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

NCT07337785 | Recruiting Early Phase 1

• 1 other identifier: BHCT-RD06-05-NI-01
• Study Type: interventional
• Target: 36
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Autoimmune Encephalitis (AE); Relapsing or Refractory Multiple Sclerosis (MS); Myasthenia Gravis (MG); Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)

Outcome Measures

Primary Outcomes (3)

• The incidence rates of Treatment-Emergent Adverse Events (TEAEs)

  From the start of cell infusion to 24 months after infusion

• Serious Adverse Events (SAEs)

  From the start of cell infusion to 24 months after infusion

• The incidence rate of Adverse Events of Special Interest (AESI)

  From the start of cell infusion to 24 months after infusion

Secondary Outcomes (22)

• MS:Time to first relapse; Annual Relapse Rate (ARR)

  Day 14, Day 28, Week 8, Week 12, Month 6, Month 9, Month 12, Month 18, and Month 24 post infusion

• MS: The number of new or enlarged T2 lesions on MRI during the study;

  Day 14, Day 28, Week 8, Week 12, Month 6, Month 9, Month 12, Month 18, and Month 24 post infusion

• MS: The number of newly emerged gadolinium-enhancing lesions on MRI

  Day 14, Day 28, Week 8, Week 12, Month 6, Month 9, Month 12, Month 18, and Month 24 post infusion

• MS: The change in Expanded Disability Status Scale (EDSS) score from baseline during the study

  Day 14, Day 28, Week 8, Week 12, Month 6, Month 9, Month 12, Month 18, and Month 24 post infusion

• MG: Changes in Myasthenia Gravis Activities of Daily Living Scale (MG-ADL) scores compared to baseline

  At Week 12, and Months 6, 12, 18, and 24 post infusion

Study Arms (1)

CART Treatment Group

EXPERIMENTAL

Participants receive lymphodepleting chemotherapy with fludarabine and cyclophosphamide from Day -5 to Day -3, followed by a single intravenous infusion of RD06-05.

Biological: CD19/BCMA-targeted CAR-T cells; Drug: Lymphodepleting Conditioning

Interventions

CD19/BCMA-targeted CAR-T cells BIOLOGICAL

Participants 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).

Also known as: RD06-05

CART Treatment Group

Lymphodepleting Conditioning DRUG

From 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;

CART Treatment Group

Eligibility Criteria

• Age: 18 Years - 70 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Patients voluntarily agree to participate in this trial and sign the informed consent form.
• Aged ≥ 18 years and ≤ 70 years, regardless of gender.
• Organ function and laboratory test requirements:
• 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.
• Complete blood count: Neutrophil count ≥ 1 × 10⁹/L; hemoglobin ≥ 60 g/L; platelet count ≥ 20 × 10⁹/L; lymphocyte count \> 0.3 × 10⁹/L.
• Coagulation function: International Normalized Ratio (INR) ≤ 1.5 × ULN OR Prothrombin Time (PT) ≤ 1.5 × ULN.
• Oxygen saturation (SpO₂) ≥ 92% at rest while breathing room air.
• Echocardiography shows Left Ventricular Ejection Fraction (LVEF) ≥ 50%.
• For female patients of childbearing potential at screening, the result of serum or urine pregnancy test is negative.
• 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.
• 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.
• Expanded Disability Status Scale (EDSS) score ranging from 3.0 to 7.5 (inclusive of the cutoff values).
• 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).
• Previous cerebrospinal fluid (CSF) examination or CSF examination report during the screening period indicating at least one of the following conditions:

You may not qualify if:

• 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.
• For CIDP patients: Pure sensory CIDP.
• Presence of clinically significant cardiovascular dysfunction within 12 months prior to screening, including but not limited to: New York Heart Association (NYHA) Class III or IV heart failure, myocardial infarction, unstable angina pectoris, uncontrolled or symptomatic atrial arrhythmia, or any ventricular arrhythmia.
• Presence of significant pulmonary or cardiac manifestations (e.g., pericarditis, pleural effusion) at screening, which the investigator assesses as making the patient unsuitable for participation in this study.
• Patients with severe asthma or chronic obstructive pulmonary disease (COPD); patients with mild or moderate asthma or COPD receiving stable treatment are eligible for enrollment.
• History of malignancy within 5 years prior to signing the ICF, except for fully treated or surgically resected non-melanoma skin cancer or carcinoma in situ (e.g., cervical cancer, bladder cancer, breast cancer) with no residual disease.
• Pregnant or lactating females.
• History of recurrent infections requiring hospitalization and intravenous antibiotics (e.g., 3 or more infections of the same type within the past year).
• Active infection requiring systemic treatment (e.g., infectious pneumonia, tuberculosis) within 2 weeks prior to lymphodepletion.
• Positive for hepatitis B surface antigen (HBsAg); or positive for hepatitis B core antibody (HBcAb) with positive hepatitis B virus (HBV) DNA detection in peripheral blood; positive for hepatitis C virus (HCV) antibody with positive HCV RNA in peripheral blood; positive for human immunodeficiency virus (HIV) antibody; positive for syphilis antibody.
• Vaccination with live-attenuated vaccines within 4 weeks prior to lymphodepletion, or planned vaccination with live-attenuated vaccines during the study.
• Receipt of high-dose corticosteroids (prednisone ≥ 60 mg/day or equivalent dose) within 4 weeks prior to lymphodepletion, or inability to taper prednisone to ≤ 20 mg/day gradually within 3 days prior to lymphodepletion.

Sponsors & Collaborators

• Daishi Tian: lead
• Nanjing Bioheng Biotech Co., Ltd.: collaborator

Study Sites (1)

Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology

Wuhan, Hubei, 430000, China

RECRUITING

MeSH Terms

Conditions

Recurrence; Multiple Sclerosis; Myasthenia Gravis; Polyradiculoneuropathy, Chronic Inflammatory Demyelinating; Autoimmune Diseases of the Nervous System

Condition Hierarchy (Ancestors)

Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Demyelinating Autoimmune Diseases, CNS; Nervous System Diseases; Demyelinating Diseases; Autoimmune Diseases; Immune System Diseases; Paraneoplastic Syndromes, Nervous System; Nervous System Neoplasms; Neoplasms by Site; Neoplasms; Paraneoplastic Syndromes; Neurodegenerative Diseases; Neuromuscular Junction Diseases; Neuromuscular Diseases; Polyradiculoneuropathy; Polyneuropathies; Peripheral Nervous System Diseases; Chronic Disease

Central Study Contacts

Ke Shang

CONTACT

027-83663337; kay_sang@qq.com

Study Design

• Study Type: interventional
• Phase: early phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Professor

Model Details: This is a single-group assignment design. All eligible participants undergo lymphodepleting chemotherapy followed by a single intravenous infusion of RD06-05. The trial includes multiple disease cohorts (MS, MG, AE, and CIDP), but all participants receive the same intervention without randomization or comparator arms.

Study Record Dates

• First Submitted: December 5, 2025
• First Posted: January 13, 2026
• Study Start: December 12, 2025
• Primary Completion (Estimated): December 1, 2026
• Study Completion (Estimated): November 1, 2028
• Last Updated: January 22, 2026

Record last verified: 2026-01

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)