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.
Patients voluntarily agree to participate in this trial and sign the informed consent form.
Aged ≥ 18 years and ≤ 70 years, regardless of gender.
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).
Renal function: Serum creatinine ≤ 1.5 × ULN OR creatinine clearance rate ≥ 40 ml/min.
Primary diagnosis of an autoimmune disease different from the study disease, which the investigator believes may confound the efficacy evaluation of the study disease.
Comorbidity with other clinically significant central nervous system (CNS) diseases or pathological changes prior to screening, including but not limited to: cerebrovascular accident, aneurysm, epilepsy, convulsions/seizures, aphasia, stroke, severe brain injury, dementia, Parkinson's disease, cerebellar disease, organic brain syndrome, or psychosis.
History of allogeneic bone marrow or stem cell transplantation, or solid organ transplantation (e.g., kidney, lung, heart, liver), or planned future transplantation of such organs/cells.
For MG patients: Uncontrolled myasthenic crisis within 2 weeks prior to screening.
Single group
CD19/BCMA-targeted CAR-T cells
Biological/VaccineParticipants will receive lymphodepleting chemotherapy with fludarabine and cyclophosphamide followed by a single intravenous infusion of CD19/BCMA-targeted CAR-T cells (RD06-05) at dose of 1 × 10\^7 CAR+ T cells/kg (Additional dose levels will be determined by safety, PK/PD, and preliminary efficacy).
Lymphodepleting Conditioning
DrugFrom Day -5 to Day -3 prior to cell infusion (Day 0), subjects will receive chemotherapy preconditioning based on the "Fludarabine + Cyclophosphamide" (FC regimen). The recommended preconditioning regimen is as follows: Fludarabine: 30 mg/m² per day, once daily for 3 consecutive days; Cyclophosphamide: 300 mg/m² per day, once daily for 3 consecutive days;
The incidence rates of Treatment-Emergent Adverse Events (TEAEs)
Serious Adverse Events (SAEs)
The incidence rate of Adverse Events of Special Interest (AESI)
MS:Time to first relapse; Annual Relapse Rate (ARR)
MS: The number of new or enlarged T2 lesions on MRI during the study;
MS: The number of newly emerged gadolinium-enhancing lesions on MRI
MS: The change in Expanded Disability Status Scale (EDSS) score from baseline during the study
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Tongji Hospital
Lead sponsor
Nanjing Bioheng Biotech Co., Ltd.
Collaborator
