limited to: anti-CD20 monoclonal antibodies (e.g., Ocrelizumab), lymphocyte-depleting therapies (e.g., Alemtuzumab, Cladribine), and α4 integrin blockers (e.g., Natalizumab). 6. RRMS patients must meet one of the following criteria: at least 1 documented relapse within 1 year prior to screening, or at least 2 documented relapses within 2 years prior to screening, or brain MRI indicating active gadolinium-enhanced lesions or new T2 lesions within 1 year prior to screening. PPMS or SPMS patients must have documented evidence of disability progression within 2 years prior to screening. All relapses or MRI activity must be supported by medical records (e.g., outpatient/inpatient records, MRI reports, EDSS assessment forms). Specific Inclusion Criteria for Patients with MG: 1. Meet the diagnostic criteria for generalized myasthenia gravis (gMG) in line with international myasthenia gravis (MG) consensus guidelines (e.g., the 2020 Myasthenia Gravis Foundation of America \[MGFA\] Guidelines). 2. Classified as MGFA Clinical Class II, III, or IV (per the MGFA Clinical Classification system for myasthenia gravis). 3. Serological testing at screening shows positivity for acetylcholine receptor antibodies (AChR-Ab), muscle-specific tyrosine kinase antibodies (MuSK-Ab), or low-density lipoprotein receptor-related protein 4 antibodies (LRP4-Ab); or there is a documented history of positivity for AChR-Ab, MuSK-Ab, or LRP4-Ab in previous medical records. 4. Score of ≥ 6 points on the Myasthenia Gravis Activities of Daily Living (MG-ADL) Scale, with the score related to ocular symptoms accounting for less than 50% of the total score. 5. Score of ≥ 8 points on the Quantitative Myasthenia Gravis (QMG) Score, with ≥ 4 items each scoring at least 2 points. 6. Having received at least one of the following treatments prior to screening, with relevant medical documentation provided: 1. Immunosuppressants (including but not limited to azathioprine, mycophenolate mofetil, methotrexate, cyclosporine, tacrolimus, cyclophosphamide, etc.) 2. Biologic agents (including but not limited to complement C5 inhibitors, FcRn blockers, etc.). 7. As judged by the investigator, the subject has received stable current treatment for MG for at least 3 months and has experienced any of the following: 1. An increase of ≥ 2 points in the total MG-ADL score, with an increase of ≥ 1 point in non-ocular items; 2. An increase of ≥ 3 points in the total QMG score, or an increase of ≥ 1 point in each of ≥ 2 non-ocular muscle items; 3. Need for increased medication dosage, hospitalization, or emergency intervention due to MG exacerbation. Definition of "stable treatment": i). If the subject is taking acetylcholinesterase inhibitors, they must have received treatment with a stable dosage and regimen for at least 2 weeks prior to screening; ii). If the subject is using glucocorticoids, they must have received treatment with a stable dosage and regimen for at least 2 weeks prior to screening; iii). If the subject is receiving biologics, complemen
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What it tests

  • CD19/BCMA-targeted CAR-T cells
  • Lymphodepleting Conditioning
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Where

1 site
Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology
Wuhan, China
🤝 Taking part is your choice, and you can leave at any time. Only the trial team can confirm you are eligible — always decide together with your own doctor.
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